3. Spinal muscular atrophy Clinical trials / Disease details
Clinical trials : 217 / Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 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 | 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 | ||
| 2 | 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 | |||
| 3 | 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 |