3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 237 / 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05626855 (ClinicalTrials.gov) | April 24, 2023 | 7/11/2022 | Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX | An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab | Spinal Muscular Atrophy;Spinal Muscular Atrophy Type 3;Spinal Muscular Atrophy Type II;SMA;Neuromuscular Diseases;Muscular Atrophy;Atrophy;Muscular Atrophy, Spinal;Neuromuscular Manifestations;Anti-myostatin | Drug: Apitegromab | Scholar Rock, Inc. | NULL | Not yet recruiting | 2 Years | N/A | All | 260 | Phase 3 | United States;Belgium;Italy;Netherlands;Poland;Spain |
| 2 | NCT05769465 (ClinicalTrials.gov) | April 1, 2023 | 3/3/2023 | MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy | MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: disease modifying treatments | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | NULL | Not yet recruiting | N/A | N/A | All | 247 | NULL | |
| 3 | NCT05386680 (ClinicalTrials.gov) | January 12, 2023 | 18/5/2022 | Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam | Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to 12 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®) | Spinal Muscular Atrophy | Genetic: OAV101 | Novartis Pharmaceuticals | NULL | Recruiting | 2 Years | 12 Years | All | 28 | Phase 3 | Canada;Japan;Netherlands;Spain |
| 4 | EUCTR2021-003474-31-GR (EUCTR) | 03/01/2023 | 15/09/2022 | Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER) | A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma INN or Proposed INN: Onasemnogene abeparvovec Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Okrido 6mg/mL oral solution Product Name: Okrido INN or Proposed INN: PREDNISOLONE SODIUM PHOSPHATE Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 125 | Phase 3 | United States;United Arab Emirates;Saudi Arabia;Taiwan;Greece;Thailand;Russian Federation;Colombia;Italy;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Viet Nam;Denmark;South Africa;China | ||
| 5 | NCT05335876 (ClinicalTrials.gov) | December 19, 2022 | 12/4/2022 | Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials | Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials | Spinal Muscular Atrophy (SMA) | Biological: onasemnogene abeparvovec | Novartis Pharmaceuticals | NULL | Recruiting | N/A | N/A | All | 260 | Phase 3 | Australia;Belgium |
| 6 | NCT05614531 (ClinicalTrials.gov) | November 1, 2022 | 2/11/2022 | Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type 1 | A Multicenter, Nonrandomized, Open-label,Dose Escalation Clinical Trial to Assess the Safety and Efficacy of EXG001 307 After Intravenous Injection in Patients With Spinal Muscular Atrophy Type 1 | Spinal Muscular Atrophy Type I | Genetic: EXG001-307 injection | Hangzhou Jiayin Biotech Ltd | NULL | Recruiting | 1 Day | 180 Days | All | 12 | Phase 1/Phase 2 | China |
| 7 | NCT05522361 (ClinicalTrials.gov) | September 15, 2022 | 24/5/2022 | Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen | Risdiplam Exchange in Patients With Spinal Muscular Atrophy (SMA) Previously and Exclusively Treated With Nusinersen | Spinal Muscular Atrophy | Drug: Risdiplam | Clinic for Special Children | Genentech, Inc. | Not yet recruiting | 2 Years | 35 Years | All | 10 | Phase 4 | United States |
| 8 | NCT05337553 (ClinicalTrials.gov) | July 6, 2022 | 14/4/2022 | A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy | A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension | Spinal Muscular Atrophy;Neuromuscular Diseases;SMA | Drug: taldefgrobep alfa;Drug: Placebo | Biohaven Pharmaceuticals, Inc. | NULL | Recruiting | 4 Years | 21 Years | All | 225 | Phase 3 | United States;Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom |
| 9 | NCT05115110 (ClinicalTrials.gov) | June 2, 2022 | 1/11/2021 | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy | A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) | Drug: RO7204239;Drug: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Recruiting | 2 Years | 25 Years | All | 259 | Phase 2/Phase 3 | United States;Belgium;Italy;Netherlands;Poland;United Kingdom;Germany |
| 10 | JPRN-jRCT2031220035 | 26/05/2022 | 23/04/2022 | Long-Term Extension Study of Nusinersen in Participants With SMA | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen | Spinal muscular atrophy | Research Name: BIIB058 Generic Name: nusinersen Trade Names: Spinraza Participants in Study 203 who received maintenance doses of 28 mg nusinersen every 4 months will continue this dosing scheme in Study 302. Those who received the currently approved maintenance dose of 12 mg nusinersen in Study 203 will be administered a bolus dose of 50 mg nusinersen on Day 1 followed by 28 mg nusinersen maintenance doses every 4 months thereafter, which is identical to the regimen in Part C of Study 203. | Irzhevsky Victoria | NULL | Pending | Not applicable | Not applicable | Both | 172 | Phase 3 | United States;Canada;Estonia;Hungary;Ireland;Italy;Latvia;Poland;Spain;Taiwan;France;Germany;Greece;Australia;South Korea;United Kingdom;Israel;Lebanon;Russia;Turkey;Japan |
| 11 | EUCTR2021-003417-19-NL (EUCTR) | 25/05/2022 | 11/01/2022 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy | A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM329 Trade Name: Evrysdi INN or Proposed INN: Risdiplam | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 12 | EUCTR2019-002663-10-NL (EUCTR) | 13/05/2022 | 05/02/2022 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 172 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Netherlands;Latvia;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan | ||
| 13 | EUCTR2021-005314-34-ES (EUCTR) | 11/05/2022 | 09/03/2022 | Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or Risdiplam | Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Apitegromab Product Code: SRK-015 INN or Proposed INN: Apitegromab | Scholar Rock, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 204 | Phase 3 | France;United States;Poland;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy | ||
| 14 | EUCTR2020-004708-32-DE (EUCTR) | 05/04/2022 | 18/11/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Lebanon;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan | ||
| 15 | JPRN-jRCT2051210186 | 31/03/2022 | 03/03/2022 | Study of Nusinersen Among Patients with Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | Spinal muscular atrophy (SMA) | Research Name: BIIB058 Generic Name: Nusinersen Trade Name: Spinraza Participants will undergo a loading period during which they will receive 12 mg nusinersen administered IT on Days 1,15, 29, and 64, followed by a maintenance period during which the participants will receive 12 mg nusinersen IT every 4 months. | Nagamine Satoshi | NULL | Pending | >= 2month old | <= 36month old | Both | 60 | Phase 4 | United States;Italy;Spain;Israel;Germany;Japan |
| 16 | EUCTR2021-001294-23-ES (EUCTR) | 31/03/2022 | 20/12/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with Risdiplam | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 135 | Phase 3 | Canada;Poland;Brazil;Belgium;Spain;Australia;Germany;Italy;Switzerland;Japan | ||
| 17 | NCT05232929 (ClinicalTrials.gov) | March 29, 2022 | 31/1/2022 | Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment | Spinal Muscular Atrophy | Drug: Risdiplam | Genentech, Inc. | NULL | Recruiting | N/A | N/A | All | 500 | Phase 4 | United States;Puerto Rico |
| 18 | EUCTR2021-001294-23-IT (EUCTR) | 25/02/2022 | 17/12/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with Risdiplam | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam - . | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: .Nusinersen Product Code: [ ISIS 396443, BIIB058] Other descriptive name: NUSINERSEN SODIUM Trade Name: Spinraza Product Name: Nusinersen Product Code: [ ISIS 396443, BIIB058] Other descriptive name: NUSINERSEN SODIUM | BIOGEN IDEC RESEARCH LIMITED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 135 | Phase 3 | Canada;Spain;Poland;Brazil;Belgium;Australia;Germany;Switzerland;Japan;Italy | ||
| 19 | NCT05156320 (ClinicalTrials.gov) | February 24, 2022 | 1/12/2021 | Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam | Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy | Spinal Muscular Atrophy;Spinal Muscular Atrophy Type 3;Spinal Muscular Atrophy Type 2;SMA;Neuromuscular Diseases;Muscular Atrophy;Atrophy;Muscular Atrophy, Spinal;Neuromuscular Manifestations;Anti-myostatin | Drug: Apitegromab;Drug: Placebo | Scholar Rock, Inc. | NULL | Recruiting | 2 Years | 21 Years | All | 204 | Phase 3 | Germany;Italy;Netherlands;Poland;Spain;United Kingdom;Belgium;France;United States |
| 20 | NCT05067790 (ClinicalTrials.gov) | January 21, 2022 | 24/9/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | Recruiting | 15 Years | 50 Years | All | 135 | Phase 3 | United States;Belgium;Brazil;Germany;Italy;Poland;Spain |
| 21 | EUCTR2021-003417-19-PL (EUCTR) | 18/01/2022 | 25/10/2021 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy | A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM329 Trade Name: Evrysdi INN or Proposed INN: Risdiplam | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Belgium;Poland;Netherlands;United Kingdom;Italy | ||
| 22 | NCT05089656 (ClinicalTrials.gov) | January 12, 2022 | 11/10/2021 | Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) | A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are = 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory | Type 2 Spinal Muscular Atrophy | Genetic: OAV101;Procedure: Sham control | Novartis Pharmaceuticals | NULL | Recruiting | 2 Years | 17 Years | All | 125 | Phase 3 | United States;China;Denmark;India;Malaysia;Mexico;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;Vietnam |
