Actively Recruiting
Phase 2 Study of Apitegromab Treatment in Children Under 2 Years with Spinal Muscular Atrophy
Led by Scholar Rock, Inc. · Updated on 2026-05-01
52
Participants Needed
25
Research Sites
17 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating apitegromab in children younger than 2 years old who have 5q autosomal recessive Spinal Muscular Atrophy SMA and delayed motor milestones linked to SMA or a Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders CHOP-INTEND score below 55. This Phase 2, double-blind study aims to assess how the drug acts in the body pharmacokinetics and pharmacodynamics, its effects on motor function, and its safety and tolerability. Participants will receive apitegromab through intravenous IV infusion every 4 weeks during a 48-week treatment period. All enrolled children must have been treated with an approved SMN1-targeted therapy like onasemnogene abeparvovec-xioi or be currently receiving an approved SMN2-targeted therapy such as nusinersen or risdiplam. The study compares low and high doses of apitegromab alongside these standard SMN therapies. During the study, researchers will monitor apitegromab levels in the body, its biological effects, and motor function improvements over 48 to 52 weeks. Safety and tolerability will also be assessed throughout this time. The trial involves regular assessments including motor function tests and safety evaluations to track progress and any adverse effects. Participation lasts approximately one year, with detailed follow-up to understand the treatments impact.
CONDITIONS
Brief Title
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Less than 2 years old at the time of informed consent
- Gestational age of 35 weeks or more and birth weight of at least 2.0 kg
- Confirmed diagnosis of 5q autosomal recessive Spinal Muscular Atrophy (SMA)
- Confirmed presence of SMN2 gene copy or copies
- Must have been treated with approved SMN1-targeted therapy (e.g., onasemnogene abeparvovec-xioi) or be currently treated with approved SMN2-targeted therapy (e.g., nusinersen or risdiplam)
- Body weight for age no less than the 1st percentile based on WHO Child Growth Standards at screening
- Delayed motor milestones for age attributed to SMA or CHOP-INTEND score less than 55
You will not qualify if you...
- Unstable nutritional status or medical need for mostly gastric feeding tube
- Severe orthopedic problems such as severe scoliosis, contractures, or recent/anticipated spine or hip surgery limiting motor function evaluation within 6 months before screening or during study
- Physical limitations like requiring a cast for contractures that prevent motor function testing during the study
Research Team
S
Scholar Rock
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