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)
Status: Recruiting
Location: See all (14) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Maximum Age: 2
Healthy Volunteers: f
View:
• Is \<2 years old at the time of the informed consent
• Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
• Has confirmed diagnosis of 5q autosomal recessive SMA
• Has confirmed presence of SMN2 gene copy(ies)
• Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)
• Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit
• Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \<55
Locations
United States
Arizona
Phoenix Children's Hospital
RECRUITING
Phoenix
California
Stanford Neuroscience Health Center (SNHC)
RECRUITING
Palo Alto
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Iowa
University of Iowa
RECRUITING
Iowa City
North Carolina
Atrium Health Wake Forest Baptist
RECRUITING
Winston-salem
Texas
University of Texas Southwestern Medical Center
RECRUITING
Dallas
Neurology Rare Disease Center
RECRUITING
Flower Mound
Cook Children's Medical Center
RECRUITING
Fort Worth
Other Locations
Belgium
UZ Leuven - Campus Gasthuisberg
RECRUITING
Leuven
CHR Citadelle
RECRUITING
Liège
Italy
Centro Clinico NeMO Milano - Fondazione Serena Onlus
RECRUITING
Milan
Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta
RECRUITING
Milan
Spain
Hospital Sant Joan de Déu Barcelona
RECRUITING
Barcelona
Hospital Universitari i Politecnico La Fecnic La Fe
RECRUITING
Valencia
Contact Information
Primary
Scholar Rock
MedicalInformation@scholarrock.com
857-259-3860
Time Frame
Start Date:2025-09-15
Estimated Completion Date:2029-03
Participants
Target number of participants:52
Treatments
Experimental: Apitegromab low dose + SMN Therapy
Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period.~Patients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam).
Experimental: Apitegromab high dose + SMN Therapy
Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period.~Patients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam).