clinical trial · NCT07047144
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Scholar Rock, Inc.·phase2·recruiting·n = 52
Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 2Spinal Muscular Atrophy Type 3Neuromuscular ManifestationsAnti-myostatinApitegromabNusinersenRisdiplam
brief 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.
started
Sep 15, 2025
primary completion
Nov 1, 2028
completion
Mar 1, 2029
last updated
Mar 25, 2026
official 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)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol