3. Spinal muscular atrophy
179 clinical trials,   102 drugs   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
4 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-004383-65-DE
(EUCTR)
21/11/201926/07/2019A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 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]
Scholar Rock, Inc.NULLNot Recruiting Female: yes
Male: yes
55Phase 2United States;Spain;Netherlands;Germany;Italy
2EUCTR2018-004383-65-NL
(EUCTR)
30/10/201903/04/2019A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
55Phase 2United States;Canada;Spain;Germany;Netherlands;Italy
3EUCTR2018-004383-65-ES
(EUCTR)
10/06/201912/04/2019A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 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]
Scholar Rock, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
55Phase 2United States;Canada;Spain;Netherlands;Germany;Italy
4NCT03921528
(ClinicalTrials.gov)
April 22, 201916/4/2019An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular AtrophyPhase 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 ManifestationsBiological: SRK-015Scholar Rock, Inc.NULLActive, not recruiting2 Years21 YearsAll58Phase 2United States;Italy;Netherlands;Spain