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Clinical trial

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

NCT07047144 is registered in ClinicalTrials.gov with Scholar Rock, Inc. as lead sponsor.

NCT NCT07047144
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)
Lead sponsor
Scholar Rock, Inc.
Phase
Phase 2
Overall status
Recruiting
Start date
September 15, 2025
Completion date
March 31, 2029
Enrollment
52 (estimated)
Sites
23

Conditions

As the registry lists them; a condition with its own page is linked.

anti-myostatin, neuromuscular manifestations, sma, spinal muscular atrophy, spinal muscular atrophy type 2, spinal muscular atrophy type 3