| 23 | EUCTR2021-003417-19-IT (EUCTR) | 03/01/2022 | 18/10/2021 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy | A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY - NA | Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;Classification code 10079418;Term: Spinal muscular atrophy later onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: N/A Product Code: [RO7204239/F01-01] Trade Name: Evrysdi Product Name: Risdiplam Product Code: [RO7034067] INN or Proposed INN: Risdiplam | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Netherlands;United Kingdom;Italy | ||
| 24 | NCT05073133 (ClinicalTrials.gov) | November 4, 2021 | 20/9/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) | A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients From Latin America and Canada With Spinal Muscular Atrophy (SMA) - OFELIA | Muscular Atrophy, Spinal | Genetic: OAV101 | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 24 Months | All | 16 | Phase 4 | Argentina;Brazil |
| 25 | EUCTR2020-005995-37-PT (EUCTR) | 15/10/2021 | 19/07/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) | A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 3 | United States;France;Portugal;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy | ||
| 26 | EUCTR2020-005995-37-IT (EUCTR) | 28/09/2021 | 17/08/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) | A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Prednisolone Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Trade Name: Prednisolone Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Trade Name: Zolgensma Product Name: OAV101 Product Code: [AVXS-101] INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: Previously termed sc.AAV9.CB.SMN and AVXS-101 | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy | ||
| 27 | NCT04851873 (ClinicalTrials.gov) | September 8, 2021 | 6/4/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) | A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) | Spinal Muscular Atrophy | Genetic: OAV101 | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 17 Years | All | 24 | Phase 3 | United States;Australia;Belgium;Canada;France;Italy;Portugal;Taiwan;United Kingdom;Switzerland |
| 28 | EUCTR2020-005995-37-BE (EUCTR) | 03/09/2021 | 28/06/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) | A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 3 | Portugal;France;United States;Taiwan;Canada;Belgium;Australia;Germany;United Kingdom;Italy;Switzerland | ||
| 29 | EUCTR2020-005995-37-FR (EUCTR) | 25/08/2021 | 21/06/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) | A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;United States;France;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy | ||
| 30 | JPRN-jRCT2061200040 | 03/08/2021 | 25/12/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Spinal Muscular Atrophy | - Part A: Participants with later-onset SMA will receive loading doses of 28 mg of nusinersen intrathecally on Days 1, 15 and 29 followed by maintenance doses of 28 mg on Days 149 and 269. - Active Comparator Part B: Participants with infantile- or later-onset SMA will receive loading doses of 12 mg of nusinersen intrathecally on Days 1, 15, 29, and 64 followed by maintenance doses of 12 mg on Days 183 and 279. Sham procedure will be administered on Day 135. - 50/28 mg Group Part B: Participants with infantile- or later-onset SMA will receive loading doses of 50 mg of nusinersen intrathecally on Days 1 and 15 followed by maintenance doses of 28 mg on Days 135 and 279. Sham procedure will be administered on Days 29, 64 and 183. - Part C: Participants who have been receiving the approved dose for at least 1 year prior to entry in this study, will receive a single bolus dose of 50 mg of nusinersen intrathecally on Day 1 (4 months after their most recent maintenance dose) followed by maintenance doses of 28 mg on Days 121 and 241. | Berger Zdenek | NULL | Recruiting | Not applicable | Not applicable | Both | 152 | Phase 2-3 | Spain;Taiwan;United States;Japan;Estonia;Ireland;Latvia;Colombia |
| 31 | EUCTR2020-004708-32-HU (EUCTR) | 15/06/2021 | 17/04/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 3 | United States;Taiwan;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 32 | EUCTR2020-003492-18-DE (EUCTR) | 07/06/2021 | 18/03/2021 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 4 | United States;Spain;Israel;Germany;Italy | ||
| 33 | EUCTR2020-003492-18-ES (EUCTR) | 02/06/2021 | 01/09/2021 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 4 | United States;Spain;Israel;Germany;Italy | ||
| 34 | EUCTR2020-004708-32-ES (EUCTR) | 05/05/2021 | 09/02/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 3 | United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 35 | EUCTR2020-003492-18-IT (EUCTR) | 26/04/2021 | 08/06/2021 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: . Product Code: [.] INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM | BIOGEN IDEC RESEARCH LIMITED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 4 | United States;Spain;Israel;Germany;Italy | ||
| 36 | NCT04729907 (ClinicalTrials.gov) | April 19, 2021 | 11/12/2020 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Enrolling by invitation | N/A | N/A | All | 172 | Phase 3 | United States;Brazil;Canada;Chile;Colombia;Estonia;Germany;Hungary;Japan;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Argentina;Australia;France;Greece;Ireland;Israel;Italy;Korea, Republic of;Latvia;Turkey;United Kingdom |
| 37 | EUCTR2019-002663-10-DE (EUCTR) | 18/02/2021 | 23/10/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 38 | NCT04488133 (ClinicalTrials.gov) | January 7, 2021 | 20/7/2020 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Recruiting | 2 Months | 36 Months | All | 60 | Phase 4 | United States;Germany;Israel;Italy;Japan;Spain |
| 39 | ChiCTR2000041208 | 2020-12-30 | 2020-12-22 | A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy | A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy | spinal muscular atrophy | experimental group: salbutamol sulfate tablets ;control group:placebo; | The Children's Hospital, Zhejiang University School of Medicine | NULL | Recruiting | 2 | 17 | Both | experimental group:60;control group:30; | Phase 4 | China |
| 40 | EUCTR2019-002663-10-FR (EUCTR) | 23/12/2020 | 23/10/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 41 | EUCTR2019-002663-10-GB (EUCTR) | 17/12/2020 | 01/09/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal MuscularAtrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nursinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: Nursinersen Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 42 | NCT04576494 (ClinicalTrials.gov) | December 2020 | 29/9/2020 | Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) | Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms): a Multicenter Single-case Experimental Design in Multiple Baselines Across Subjects, Randomized, Single-blinded Evaluation | Spinal Muscular Atrophy | Other: Monthly assessments of functional motor abilities by a trained therapist;Drug: Nusinersen | CHU de Reims | NULL | Not yet recruiting | 18 Years | N/A | All | 24 | N/A | France |
| 43 | EUCTR2016-000750-35-GB (EUCTR) | 25/11/2020 | 23/06/2016 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RISDIPLAM (RO7034067) IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) Type 2 and 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 2 | United States;Serbia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Bulgaria;Germany;China;Japan | ||
| 44 | EUCTR2019-002611-26-FR (EUCTR) | 16/09/2020 | 10/03/2020 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 308 | Phase 3 | United States;France;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republic of | ||
| 45 | NCT04404764 (ClinicalTrials.gov) | May 27, 2020 | 19/5/2020 | Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study | Characterization of the Clinical-epidemiological Profile of Patients With Spinal Muscular Atrophy (SMA) 5q Types II and III in Follow-up in the Brazilian Unified Public Health System: A Cross-sectional Observational Study (Registry) | Spinal Muscular Atrophy | Drug: Nusinersen Injectable Product | Hospital Israelita Albert Einstein | Ministry of Health, Brazil | Completed | 6 Months | N/A | All | 155 | Brazil | |
| 46 | EUCTR2019-002663-10-ES (EUCTR) | 06/05/2020 | 10/05/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 47 | EUCTR2019-002611-26-IT (EUCTR) | 27/04/2020 | 03/08/2021 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 - not applicable | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 Product Code: [AVXS-101] INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AVEXIS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 308 | Phase 3 | France;United States;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republic of | ||
| 48 | EUCTR2019-002663-10-HU (EUCTR) | 23/04/2020 | 05/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 49 | EUCTR2019-002611-26-GB (EUCTR) | 30/03/2020 | 31/12/2019 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 308 | Phase 3 | United States;France;Taiwan;Canada;Spain;Belgium;Australia;Japan;Italy;United Kingdom;Korea, Republic of | ||
| 50 | EUCTR2019-002663-10-LV (EUCTR) | 27/03/2020 | 12/02/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 51 | NCT04089566 (ClinicalTrials.gov) | March 26, 2020 | 11/9/2019 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Recruiting | 7 Days | N/A | All | 145 | Phase 3 | Hungary;United States;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;United Kingdom |
| 52 | EUCTR2019-002663-10-IT (EUCTR) | 05/03/2020 | 17/06/2021 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: [ ISIS 396443, BIIB058] Other descriptive name: NUSINERSEN SODIUM Trade Name: Spinraza Product Name: Nusinersen Product Code: [ISIS 396443, BIIB058] INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM | BIOGEN IDEC RESEARCH LIMITED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Taiwan;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 53 | EUCTR2019-002611-26-ES (EUCTR) | 28/02/2020 | 14/02/2020 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 308 | Phase 3 | Italy;Korea, Republic of;Spain;Australia;United Kingdom;Japan;United States;France;Taiwan;Canada;Belgium | ||
| 54 | NCT04042025 (ClinicalTrials.gov) | February 10, 2020 | 31/7/2019 | Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi | A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 | Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMA | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Active, not recruiting | N/A | N/A | All | 85 | Phase 3 | United States;Australia;Belgium;Canada;France;Italy;Japan;Taiwan;United Kingdom;Korea, Republic of;Spain |
| 55 | EUCTR2018-004383-65-DE (EUCTR) | 21/11/2019 | 26/07/2019 | A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study | Later Onset Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: SRK-015 INN or Proposed INN: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY Product Name: SRK-015 INN or Proposed INN: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY | Scholar Rock, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Spain;Netherlands;Germany;Italy | ||
| 56 | EUCTR2018-004383-65-NL (EUCTR) | 30/10/2019 | 03/04/2019 | A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study | Later Onset Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: SRK-015 INN or Proposed INN: Apitegromab Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY Product Name: SRK-015 INN or Proposed INN: Apitegromab Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY | Scholar Rock, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 58 | Phase 2 | United States;Spain;Germany;Netherlands;Italy | ||
| 57 | NCT03955679 (ClinicalTrials.gov) | August 7, 2019 | 15/5/2019 | AveXis Managed Access Program Cohort for Access to AVXS-101 | A Managed Access Program (MAP) Cohort Treatment Protocol to Provide AVXS-101 to Patients With a Genetic Diagnosis of Spinal Muscular Atrophy (SMA) With 1, 2 or 3 Copies of SMN2 | Spinal Muscular Atrophy | Genetic: AVXS-101 | United BioSource, LLC | AveXis, Inc. | Approved for marketing | N/A | N/A | All | United States | ||
| 58 | NCT03779334 (ClinicalTrials.gov) | August 7, 2019 | 17/12/2018 | A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Risdiplam | Hoffmann-La Roche | NULL | Active, not recruiting | 1 Day | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Belgium;Brazil;China;Poland;Russian Federation;Taiwan;Canada;Italy;Saudi Arabia |
| 59 | NCT04050852 (ClinicalTrials.gov) | July 17, 2019 | 7/8/2019 | Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments | Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments | Spinal Muscular Atrophy | Drug: Nusinersen Treatments | NYU Langone Health | Winthrop University Hospital | Withdrawn | 5 Years | 21 Years | All | 0 | Early Phase 1 | United States |
| 60 | NCT03988907 (ClinicalTrials.gov) | June 18, 2019 | 14/6/2019 | A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants | A Phase I, 2-Part, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Risdiplam and the Effect of Risdiplam on the Pharmacokinetics of Midazolam Following Oral Administration in Healthy Participants | Spinal Muscular Atrophy | Drug: Risdiplam;Drug: Midazolam | Hoffmann-La Roche | NULL | Completed | 18 Years | 55 Years | All | 35 | Phase 1 | United States |
| 61 | EUCTR2018-004383-65-IT (EUCTR) | 16/06/2019 | 25/01/2021 | A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study | Later Onset Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: SRK-015 Product Code: [NA] INN or Proposed INN: SRK-015 Product Name: SRK-015 Product Code: [NA] INN or Proposed INN: SRK-015 | Scholar Rock, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 2 | United States;Spain;Germany;Netherlands;Italy | ||
| 62 | EUCTR2018-004383-65-ES (EUCTR) | 10/06/2019 | 12/04/2019 | A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study | Later Onset Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: SRK-015 INN or Proposed INN: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY Product Name: SRK-015 INN or Proposed INN: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY | Scholar Rock, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Spain;Netherlands;Germany;Italy | ||
| 63 | NCT03837184 (ClinicalTrials.gov) | May 31, 2019 | 8/2/2019 | Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies | Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | Spinal Muscular Atrophy Type I | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | PRA Health Sciences | Completed | 0 Days | 6 Months | All | 2 | Phase 3 | Japan;Korea, Republic of;Taiwan |
| 64 | EUCTR2016-004184-39-NL (EUCTR) | 08/05/2019 | 20/11/2018 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RISDIPLAM (RO7034067) IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2 | France;United States;Poland;Belgium;Germany;Netherlands;United Kingdom;Switzerland;Italy | ||
| 65 | NCT03921528 (ClinicalTrials.gov) | April 22, 2019 | 16/4/2019 | An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ) | Spinal Muscular Atrophy;Spinal Muscular Atrophy Type 3;Spinal Muscular Atrophy Type 2;SMA;Neuromuscular Diseases;Muscular Atrophy;Atrophy;Muscular Atrophy, Spinal;Neuromuscular Manifestations | Biological: SRK-015 | Scholar Rock, Inc. | NULL | Active, not recruiting | 2 Years | 21 Years | All | 58 | Phase 2 | United States;Italy;Netherlands;Spain |
| 66 | NCT03878030 (ClinicalTrials.gov) | April 1, 2019 | 10/3/2019 | Effect of Nusinersen on Adults With Spinal Muscular Atrophy | Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3 | Adult Spinal Muscular Atrophy | Drug: nusinersen | Northwell Health | NULL | Active, not recruiting | 18 Years | 60 Years | All | 12 | United States | |
| 67 | EUCTR2017-000266-29-NL (EUCTR) | 25/03/2019 | 05/06/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Spain;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 68 | EUCTR2018-002087-12-PL (EUCTR) | 21/03/2019 | 05/12/2018 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: EVRYSDI Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam Other descriptive name: RO7034067 Trade Name: EVRYSDI Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: Risdiplam Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Belgium;Brazil;Poland;Australia;Russian Federation;Italy;China | ||
| 69 | NCT03819660 (ClinicalTrials.gov) | March 7, 2019 | 23/1/2019 | Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 | Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | Muscle Atrophy | Drug: Amifampridine Phosphate 10 MG Oral Tablet | Catalyst Pharmaceuticals, Inc. | NULL | Enrolling by invitation | 6 Years | 50 Years | All | 12 | Phase 2 | Italy |
| 70 | EUCTR2017-004087-35-NL (EUCTR) | 28/02/2019 | 09/07/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Netherlands;Germany;Japan | ||
| 71 | NCT03648658 (ClinicalTrials.gov) | February 18, 2019 | 8/8/2018 | Paracetamol Study in Patients With Low Muscle Mass | Pharmacokinetics and Safety of Treatment With Paracetamol in Children and Adults With Spinal Muscular Atrophy and Cerebral Palsy | SMA II;Cerebral Palsy | Drug: Paracetamol 120Mg/5mL Oral Suspension | Mette Cathrine Oerngreen | Elsass Foundation | Recruiting | 6 Years | 45 Years | All | 48 | Phase 4 | Denmark |
| 72 | EUCTR2018-002087-12-IT (EUCTR) | 05/02/2019 | 17/06/2021 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY - RAINBOWFISH | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: [RO7034067/F13] INN or Proposed INN: Risdiplam Product Name: RO7034067 Product Code: [RO7034067/F12] INN or Proposed INN: Risdiplam | F. HOFFMANN - LA ROCHE LTD. | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Poland;Belgium;Brazil;Australia;Russian Federation;China;Italy | ||
| 73 | EUCTR2017-004087-35-BE (EUCTR) | 31/01/2019 | 16/05/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 74 | EUCTR2017-000266-29-BE (EUCTR) | 31/01/2019 | 23/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;United Kingdom;Italy;Sweden | ||
| 75 | NCT02876094 (ClinicalTrials.gov) | January 29, 2019 | 9/8/2016 | Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy | A Pilot, Open-Label, Dose Response Study Investigating the Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA) | Spinal Muscular Atrophy (SMA) | Drug: celecoxib | Hugh McMillan | Families of Spinal Muscular Atrophy;Gwendolyn Strong Foundation | Terminated | 2 Years | 80 Years | All | 1 | Phase 2 | Canada |
| 76 | NCT03781479 (ClinicalTrials.gov) | January 21, 2019 | 18/12/2018 | Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients | A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | Muscular Atrophy, Spinal | Drug: Amifampridine Phosphate;Drug: Placebo Oral Tablet | Catalyst Pharmaceuticals, Inc. | NULL | Completed | 6 Years | 50 Years | All | 13 | Phase 2 | Italy |
| 77 | EUCTR2017-004087-35-IT (EUCTR) | 24/12/2018 | 23/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 78 | EUCTR2017-004087-35-ES (EUCTR) | 21/12/2018 | 20/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 79 | EUCTR2017-000266-29-ES (EUCTR) | 18/12/2018 | 28/05/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 80 | EUCTR2018-002087-12-BE (EUCTR) | 07/12/2018 | 23/10/2018 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: EVRYSDI Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam Other descriptive name: RO7034067 Trade Name: EVRYSDI Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: Risdiplam Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Poland;Brazil;Belgium;Australia;Russian Federation;Italy;China | ||
