Sponsor:
Scholar Rock, Inc.
Code:
NCT07047144
Conditions
Spinal Muscular Atrophy
SMA
Spinal Muscular Atrophy Type 2
Spinal Muscular Atrophy Type 3
Neuromuscular Manifestations
Eligibility Criteria
Sex: All
Age: 0 - 2
Healthy Volunteers: Not accepted
Interventions
Apitegromab
Nusinersen
Risdiplam
Brief summary:
Conditions
Spinal Muscular Atrophy
SMA
Spinal Muscular Atrophy Type 2
Spinal Muscular Atrophy Type 3
Neuromuscular Manifestations
Study ID
NCT07047144
Start date
Sep 15, 2025
Status verified date
Apr, 2026
Completion date
Mar, 2029
Anticipated
Primary completion date
Nov, 2028
Anticipated
Eligibility Criteria
Sex: All
Age: 0 - 2
Healthy Volunteers: Not accepted
Enrollment
52 participants
Anticipated
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary purpose
Treatment
Arms
experimental: Apitegromab low dose + SMN Therapy
experimental: Apitegromab high dose + SMN Therapy
Interventions
Apitegromab
Nusinersen
Risdiplam
Primary outcome measure
Central contacts
Locations
Phoenix Children's Hospital
Recruiting
Phoenix, Arizona, United States, 85016
Contacts
Children's Hospital of Orange County (CHOC)
Recruiting
Orange, California, United States, 92868
Contacts
Stanford Neuroscience Health Center (SNHC)
Recruiting
Palo Alto, California, United States, 94304
Contacts
Children's Hospital Colorado
Recruiting
Aurora, Colorado, United States, 80045
Contacts
Children's Healthcare of Atlanta
Recruiting
Atlanta, Georgia, United States, 30329
Contacts
University of Iowa
Recruiting
Iowa City, Iowa, United States, 52242
Contacts
Helen DeVos Children's Hospital at Spectrum Health
Recruiting
Grand Rapids, Michigan, United States, 49503
Contacts
Atrium Health Wake Forest Baptist
Recruiting
Winston-Salem, North Carolina, United States, 27157
Contacts
The Children's Hospital of Philadelphia
Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contacts
University of Texas Southwestern Medical Center
Recruiting
Dallas, Texas, United States, 75207
Contacts
Neurology Rare Disease Center
Recruiting
Flower Mound, Texas, United States, 75028
Contacts
Cook Children's Medical Center
Recruiting
Fort Worth, Texas, United States, 76104
Contacts
Texas Children's Hospital (TCH)-Clinical Care Center (CCC)
Recruiting
Houston, Texas, United States, 77030
Contacts
Sponsor
Scholar Rock, Inc.
Last update posted
May 1, 2026
Last verified
Apr, 2026
Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Scholar Rock, Inc. on 2026-05-01.