3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
Showing 1 to 10 of 217 diseases
| 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 | NCT05272969 (ClinicalTrials.gov) | March 31, 2022 | 28/1/2022 | Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease | Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD) Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD) ... | Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHD | Diagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencing Diagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain I ... | LMU Klinikum | NULL | Not yet recruiting | 18 Years | N/A | All | 95 | Germany | |
| 2 | NCT05232929 (ClinicalTrials.gov) | March 31, 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 | 2 Months | N/A | All | 500 | Phase 4 | United States |
| 3 | NCT05115110 (ClinicalTrials.gov) | March 31, 2022 | 1/11/2021 | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulatory Children With Spinal Muscular Atrophy A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067 ... | 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 A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigat ... | Spinal Muscular Atrophy (SMA) | Drug: RO7204239;Drug: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Not yet recruiting | 2 Years | 10 Years | All | 180 | Phase 2/Phase 3 | United States;Belgium;Germany;Italy;Netherlands;Poland;United Kingdom |
| 4 | 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 Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrop ... | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrop ... | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | Recruiting | 5 Years | 39 Years | All | 135 | Phase 3 | United States |
| 5 | 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 Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of ... | 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 A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGAT ... | 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 Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM32 ... | 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 | ||
| 6 | 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 Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of ... | 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 A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGAT ... | 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] Spinal Muscular Atrophy(SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spin ... | Product Name: N/A Product Code: [RO7204239/F01-01] Trade Name: Evrysdi Product Name: Risdiplam Product Code: [RO7034067] INN or Proposed INN: Risdiplam Product Name: N/A Product Code: [RO7204239/F01-01] Trade Name: Evrysdi Product Name: Risdiplam Product C ... | 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 | ||
| 7 | 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) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | 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 A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | 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 Trade Name: Zolgensma2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Cod ... | 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 United States;France;Portugal;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
| 8 | 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) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | 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 A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | 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 Trade Name: Prednisolone Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE ... | 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 Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
| 9 | 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) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | 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 A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | 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 Trade Name: Zolgensma2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Cod ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 3 | Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
| 10 | 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) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | 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 A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | 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 Trade Name: Zolgensma Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABE ... | 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 Portugal;United States;France;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... |