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NCT05156320 · ClinicalTrials.gov registry record · Phase 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
A Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Scholar Rock.
- Completed
- Registry status
- Phase 3
- Development phase
- 188
- Enrollment target
- 20
- Study locations
NCT05156320: Completed Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Scholar Rock.
NCT05156320 is a Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases that has completed, run by Scholar Rock. The registered enrollment target is 188 participants, above the 101-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (86% higher). The trial reports 20 study locations across 18 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05156320, a Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, has completed, sponsored by Scholar Rock.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 188 participants
- Enrollment target
- 20
- Study locations
Study Summary
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
Primary Outcome
The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Apitegromab
Study Locations (20)
California
- Children's Hospital of Los Angeles - Los Angeles
- Stanford University Medical Center - Palo Alto
- Rady's Children's Hospital/UCSD - San Diego
Alabama
- Children's of Alabama - Birmingham
Arizona
- Phoenix Children's Hospital - Phoenix
Colorado
- Children's Hospital Colorado - Aurora
Florida
- Nemours Children's Hospital - Orlando
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Iowa
- University of Iowa - Iowa City
Kansas
- University of Kansas Medical Center - Kansas City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 188 participants |
| Start Date | 2022-04-14 |
| Est. Completion | 2024-12-18 |
| Phase | Phase 3 |
What the finished NCT05156320 record still lists
NCT05156320 is an interventional study that assigns participants to a tested intervention. The registered 188 participants enrollment target is mid-sized for trials with a published cap, above the 101-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (86% higher).
The record links to 10 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT05156320 names 20 study sites across 18 states, led by California, Alabama, Arizona.
Frequently Asked Questions
What is clinical trial NCT05156320 about?
NCT05156320 is a clinical study titled "Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam". This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusiners...
What is the current status of trial NCT05156320?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 188 participants. The study started on 2022-04-14. Estimated completion is 2024-12-18.
What conditions does trial NCT05156320 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 NCT05156320?
The interventions under investigation include: Placebo (DRUG), Apitegromab (DRUG).
Who is sponsoring clinical trial NCT05156320?
This trial is sponsored by Scholar Rock, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05156320 being conducted?
This trial has 20 study locations across Alabama, Arizona, California, Colorado, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05156320's enrollment target sits among peer trials
188 5th of 22 higher than 18 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.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
ACTIVE NOT RECRUITING · Phase 3
-
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
ACTIVE NOT RECRUITING · Phase 3
-
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
ACTIVE NOT RECRUITING · Phase 3
-
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
RECRUITING
-
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
RECRUITING · NA
-
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
RECRUITING · Phase 2
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