Actively Recruiting
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Led by Scholar Rock, Inc. · Updated on 2026-05-01
52
Participants Needed
25
Research Sites
180 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Younger than 2 years old at the time of informed consent
- Born at gestational age of 35 weeks or more with birth weight of at least 2.0 kg
- Confirmed diagnosis of 5q autosomal recessive spinal muscular atrophy (SMA)
- Confirmed presence of SMN2 gene copy or copies
- Currently treated or previously treated with approved SMN1 or SMN2-targeted therapies (e.g., onasemnogene abeparvovec, nusinersen, risdiplam)
- Body weight for age not less than 1st percentile based on WHO Child Growth Standards at screening
- Delayed motor milestones for age due to SMA or CHOP-INTEND score less than 55
You will not qualify if you...
- Unstable nutritional status or medical need for gastric feeding tube with most feeds administered this way
- Major orthopedic problems such as severe scoliosis, severe contractures, or recent/anticipated spine or hip surgery that limit motor function evaluation
- Other physical limitations (e.g., requiring a cast for contractures) preventing motor function assessments during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 25 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Children's Hospital of Orange County (CHOC)
Orange, California, United States, 92868
Actively Recruiting
3
Stanford Neuroscience Health Center (SNHC)
Palo Alto, California, United States, 94304
Actively Recruiting
4
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
6
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
7
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
8
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States, 27157
Actively Recruiting
9
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
10
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Not Yet Recruiting
11
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75207
Actively Recruiting
12
Neurology Rare Disease Center
Flower Mound, Texas, United States, 75028
Actively Recruiting
13
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
14
Texas Children's Hospital (TCH)-Clinical Care Center (CCC)
Houston, Texas, United States, 77030
Actively Recruiting
15
UZ Gent
Ghent, East-Flanders, Belgium, 9000
Actively Recruiting
16
UZ Leuven - Campus Gasthuisberg
Leuven, Belgium, 3000
Actively Recruiting
17
CHR Citadelle
Liège, Belgium, 4000
Actively Recruiting
18
Hopital Trousseau - I-Motion
Paris, France, 75012
Actively Recruiting
19
Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Actively Recruiting
20
Centro Clinico NeMO Milano - Fondazione Serena Onlus
Milan, Italy, 20162
Actively Recruiting
21
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy, 00168
Actively Recruiting
22
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands, 3584 CX
Actively Recruiting
23
Hospital Sant Joan de Déu Barcelona
Barcelona, Spain, 08950
Actively Recruiting
24
Hospital Universitari i Politecnico La Fecnic La Fe
Valencia, Spain, 46026
Actively Recruiting
25
Leeds General Infirmary Children's Research Department
Leeds, West Yorkshire, United Kingdom, LS1 3EX
Actively Recruiting
Research Team
S
Scholar Rock
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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