| 81 | EUCTR2018-000160-28-IT (EUCTR) | 06/11/2018 | 24/05/2021 | Clinical study evaluating the long term safety of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 | Long term safety study of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 - SMA-002 | Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE Product Name: Amifampridina fosfato Product Code: [Amifampridina fosfato] INN or Proposed INN: AMIFAMPRIDINA | CATALYST PHARMACEUTICALS INC. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Italy | ||
| 82 | EUCTR2017-000266-29-DE (EUCTR) | 05/11/2018 | 05/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 83 | EUCTR2014-002053-19-BG (EUCTR) | 04/10/2018 | 31/07/2018 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Not established Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy | ||
| 84 | NCT04591678 (ClinicalTrials.gov) | October 1, 2018 | 23/10/2019 | Adults With SMA Treated With Nusinersen | Characterizing Longitudinal Outcomes in Adults With SMA Treated With Nusinersen | Spinal Muscular Atrophy | Drug: nusinersen | Ohio State University | Biogen;Cure SMA | Completed | 18 Years | 60 Years | All | 15 | United States | |
| 85 | NCT04174157 (ClinicalTrials.gov) | September 25, 2018 | 12/9/2019 | Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) | A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) | Spinal Muscular Atrophy (SMA) | Other: Prospective observational registry;Drug: Zolgensma | AveXis, Inc. | United BioSource, LLC | Recruiting | N/A | N/A | All | 500 | United States;Greece;Ireland;Israel;Japan;Korea, Republic of;Portugal;Russian Federation;Taiwan | |
| 86 | EUCTR2014-002053-19-HU (EUCTR) | 17/09/2018 | 17/07/2018 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Czech Republic;Hungary;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy | ||
| 87 | EUCTR2018-002295-40-DK (EUCTR) | 11/09/2018 | 11/07/2018 | Is treatment with the painkiller paracetamol safe in patients with spinal muscular atrophy and cerebral palsy? | Pharmacokinetics and safety of treatment with paracetamol in children and adults with spinal muscular atrophy and cerebral palsy - Paracetamol study in patients with low muscle mass | Spinal muscular atrophy type II (SMA II)Cerebral palsy (CP);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | INN or Proposed INN: Paracetamol Other descriptive name: PARACETAMOL | Copenhagen Neuromuscular Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 4 | Denmark | ||
| 88 | EUCTR2017-004087-35-GB (EUCTR) | 24/08/2018 | 08/05/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | Novartis Gene Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 89 | EUCTR2016-000778-40-HR (EUCTR) | 15/08/2018 | 07/12/2018 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | United States;Serbia;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Germany;China;Japan | ||
| 90 | EUCTR2016-000778-40-ES (EUCTR) | 10/08/2018 | 05/08/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 64 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China | ||
| 91 | EUCTR2016-000778-40-PL (EUCTR) | 27/06/2018 | 22/05/2018 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China | ||
| 92 | EUCTR2016-000750-35-BG (EUCTR) | 20/06/2018 | 04/06/2018 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: n.a | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 219 | Phase 2 | Serbia;United States;Spain;Ukraine;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;Japan;China | ||
| 93 | EUCTR2017-000266-29-GB (EUCTR) | 12/06/2018 | 12/02/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 3 | France;Spain;Belgium;Netherlands;Italy;United Kingdom;Sweden | ||
| 94 | JPRN-JapicCTI-183891 | 12/6/2018 | 08/03/2018 | Investigate Safety, Tolerability, PK, PD and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (FIREFISH) | A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy | Spinal Muscular Atrophy | Intervention name : RO7034067 INN of the intervention : risdiplam Dosage And administration of the intervention : po Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : - | CHUGAI PHARMACEUTICAL CO., LTD. | NULL | complete | BOTH | 40 | Phase 2 | Japan, Asia except Japan, North America, South America, Europe | ||
| 95 | EUCTR2017-000266-29-IT (EUCTR) | 21/05/2018 | 09/06/2020 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion - N/A | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 | AVEXIS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 96 | NCT03505099 (ClinicalTrials.gov) | April 10, 2018 | 13/4/2018 | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2 | Spinal Muscular Atrophy | Biological: onasemnogene abeparvovec-xioi | Novartis Gene Therapies | PRA Health Sciences | Completed | N/A | 42 Days | All | 30 | Phase 3 | United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom |
| 97 | EUCTR2017-004600-22-IT (EUCTR) | 04/04/2018 | 07/04/2021 | Clinical study evaluating the effect of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3. | A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3 - SMA-001 | Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE Product Name: Amifampridina fosfato Product Code: [Amifampridina fosfato] INN or Proposed INN: AMIFAMPRIDINA Other descriptive name: 3,4-Diaminopyridine Phosphate | CATALYST PHARMACEUTICALS INC. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Italy | ||
| 98 | JPRN-JapicCTI-173722 | 03/4/2018 | 04/10/2017 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish) | A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | Spinal Muscular Atrophy | Intervention name : RO7034067 INN of the intervention : risdiplam Dosage And administration of the intervention : po Control intervention name : Placebo INN of the control intervention : - Dosage And administration of the control intervention : po | CHUGAI PHARMACEUTICAL CO., LTD. | NULL | complete | 2 | 25 | BOTH | 168 | Phase 2 | Japan, Asia except Japan, North America, South America, Europe |
| 99 | EUCTR2016-000750-35-HR (EUCTR) | 27/03/2018 | 12/04/2018 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) Type 2 and 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 219 | Phase 2 | United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan | ||
| 100 | EUCTR2014-002053-19-CZ (EUCTR) | 14/02/2018 | 30/10/2017 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Not established Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 101 | NCT03381729 (ClinicalTrials.gov) | December 14, 2017 | 13/12/2017 | Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy | Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Completed | 6 Months | 60 Months | All | 32 | Phase 1 | United States |
| 102 | NCT04825119 (ClinicalTrials.gov) | October 1, 2017 | 2/3/2021 | Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen | Hyperkinetic Movements in Patients With Motor Neuron Disease and in Patients With Spinal Muscular Atrophy and Their Response to Treatment With Nusinersen | Tremor;Involuntary Movements;SMA;MND (Motor Neurone DIsease) | Drug: Nusinersen | University Medical Centre Ljubljana | NULL | Recruiting | N/A | N/A | All | 110 | Slovenia | |
| 103 | NCT03421977 (ClinicalTrials.gov) | September 21, 2017 | 30/1/2018 | Long-Term Follow-up Study for Patients From AVXS-101-CL-101 | A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101 | Spinal Muscular Atrophy 1 | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Active, not recruiting | N/A | N/A | All | 13 | United States | |
| 104 | EUCTR2016-004184-39-GB (EUCTR) | 05/09/2017 | 04/05/2017 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophy | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2 | France;United States;Poland;Belgium;Netherlands;Germany;Italy;Switzerland;United Kingdom | ||
| 105 | EUCTR2016-004184-39-IT (EUCTR) | 30/05/2017 | 06/02/2018 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy. | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY - A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 + solvente (RO Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 + solvente (RO Other descriptive name: RO7034067 | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | United States;United Kingdom;Switzerland;Italy | ||
| 106 | EUCTR2016-000778-40-FR (EUCTR) | 21/04/2017 | 24/10/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | France;Belgium;Germany;Italy | ||
| 107 | NCT03032172 (ClinicalTrials.gov) | March 3, 2017 | 24/1/2017 | A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy | An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Risdiplam | Hoffmann-La Roche | NULL | Active, not recruiting | 6 Months | 60 Years | All | 174 | Phase 2 | United States;Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom |
| 108 | EUCTR2016-000778-40-DE (EUCTR) | 05/01/2017 | 21/07/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 / F12 INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 / F13 INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China | ||
| 109 | NCT04587492 (ClinicalTrials.gov) | January 1, 2017 | 7/10/2020 | Metabolomics of Children With SMA | Metabolome of Children With Spinal Muscular Atrophy Treated With Nusinersen | Spinal Muscular Atrophy | Drug: Nusinersen | University Medical Centre Ljubljana | NULL | Completed | N/A | 21 Years | All | 35 | Slovenia | |
| 110 | NCT02913482 (ClinicalTrials.gov) | December 23, 2016 | 21/9/2016 | Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy | A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Risdiplam | Hoffmann-La Roche | NULL | Active, not recruiting | 1 Month | 7 Months | All | 62 | Phase 2/Phase 3 | United States;Belgium;Brazil;China;Croatia;France;Italy;Japan;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;Australia;Germany;Taiwan |
