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NCT07047144 · ClinicalTrials.gov registry record · Phase 2
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
A Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, sponsored by Scholar Rock.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 52
- Enrollment target
- 14
- Study locations
NCT07047144: Recruiting Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, sponsored by Scholar Rock.
NCT07047144 is a Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3 that is actively recruiting participants, run by Scholar Rock. The registered enrollment target is 52 participants, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (51% lower). The trial reports 14 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07047144, a Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, is actively recruiting participants, sponsored by Scholar Rock.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 52 participants
- Enrollment target
- 14
- Study locations
Study Summary
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.
Primary Outcome
Apitegromab concentrations in serum
Conditions Studied
Interventions
- DRUG Risdiplam
- DRUG Apitegromab
- DRUG Nusinersen
Study Locations (14)
Other
- UZ Leuven - Campus Gasthuisberg - Leuven
- CHR Citadelle - Liège
- Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta - Milan
- Centro Clinico NeMO Milano - Fondazione Serena Onlus - Milan
- Hospital Sant Joan de Déu Barcelona - Barcelona
- Hospital Universitari i Politecnico La Fecnic La Fe - Valencia
Texas
- University of Texas Southwestern Medical Center - Dallas
- Neurology Rare Disease Center - Flower Mound
- Cook Children's Medical Center - Fort Worth
Arizona
- Phoenix Children's Hospital - Phoenix
California
- Stanford Neuroscience Health Center (SNHC) - Palo Alto
Colorado
- Children's Hospital Colorado - Aurora
Iowa
- University of Iowa - Iowa City
North Carolina
- Atrium Health Wake Forest Baptist - Winston-Salem
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 52 participants |
| Start Date | 2025-09-15 |
| Est. Completion | 2029-03 |
| Phase | Phase 2 |
What NCT07047144 shows while recruiting
NCT07047144 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 52 participants, a relatively small participant target, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (51% lower).
The record links to 6 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 3 interventions - of which Risdiplam is the first listed.
NCT07047144 lists 14 locations in 7 states (Other, Texas, Arizona).
Frequently Asked Questions
What is clinical trial NCT07047144 about?
NCT07047144 is a clinical study titled "A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy". This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator...
What is the current status of trial NCT07047144?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 52 participants. The study started on 2025-09-15. Estimated completion is 2029-03.
What conditions does trial NCT07047144 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, SMA, Spinal Muscular Atrophy Type 2.
What interventions are being tested in trial NCT07047144?
The interventions under investigation include: Risdiplam (DRUG), Apitegromab (DRUG), Nusinersen (DRUG).
Who is sponsoring clinical trial NCT07047144?
This trial is sponsored by Scholar Rock, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07047144 being conducted?
This trial has 14 study locations across Arizona, California, Colorado, Iowa, North Carolina. 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 NCT07047144's enrollment target sits among peer trials
52 13th of 22 higher than 10 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
-
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
RECRUITING · Phase 2
-
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
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-
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
COMPLETED · Phase 2
-
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
RECRUITING
-
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
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-
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
RECRUITING · NA
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