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NCT03921528 · ClinicalTrials.gov registry record · Phase 2
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
A Phase 2 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Scholar Rock.
- Completed
- Registry status
- Phase 2
- Development phase
- 58
- Enrollment target
- 16
- Study locations
NCT03921528: Completed Phase 2 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Scholar Rock.
NCT03921528 is a Phase 2 study of Spinal Muscular Atrophy and Neuromuscular Diseases that has completed, run by Scholar Rock. The registered enrollment target is 58 participants, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (46% lower). The trial reports 16 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03921528, a Phase 2 study of Spinal Muscular Atrophy and Neuromuscular Diseases, has completed, sponsored by Scholar Rock.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 58 participants
- Enrollment target
- 16
- Study locations
Study Summary
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
Primary Outcome
The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA. For the ambulatory Type 3 patients 5-21 years of age in Cohort 1 (N=23), the primary endpoint was the change from baseline in RHS total score at month 12. The RHS has a minimum achievable score of 0 and a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the l
Conditions Studied
Interventions
- BIOLOGICAL SRK-015
Study Locations (16)
Other
- ASST Grande Ospedale Metropolitano Niguarda - Milan
- Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore - Roma
- Universitair Medisch Centrum Utrecht - Utrecht
- Hospital Sant Joan de Déu - Barcelona
- Hospital Universitari i Politecnic La Fe - Valencia
Arizona
- Phoenix Children's Hospital - Phoenix
California
- Stanford University - Palo Alto
Colorado
- Children's Hospital Colorado - Aurora
Maryland
- The Johns Hopkins University - Baltimore
Massachusetts
- Boston Children's Hospital - Boston
Michigan
- Helen DeVos Children's Hospital - Grand Rapids
New York
- Columbia University - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 58 participants |
| Start Date | 2019-04-22 |
| Est. Completion | 2024-02-28 |
| Phase | Phase 2 |
What the finished NCT03921528 record still lists
NCT03921528 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 58 participants, a relatively small participant target, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (46% lower).
The record links to 9 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 1 intervention - of which SRK-015 is the first listed.
NCT03921528 lists 16 locations in 12 states (Other, Arizona, California).
Frequently Asked Questions
What is clinical trial NCT03921528 about?
NCT03921528 is a clinical study titled "An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy". The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
What is the current status of trial NCT03921528?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 58 participants. The study started on 2019-04-22. Estimated completion is 2024-02-28.
What conditions does trial NCT03921528 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy, Neuromuscular Diseases, Muscular Atrophy, Spinal, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations.
What interventions are being tested in trial NCT03921528?
The interventions under investigation include: SRK-015 (BIOLOGICAL).
Who is sponsoring clinical trial NCT03921528?
This trial is sponsored by Scholar Rock, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03921528 being conducted?
This trial has 16 study locations across Arizona, California, Colorado, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT03921528's enrollment target sits among peer trials
58 11th of 22 higher than 12 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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