| 111 | EUCTR2016-000778-40-BE (EUCTR) | 09/12/2016 | 13/09/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | United States;Serbia;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Germany;China;Japan | ||
| 112 | EUCTR2016-000750-35-DE (EUCTR) | 09/11/2016 | 13/07/2016 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) Type 2 and 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: Risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 219 | Phase 2 | Serbia;United States;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Bulgaria;Germany;China;Japan | ||
| 113 | NCT02908685 (ClinicalTrials.gov) | October 20, 2016 | 19/9/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants | A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Active, not recruiting | 2 Years | 25 Years | All | 231 | Phase 2/Phase 3 | United States;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;United Kingdom;Argentina;Australia;Sweden;Switzerland;Taiwan |
| 114 | EUCTR2016-000778-40-IT (EUCTR) | 04/10/2016 | 16/08/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | France;Belgium;Germany;Italy | ||
| 115 | EUCTR2015-001870-16-GB (EUCTR) | 04/10/2016 | 29/10/2015 | A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 116 | NCT02865109 (ClinicalTrials.gov) | September 13, 2016 | 10/8/2016 | Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA) | Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA) | Infantile-onset Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | No longer available | N/A | N/A | All | Korea, Republic of;Mexico;Netherlands;Norway;Poland;Portugal;Slovenia;Spain;Sweden;Switzerland;Taiwan;United Kingdom;United States;Colombia;New Zealand;Turkey;Australia;Austria;Belgium;Canada;China;Denmark;Finland;France;Germany;Greece;Ireland;Israel;Italy | ||
| 117 | EUCTR2016-000750-35-BE (EUCTR) | 06/09/2016 | 12/07/2016 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) Type 2 and 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 219 | Phase 2 | United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan | ||
| 118 | EUCTR2016-000750-35-IT (EUCTR) | 29/08/2016 | 28/06/2016 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n.a | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 186 | Phase 2 | United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 119 | JPRN-JMA-IIA00259 | 16/08/2016 | 10/08/2016 | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial | spinal muscular atrophy | Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. | Institute of Medical Genetics, Tokyo Women's Medical University | NULL | Recruiting | >=1 YEARS | <8 YEARS | BOTH | 28 | Phase 2B | Japan |
| 120 | EUCTR2015-001870-16-BE (EUCTR) | 29/07/2016 | 23/05/2016 | A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 121 | EUCTR2014-003626-40-BE (EUCTR) | 09/06/2016 | 30/03/2016 | Nivolumab versus placebo after surgery in subjects with urinary tract cancer | A Phase 3 Randomized, Double-blind, Multi-center Study of Adjuvant Nivolumab versus Placebo in Subjects with High Risk Invasive Urothelial Carcinoma - CheckMate 274 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 Other descriptive name: LMI070 | Bristol-Myers Squibb International Corporation | NULL | Not Recruiting | Female: yes Male: yes | 800 | Phase 1;Phase 2 | Netherlands;Germany;Italy;United States;Czech Republic;Hungary;Poland;Belgium;Denmark;Russian Federation;Bulgaria | ||
| 122 | EUCTR2015-001870-16-SE (EUCTR) | 26/05/2016 | 22/01/2016 | A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Not Recruiting | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of | ||
| 123 | EUCTR2015-001589-25-NL (EUCTR) | 05/04/2016 | 07/10/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Poland;Belgium;Germany;Netherlands;Italy;United Kingdom | ||
| 124 | EUCTR2015-001870-16-ES (EUCTR) | 08/03/2016 | 03/02/2016 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 274 | United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | |||
| 125 | EUCTR2015-001870-16-DE (EUCTR) | 08/03/2016 | 28/10/2015 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 126 | JPRN-JMA-IIA00231 | 29/01/2016 | 01/12/2015 | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial | spinal muscular atrophy | Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. Control intervention1:Placebo, Dose form:GRANULES, Route of administration:ORAL, Intended dose regimen:Placebo is to be taken once a day after supper.. | Institute of Medical Genetics, Tokyo Women's Medical University | NULL | Completed | >=1 YEARS | <7 YEARS | BOTH | 28 | Phase 2B | Japan |
| 127 | NCT02628743 (ClinicalTrials.gov) | January 20, 2016 | 1/12/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) | Multicenter, Open-Label, Single-Arm Study to Evaluate Long-Term Safety, Tolerability, and Effectiveness of 10 mg/kg BID Olesoxime in Patients With Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Olesoxime | Hoffmann-La Roche | NULL | Completed | N/A | N/A | All | 131 | Phase 2 | Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom |
| 128 | NCT02644668 (ClinicalTrials.gov) | January 14, 2016 | 23/12/2015 | A Study of CK-2127107 in Patients With Spinal Muscular Atrophy | A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Placebo;Drug: Reldesemtiv 150 mg;Drug: Reldesemtiv 450 mg | Cytokinetics | Astellas Pharma Global Development, Inc. | Completed | 12 Years | N/A | All | 70 | Phase 2 | United States;Canada |
| 129 | NCT02633709 (ClinicalTrials.gov) | January 7, 2016 | 15/12/2015 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers | A Single-Center, Randomized, Investigator/Subject-Blind, Adaptive Single-Ascending-Dose(SAD), Placebo-Controlled, Parallel Study to Investigate the Safety, Tolerability, Pharmacokinetics (Including the Effect of Food and the Effect of Itraconazole on the Pharmacokinetics of a Single Oral Dose of RO7034067), and Pharmacodynamics of RO7034067 Following Oral Administration in Healthy Subjects | Spinal Muscular Atrophy | Drug: Itraconazole;Other: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Completed | 18 Years | 45 Years | Male | 33 | Phase 1 | Netherlands |
| 130 | EUCTR2015-001589-25-FR (EUCTR) | 01/12/2015 | 07/12/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA | Spinal Muscular Atrophy MedDRA version: 18.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 131 | NCT02941328 (ClinicalTrials.gov) | December 2015 | 30/9/2016 | SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 | A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4 | Spinal Muscular Atrophy;SMA;Kugelberg-Welander Disease | Drug: Pyridostigmine;Drug: Placebo | UMC Utrecht | NULL | Completed | 12 Years | N/A | All | 39 | Phase 2 | Netherlands |
| 132 | EUCTR2015-001589-25-BE (EUCTR) | 20/11/2015 | 25/09/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 133 | EUCTR2015-001589-25-GB (EUCTR) | 19/11/2015 | 04/08/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 134 | EUCTR2014-003657-33-GB (EUCTR) | 17/11/2015 | 19/06/2015 | A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA). | A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Spinraza Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Germany;United Kingdom | ||
| 135 | NCT02594124 (ClinicalTrials.gov) | November 4, 2015 | 30/10/2015 | A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Active, not recruiting | N/A | N/A | All | 292 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan |
| 136 | EUCTR2013-004422-29-BE (EUCTR) | 22/10/2015 | 04/08/2015 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 137 | EUCTR2015-001589-25-IT (EUCTR) | 05/10/2015 | 11/08/2015 | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA | Spinal Muscular Atrophy MedDRA version: 18.0;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 138 | EUCTR2014-003657-33-DE (EUCTR) | 21/09/2015 | 05/05/2015 | A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA). | A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United States;Germany;United Kingdom | ||
| 139 | NCT02462759 (ClinicalTrials.gov) | August 19, 2015 | 14/5/2015 | A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). | A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4 | Spinal Muscular Atrophy | Drug: Nusinersen;Procedure: Sham Procedure | Biogen | NULL | Terminated | N/A | N/A | All | 21 | Phase 2 | United States;Germany |
| 140 | EUCTR2014-002098-12-DE (EUCTR) | 05/08/2015 | 09/04/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Qatar;Taiwan;Canada;Turkey;Australia;Germany;United Kingdom;Italy | ||
| 141 | EUCTR2014-001947-18-DE (EUCTR) | 07/07/2015 | 02/12/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 117 | Phase 3 | France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden | ||
| 142 | EUCTR2014-002098-12-GB (EUCTR) | 10/06/2015 | 30/03/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Spinraza Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Qatar;Taiwan;Turkey;Australia;Germany;Italy;United Kingdom | ||
| 143 | NCT02855112 (ClinicalTrials.gov) | June 2015 | 22/7/2016 | Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients | The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients | Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] | Biological: Adipose derived mesenchymal stem cell | Tehran University of Medical Sciences | NULL | Recruiting | 5 Months | 12 Months | Both | 10 | Phase 1/Phase 2 | Iran, Islamic Republic of |
| 144 | NCT02386553 (ClinicalTrials.gov) | May 20, 2015 | 27/2/2015 | A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | Active, not recruiting | N/A | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom |
| 145 | EUCTR2014-002098-12-IT (EUCTR) | 12/05/2015 | 24/03/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE | Spinal Muscular Atrophy (SMA) MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | United States;Qatar;Taiwan;Argentina;Turkey;Australia;Israel;Germany;United Kingdom;Italy | |||
