A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Phase 2 Recruiting 52 enrolled Scholar Rock, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

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.

Timeline

Start: 2025-09-15
Primary completion: 2028-11
Completion: 2029-03

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Apitegromab	Monoclonal antibody	—	Intravenous
Background	Nusinersen	Antisense oligonucleotide	—	Intrathecal
Background	Risdiplam	Small molecule	—	Oral

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Summary

Timeline

Drugs

Indications