3. Spinal muscular atrophy Clinical trials / Disease details
Clinical trials : 237 / Drugs : 123 - (DrugBank : 29) / Drug target genes : 51 - Drug target pathways : 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 | 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 | ||
| 2 | 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 |
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 |
| 8 | 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 | |||
| 9 | 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 | |||
| 10 | 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 |