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 (2) 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
North Carolina
Atrium Health Wake Forest Baptist
RECRUITING
Winston-salem
Texas
Neurology Rare Disease Center
RECRUITING
Denton
Contact Information
Primary
Scholar Rock
clinicaltrials@scholarrock.com
857-259-3860
Time Frame
Start Date:2025-09
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).