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
| 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 | 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 |
| 2 | 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 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) | 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 |
| 3 | 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 | 5 Years | 39 Years | All | 135 | Phase 3 | United States |
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | N/A | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Belgium;Brazil;Canada;China;Italy;Poland;Russian Federation;Saudi Arabia;Taiwan |
| 8 | 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 |
| 9 | 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 | ||
| 10 | 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 | ||
| 11 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Poland;Belgium;Brazil;Australia;Russian Federation;China;Italy | ||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 |
| 16 | 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 | ||
| 17 | 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;United Kingdom;Switzerland;Italy;Germany;Netherlands;Belgium;Poland | ||
| 18 | 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 |
| 19 | NCT02913482 (ClinicalTrials.gov) | December 24, 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 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 |
| 20 | 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 | ||
| 21 | 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 | ||
| 22 | 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 |
| 23 | 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 | ||
| 24 | 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 |
| 25 | 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 | ||
| 26 | 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);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 | Belgium;Netherlands;United Kingdom;Italy;Poland;United States | |||
| 27 | 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 | |||
| 28 | 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;Spain;Poland;Brazil;Belgium;Australia;Germany;Switzerland;Japan | |||
| 29 | 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 | |||
| 30 | EUCTR2021-003417-19-NL (EUCTR) | 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 | NA | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Netherlands;United Kingdom;Italy | |||
| 31 | 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 | |||
| 32 | 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 | |||
| 33 | 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 | |||
| 34 | 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 | |||
| 35 | 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 | |||
| 36 | 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 |