| 146 | EUCTR2011-004369-34-NL (EUCTR) | 22/04/2015 | 03/11/2014 | SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 | SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4 - SPACE trial | Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive. MedDRA version: 17.1;Level: LLT;Classification code 10068209;Term: Spinal muscular atrophy adult onset;System Organ Class: 100000004850 MedDRA version: 17.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: pyridostigmine bromide Product Name: Mestinon Product Code: RVG 03820 | Universtiy Medical Center Utrecht | NULL | Not Recruiting | Female: yes Male: yes | 45 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Netherlands | ||
| 147 | EUCTR2014-002246-41-ES (EUCTR) | 15/04/2015 | 03/03/2015 | A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH) | A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH | Spinal Muscular Atrophy MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: RO6885247/F03 INN or Proposed INN: n.a. Other descriptive name: n.a. Product Code: RO6885247/F02 INN or Proposed INN: n.a. Other descriptive name: n.a. | Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 1 | France;United States;Spain;Turkey;Netherlands;Italy;United Kingdom;Switzerland;Sweden | ||
| 148 | EUCTR2014-001947-18-ES (EUCTR) | 08/04/2015 | 09/02/2015 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 117 | Phase 3 | France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden | ||
| 149 | NCT02268552 (ClinicalTrials.gov) | April 2, 2015 | 1/10/2014 | An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) | An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: branaplam | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 182 Days | All | 40 | Phase 1/Phase 2 | Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands |
| 150 | EUCTR2014-002053-19-IT (EUCTR) | 01/04/2015 | 27/10/2014 | Clinical trial of LMI070 given my mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: NA Product Code: LMI070 INN or Proposed INN: NA Other descriptive name: NA | NOVARTIS FARMA S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;European Union;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy | ||
| 151 | EUCTR2014-001947-18-IT (EUCTR) | 12/03/2015 | 12/05/2015 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 117 | Phase 3 | France;United States;Hong Kong;Canada;Spain;Germany;Japan;United Kingdom;Italy;Korea, Republic of;Sweden | ||
| 152 | EUCTR2014-001947-18-SE (EUCTR) | 03/02/2015 | 23/12/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 117 | Phase 3 | France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden | ||
| 153 | EUCTR2014-002053-19-DE (EUCTR) | 02/02/2015 | 30/10/2014 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Not established Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy | ||
| 154 | EUCTR2013-004422-29-ES (EUCTR) | 20/01/2015 | 08/09/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | Korea, Republic of;United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Sweden | ||
| 155 | EUCTR2013-004422-29-DE (EUCTR) | 08/01/2015 | 27/11/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 156 | EUCTR2014-002053-19-DK (EUCTR) | 16/12/2014 | 15/10/2014 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Not established Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 157 | EUCTR2014-002053-19-BE (EUCTR) | 15/12/2014 | 04/11/2014 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Branaplam Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 158 | EUCTR2013-004422-29-SE (EUCTR) | 09/12/2014 | 23/10/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 159 | EUCTR2013-004422-29-GB (EUCTR) | 01/12/2014 | 29/07/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy - Study to assess the Clinical Efficacy and Safety of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 160 | NCT02292537 (ClinicalTrials.gov) | November 24, 2014 | 12/11/2014 | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Nusinersen;Procedure: Sham procedure | Biogen | NULL | Completed | 2 Years | 12 Years | All | 126 | Phase 3 | United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom |
| 161 | EUCTR2013-004422-29-IT (EUCTR) | 13/11/2014 | 23/07/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 111 | Phase 3 | United States;Taiwan;Hong Kong;Spain;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of | ||
| 162 | NCT02240355 (ClinicalTrials.gov) | November 2014 | 11/9/2014 | A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) | A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH). | Muscular Atrophy, Spinal | Drug: RO6885247;Drug: placebo | Hoffmann-La Roche | NULL | Terminated | N/A | 55 Years | Both | 9 | Phase 1 | United States;Canada;France;Italy;Netherlands;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic |
| 163 | EUCTR2014-002246-41-IT (EUCTR) | 23/10/2014 | 09/09/2014 | A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH) | A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH | Spinal Muscular Atrophy MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: RO6885247/F03 INN or Proposed INN: n.a. Other descriptive name: n.a. | F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 1 | United States;Spain;Netherlands;United Kingdom;Switzerland;Italy | ||
| 164 | NCT02193074 (ClinicalTrials.gov) | August 19, 2014 | 14/7/2014 | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy | A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen;Procedure: Sham procedure | Biogen | NULL | Terminated | N/A | 210 Days | All | 122 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan |
| 165 | JPRN-JMA-IIA00190 | 22/07/2014 | 01/08/2014 | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy | spinal muscular atrophy | Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA is to be taken once a day after supper. Administration of VPA starts with an initial dosage (standard dose: 12.5 mg / kg) for 4 weeks. The dosage then increases to a maintenance dosage (standard dose: 25 mg/kg) from the 5th week to the 12th week. Administration, however, could be increased to an additional 50 mg/day in case of a blood concentration of VPA below 50 mcg/mL From the 13th week, administered doses are to be decreased (standard dose: 12.5 mg/kg) and continued to the 14th week. . Control intervention1:No. | Institute of Medical Genetics, Tokyo Women's Medical University | NULL | Completed | No Limit | <8 YEARS | BOTH | 13 | Phase 2A | Japan |
| 166 | NCT02227823 (ClinicalTrials.gov) | July 2014 | 20/8/2014 | Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 | Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3. | Spinal Muscular Atrophy Type 3 | Drug: Pyridostigmine Bromide | Centre Hospitalier Régional de la Citadelle | NULL | Recruiting | 6 Years | N/A | Both | 20 | Phase 2 | Belgium |
| 167 | NCT02122952 (ClinicalTrials.gov) | May 5, 2014 | 23/4/2014 | Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101 | Spinal Muscular Atrophy 1 | Biological: AVXS-101 | Novartis Gene Therapies | NULL | Completed | N/A | 6 Months | All | 15 | Phase 1 | United States |
| 168 | NCT04256265 (ClinicalTrials.gov) | April 2014 | 3/2/2020 | An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Risdiplam | Genentech, Inc. | NULL | Approved for marketing | 2 Months | N/A | All | United States | ||
| 169 | NCT02052791 (ClinicalTrials.gov) | January 31, 2014 | 30/1/2014 | An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) | An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | N/A | N/A | All | 47 | Phase 1 | United States |
| 170 | NCT01671384 (ClinicalTrials.gov) | August 2013 | 13/8/2012 | Valproate and Levocarnitine in Children With Spinal Muscular Atrophy | Randomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 Years | Spinal Muscular Atrophy | Drug: Valproate, Levocarnitine;Drug: Placebo | All India Institute of Medical Sciences, New Delhi | NULL | Recruiting | 2 Years | 15 Years | Both | 60 | Phase 3 | India |
| 171 | NCT01839656 (ClinicalTrials.gov) | May 8, 2013 | 22/4/2013 | A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) | A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | N/A | 210 Days | All | 21 | Phase 2 | United States;Canada |
| 172 | NCT01780246 (ClinicalTrials.gov) | January 31, 2013 | 28/1/2013 | An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) | An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | 2 Years | 15 Years | All | 18 | Phase 1 | United States |
| 173 | NCT01703988 (ClinicalTrials.gov) | October 2012 | 8/10/2012 | An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy | An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | Completed | 2 Years | 15 Years | All | 34 | Phase 1/Phase 2 | United States |
| 174 | NCT01645787 (ClinicalTrials.gov) | June 2012 | 5/7/2012 | Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients | Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action | Spinal Muscular Atrophy | Drug: 4-aminopyridine;Drug: Placebo | Columbia University | NULL | Completed | 18 Years | 50 Years | Both | 11 | Phase 2/Phase 3 | United States |
| 175 | NCT01494701 (ClinicalTrials.gov) | November 30, 2011 | 13/12/2011 | An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) | An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | NULL | Completed | 2 Years | 14 Years | All | 28 | Phase 1 | United States |
| 176 | NCT01422200 (ClinicalTrials.gov) | August 2011 | 22/8/2011 | Flu Vaccine Study in Neuromuscular Patients 2011 | Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular Diseases | Duchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular Dystrophy | Biological: 2011-2012 seasonal flu vaccine Subcutaneous;Biological: 2011-2012 seasonal flu vaccine Intramuscular | Children's Hospital Medical Center, Cincinnati | NULL | Completed | 3 Years | 35 Years | All | 22 | Phase 4 | United States |
| 177 | EUCTR2010-020386-24-GB (EUCTR) | 31/01/2011 | 08/10/2010 | Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs SMA patients | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 14.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Belgium;Germany;Netherlands;Italy;United Kingdom | ||
| 178 | EUCTR2010-020386-24-DE (EUCTR) | 29/12/2010 | 28/07/2010 | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 179 | EUCTR2010-020386-24-NL (EUCTR) | 18/11/2010 | 28/06/2010 | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 180 | NCT01302600 (ClinicalTrials.gov) | November 2010 | 18/2/2011 | Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients. | Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients. | Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III Non Ambulant | Drug: Olesoxime;Drug: Placebo | Hoffmann-La Roche | Association Française contre les Myopathies (AFM), Paris | Completed | 3 Years | 25 Years | Both | 165 | Phase 2 | Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom |
| 181 | EUCTR2010-020386-24-BE (EUCTR) | 29/10/2010 | 27/07/2010 | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 182 | ChiCTR-TRC-10001093 | 2010-10-01 | 2010-11-02 | Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial | Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial | children with spinal muscular atrophy | A:Rat nerve growth factor injection for six monthes,;B:Rat nerve growth factor injection for six monthes,; | PLA General Hospital | NULL | Completed | 3 | 8 | Both | A:20;B:20; | China | |
| 183 | EUCTR2010-020386-24-IT (EUCTR) | 14/09/2010 | 01/09/2010 | Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients | Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients | Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 years MedDRA version: 9.1;Level: LLT;Classification code 10032950 | Product Name: olesoxime Product Code: TRO19622 INN or Proposed INN: olesoxime | TROPHOS | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 184 | EUCTR2008-003915-11-DE (EUCTR) | 24/09/2009 | 25/06/2009 | Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I | Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I | Spinal Muscular Atrophy Type I in infants MedDRA version: 9.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital | Trade Name: Orfiril Saft INN or Proposed INN: VALPROATE SODIUM Trade Name: Biocarn INN or Proposed INN: LEVOCARNITINE | University of Utah | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 1/2 | Germany | ||
| 185 | EUCTR2007-001088-32-IT (EUCTR) | 29/07/2008 | 18/07/2008 | A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND | A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND | Patients affected by SMA MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Trade Name: VOLMAX*30CPR 4MG R.P. INN or Proposed INN: Salbutamol | ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy | |||
| 186 | NCT00661453 (ClinicalTrials.gov) | April 2008 | 14/4/2008 | CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I | Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I) | Spinal Muscular Atrophy Type I | Drug: Valproic Acid and Levocarnitine | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc. | Completed | N/A | 12 Months | All | 40 | Phase 1/Phase 2 | United States;Canada;Germany |
| 187 | NCT00439218 (ClinicalTrials.gov) | January 2008 | 22/2/2007 | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I | Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type I Spinal Muscular Atrophy | Spinal Muscular Atrophy Type I | Drug: sodium phenylbutyrate | Westat | National Institute of Neurological Disorders and Stroke (NINDS) | Terminated | 2 Months | 48 Months | All | 5 | Phase 1/Phase 2 | United States |
| 188 | NCT00439569 (ClinicalTrials.gov) | January 2008 | 21/2/2007 | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III | Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type II/III Spinal Muscular Atrophy | Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III | Drug: sodium phenylbutyrate | Westat | National Institute of Neurological Disorders and Stroke (NINDS) | Terminated | 2 Years | 11 Years | All | 9 | Phase 1/Phase 2 | United States |
| 189 | NCT00533221 (ClinicalTrials.gov) | October 2007 | 12/9/2007 | Pilot Study of Growth Hormon to Treat SMA Typ II and III | Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III? | Muscular Atrophy, Spinal | Drug: somatotropin;Drug: Placebo | University Hospital Freiburg | Novo Nordisk A/S | Completed | 6 Years | 35 Years | Both | 20 | Phase 2 | Germany |
| 190 | NCT00481013 (ClinicalTrials.gov) | July 2007 | 30/5/2007 | Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy | Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study | Spinal Muscular Atrophy | Drug: Valproic Acid (VPA);Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Abbott | Completed | 18 Years | 60 Years | Both | 33 | Phase 2 | United States |
| 191 | NCT00528268 (ClinicalTrials.gov) | July 2007 | 10/9/2007 | Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Sodium phenylbutyrate (NaPB) | University of Utah | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | N/A | 6 Months | All | 14 | Phase 1/Phase 2 | United States |
| 192 | NCT00485511 (ClinicalTrials.gov) | June 2007 | 11/6/2007 | A Trial of Hydroxyurea in Spinal Muscular Atrophy | A Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Hydroxyurea | Kaohsiung Medical University Chung-Ho Memorial Hospital | NULL | Completed | 4 Years | N/A | Both | Phase 2/Phase 3 | Taiwan | |
| 193 | EUCTR2006-006845-14-FR (EUCTR) | 20/03/2007 | 15/02/2007 | Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients. | Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (SMA) MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Code: TRO19622 Other descriptive name: 4-cholesten-3-one,oxime | TROPHOS | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 1 | France | ||
| 194 | EUCTR2005-002822-78-DE (EUCTR) | 09/01/2006 | 21/11/2005 | Can treatment with human growth hormone increase strength in spinal muscular atrophy type II and III? - SMA-GH | Can treatment with human growth hormone increase strength in spinal muscular atrophy type II and III? - SMA-GH | Spinal muscular atrophy (SMA) is an autosomal recessive disease. Due to the genetic defect, a molecule called spinal motor neuron” (SMN) protein is lacking, resulting in muscle weakness. In SMAs muscle weakness is found most often at the level of proximal muscles. The disease is life-threatening and chronically debilitating. | Trade Name: Norditropin SimpleXx Product Name: Norditropin SimpleXx 15 mg/1.5 ml Product Code: GH INN or Proposed INN: Somatropin Other descriptive name: Norditropin SimpleXx | Klinik Neuropädiatrie und Muskelkrankheiten | NULL | Not Recruiting | Female: yes Male: yes | 20 | Germany | |||
| 195 | NCT00774423 (ClinicalTrials.gov) | January 2006 | 16/10/2008 | Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA) | Multicentric, Randomized, Double-blind Study Versus Placebo, With Two Parallel Groups Treated to Evaluate the Efficacy and the Tolerance of Riluzole in Children and Young Adults (6 to 20 Years of Age) With SMA. (Type II and Type III) | SMA | Drug: Riluzole | Assistance Publique - Hôpitaux de Paris | NULL | Completed | 6 Years | 20 Years | Both | 141 | Phase 2/Phase 3 | France |
| 196 | NCT00227266 (ClinicalTrials.gov) | September 2005 | 23/9/2005 | Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy | Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial) | Spinal Muscular Atrophy | Drug: Valproic Acid and Levocarnitine;Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;Abbott | Completed | 2 Years | 17 Years | All | 94 | Phase 2 | United States;Canada |
| 197 | NCT00568802 (ClinicalTrials.gov) | January 2004 | 4/12/2007 | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea | Stanford University | NULL | Completed | 1 Year | 10 Years | All | 27 | Phase 1/Phase 2 | United States |
| 198 | NCT00568698 (ClinicalTrials.gov) | January 2004 | 4/12/2007 | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea | Stanford University | NULL | Completed | N/A | 2 Years | All | 29 | Phase 1/Phase 2 | United States |
| 199 | NCT00374075 (ClinicalTrials.gov) | September 2003 | 6/9/2006 | Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy | In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Valproic Acid | University of Utah | Families of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;Abbott | Completed | 2 Years | N/A | Both | 42 | Phase 1 | United States |
| 200 | NCT00004771 (ClinicalTrials.gov) | October 1992 | 24/2/2000 | Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease | Spinal Muscular Atrophy;Amyotrophic Lateral Sclerosis;Spinobulbar Muscular Atrophy | Drug: leuprolide;Drug: testosterone | National Center for Research Resources (NCRR) | Ohio State University | Completed | 18 Years | N/A | Male | 40 | Phase 2 | NULL | |
| 201 | EUCTR2015-001589-25-DE (EUCTR) | 13/08/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | |||
| 202 | EUCTR2019-002663-10-GR (EUCTR) | 02/11/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | Canada;Argentina;Brazil;Poland;Germany;United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico | |||
| 203 | EUCTR2021-001294-23-BE (EUCTR) | 16/12/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with Risdiplam | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 135 | Phase 3 | Canada;Spain;Poland;Brazil;Belgium;Australia;Germany;Switzerland;Japan | |||
| 204 | EUCTR2015-001589-25-PL (EUCTR) | 07/01/2016 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Poland;Netherlands;Germany;Italy;United Kingdom | |||
| 205 | EUCTR2021-003474-31-DK (EUCTR) | 02/11/2021 | Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER) | A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma Product Name: OAV101 Product Code: OAV101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Okrido 6mg/mL oral solution Product Name: Okrido INN or Proposed INN: prednisolone sodium phosphate Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE | Novartis Pharma AG | NULL | NA | Female: yes Male: yes | 125 | Phase 3 | United States;United Arab Emirates;Saudi Arabia;Taiwan;Thailand;Russian Federation;Colombia;Italy;Vietnam;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Denmark;South Africa;China | |||
| 206 | EUCTR2017-004087-35-DE (EUCTR) | 06/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | NA | Female: yes Male: yes | 27 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | |||
| 207 | EUCTR2016-004184-39-PL (EUCTR) | 27/12/2018 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 180 | Phase 2 | United States;France;Belgium;Poland;Netherlands;Germany;United Kingdom;Switzerland;Italy | |||
| 208 | EUCTR2017-000327-27-Outside-EU/EEA (EUCTR) | 26/01/2017 | An Open-Label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy | An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: nusinersen Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 34 | Phase 2 | United States | |||
| 209 | EUCTR2016-004184-39-DE (EUCTR) | 20/09/2018 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophy | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 180 | Phase 2 | France;United States;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy | |||
| 210 | EUCTR2016-000750-35-PL (EUCTR) | 20/02/2018 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 219 | Phase 2 | United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan | |||
| 211 | EUCTR2019-002663-10-PL (EUCTR) | 11/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 172 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan | |||
| 212 | EUCTR2021-001294-23-PL (EUCTR) | 20/04/2022 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with Risdiplam | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 135 | Phase 3 | Canada;Spain;Belgium;Brazil;Poland;Australia;Germany;Italy;Switzerland;Japan | |||
| 213 | EUCTR2014-002053-19-NL (EUCTR) | 27/11/2014 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: LMI070 | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Belgium;Denmark;Germany;Netherlands;Italy | |||
| 214 | EUCTR2021-001294-23-DE (EUCTR) | 27/12/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 135 | Phase 3 | Canada;Belgium;Spain;Poland;Brazil;Australia;Germany;Italy;Switzerland;Japan | |||
| 215 | EUCTR2016-000750-35-FR (EUCTR) | 26/01/2018 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n.a | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 186 | Phase 2 | United States;Spain;Turkey;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden | |||
| 216 | EUCTR2016-004184-39-FR (EUCTR) | 01/10/2018 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 125 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom;Switzerland | |||
| 217 | EUCTR2019-002663-10-IE (EUCTR) | 04/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 172 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | |||
| 218 | EUCTR2017-000621-12-Outside-EU/EEA (EUCTR) | 15/02/2017 | A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular Atrophy | A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: NUSINERSEN Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 20 | Phase 2 | United States;Canada | |||
| 219 | EUCTR2016-004184-39-BE (EUCTR) | 30/11/2018 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophy | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 180 | Phase 2 | United States;France;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy | |||
| 220 | EUCTR2015-001870-16-IT (EUCTR) | 22/01/2021 | An extension study for patients with Spinal Muscular Atrophy whoparticipated to the previous ISIS 396443 studies | An Open-label Extension Study for Patients with Spinal Muscular Atrophywho Previously Participated in Investigational Studies of ISIS 396443 - An Open-label Extension Study for Patients with Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: na Product Code: [0000] INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | BIOGEN IDEC RESEARCH LIMITED | NULL | NA | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Taiwan;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of | |||
| 221 | EUCTR2020-004708-32-NL (EUCTR) | 06/01/2023 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 172 | Phase 3 | United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Netherlands;Latvia;China;Korea, Republic of;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan;Turkey | |||
| 222 | EUCTR2010-020386-24-FR (EUCTR) | 01/06/2010 | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years) MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | NA | Female: yes Male: yes | Phase 2 | France;Belgium;Germany;Netherlands;United Kingdom;Italy | ||||
| 223 | EUCTR2022-001771-14-NL (EUCTR) | 20/12/2022 | Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab | An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab - ONYX | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Apitegromab Product Code: SRK-015 INN or Proposed INN: Apitegromab | Scholar Rock, Inc. | NULL | NA | Female: yes Male: yes | 260 | Phase 3 | France;United States;Belgium;Spain;Poland;Germany;Netherlands;United Kingdom;Italy | |||
| 224 | EUCTR2021-005314-34-BE (EUCTR) | 11/02/2022 | Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or Risdiplam | Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Apitegromab Product Code: SRK-015 INN or Proposed INN: Apitegromab | Scholar Rock, Inc. | NULL | NA | Female: yes Male: yes | 204 | Phase 3 | United States;France;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy | |||
| 225 | EUCTR2014-001947-18-FR (EUCTR) | 18/06/2015 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Isis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 117 | Phase 3 | United States;France;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden | |||
| 226 | EUCTR2014-002053-19-PL (EUCTR) | 03/01/2018 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Branaplam Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Belgium;Poland;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | |||
| 227 | EUCTR2021-003417-19-BE (EUCTR) | 15/12/2021 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy | A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079415;Term: Spinal muscular atrophy type III;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079418;Term: Spinal muscular atrophy later onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM329 Trade Name: Evrysdi INN or Proposed INN: Risdiplam | F.Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Germany;Netherlands;Italy;United Kingdom | |||
| 228 | EUCTR2017-000266-29-FR (EUCTR) | 05/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | |||
| 229 | EUCTR2020-004708-32-PL (EUCTR) | 21/12/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Lebanon;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan | |||
| 230 | EUCTR2021-006781-21-DK (EUCTR) | 20/12/2022 | Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical Trials | Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion Product Name: OAV101 Product Code: OAV101 Trade Name: Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion Product Name: OAV101 Product Code: OAV101 | Novartis Pharma AG | NULL | NA | Female: yes Male: yes | 260 | Phase 4 | United States;Saudi Arabia;Taiwan;Greece;Thailand;Spain;Colombia;Switzerland;Italy;India;France;Malaysia;Viet Nam;Australia;Denmark;South Africa;Netherlands;China;United Kingdom;Egypt;Mexico;Canada;Belgium;Brazil;Singapore;Germany;Japan | |||
| 231 | EUCTR2021-003417-19-DE (EUCTR) | 18/11/2021 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy | A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079415;Term: Spinal muscular atrophy type III;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079418;Term: Spinal muscular atrophy later onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM329 Trade Name: Evrysdi INN or Proposed INN: Risdiplam | F.Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy | |||
| 232 | EUCTR2020-004708-32-EE (EUCTR) | 02/02/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan | |||
| 233 | EUCTR2017-000266-29-SE (EUCTR) | 12/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | |||
| 234 | EUCTR2014-001947-18-GB (EUCTR) | 19/12/2014 | A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular Atrophy | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy (SMA) MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 117 | Phase 3 | France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden | |||
| 235 | EUCTR2015-001870-16-FR (EUCTR) | 03/05/2016 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443. | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: ISIS 396443 Other descriptive name: ISIS 396443 | Ionis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 274 | Phase 3 | United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | |||
| 236 | EUCTR2021-005314-34-NL (EUCTR) | 10/03/2022 | Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or Risdiplam | Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Apitegromab Product Code: SRK-015 INN or Proposed INN: Apitegromab | Scholar Rock, Inc. | NULL | NA | Female: yes Male: yes | 204 | Phase 3 | France;United States;Spain;Poland;Belgium;Germany;Netherlands;United Kingdom;Italy | |||
| 237 | EUCTR2019-002611-26-BE (EUCTR) | 20/01/2020 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AveXis, Inc. | NULL | NA | Female: yes Male: yes | 308 | Phase 3 | Canada;Belgium;United States;Japan;Taiwan;Korea, Republic of;Italy;United Kingdom;Australia;France;Spain |