Status: Recruiting
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 or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.
Participants needed: 52
Trial details
Phase: Phase 2Age: Up to 2Biological sex: AllType: InterventionalSponsor: Scholar Rock, Inc.Updated: May 1, 2026Locations: 25
Eligibility criteria
Is <2 years old at the time of the informed consent [+6]
Nutritional status that is not anticipated to be stable throughout the study or... [+2]