A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy (OPAL)
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)
Sponsor: Scholar Rock, Inc.
This observational or N/A phase trial investigates Anti-myostatin and Neuromuscular Manifestations and is currently actively recruiting participants. Scholar Rock, Inc. leads this study, which shows 5 recorded versions since 2025 — indicating limited longitudinal coverage. The change history captured here reflects the iterative nature of clinical trial conduct.
Status Flow
Change History
5 versions recorded-
May 4, 2026 — Present [daily]
Recruiting
Phase: PHASE2 → None
-
Feb 2026 — May 2026 [monthly]
Recruiting PHASE2
-
Dec 2025 — Feb 2026 [monthly]
Recruiting PHASE2
-
Oct 2025 — Dec 2025 [monthly]
Recruiting PHASE2
Status: Not Yet Recruiting → Recruiting
-
Aug 2025 — Oct 2025 [monthly]
Not Yet Recruiting PHASE2
First recorded
Eligibility 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.
Contact Information
- Scholar Rock, Inc.
For direct contact, visit the study record on ClinicalTrials.gov .