A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Start Date
9/15/2025
Completion Date
3/1/2029
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.
Eligibility Criteria
Age Range: No minimum to 2 years
Interventions
Apitegromab
Nusinersen
Risdiplam
Conditions
Locations
Phoenix Children's Hospital
Phoenix, Arizona 85016
United States
Children's Hospital of Orange County (CHOC)
Orange, California 92868
United States
Stanford Neuroscience Health Center (SNHC)
Palo Alto, California 94304
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Children's Healthcare of Atlanta
Atlanta, Georgia 30329
United States
University of Iowa
Iowa City, Iowa 52242
United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan 49503
United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina 27157
United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Le Bonheur Children's Hospital
Memphis, Tennessee 38103
United States
University of Texas Southwestern Medical Center
Dallas, Texas 75207
United States
Neurology Rare Disease Center
Flower Mound, Texas 75028
United States
Cook Children's Medical Center
Fort Worth, Texas 76104
United States
Texas Children's Hospital (TCH)-Clinical Care Center (CCC)
Houston, Texas 77030
United States
UZ Gent
Ghent, East-Flanders 9000
Belgium
UZ Leuven - Campus Gasthuisberg
Leuven, 3000
Belgium
CHR Citadelle
Liège, 4000
Belgium
Hopital Trousseau - I-Motion
Paris, 75012
France
Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta
Milan, 20133
Italy
Centro Clinico NeMO Milano - Fondazione Serena Onlus
Milan, 20162
Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, 00168
Italy
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
Netherlands
Hospital Sant Joan de Déu Barcelona
Barcelona, 08950
Spain
Hospital Universitari i Politecnico La Fecnic La Fe
Valencia, 46026
Spain
Leeds General Infirmary Children's Research Department
Leeds, West Yorkshire LS1 3EX
United Kingdom