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RecruitingNCT07047144

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

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
Phase
Phase 2
Study type
Interventional
Enrollment
52 (estimated)
Sponsor
Scholar Rock, Inc. · Industry
Sex
All
Age
2 Years
Healthy volunteers
Not accepted

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.

Conditions

Interventions

TypeNameDescription
DRUGApitegromabApitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 will be administered every 4 weeks by intravenous (IV) infusion.
DRUGNusinersenNusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.
DRUGRisdiplamRisdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.

Timeline

Start date
2025-09-15
Primary completion
2028-11-01
Completion
2029-03-01
First posted
2025-07-02
Last updated
2026-03-25

Locations

23 sites across 6 countries: United States, Belgium, France, Italy, Netherlands, Spain

Regulatory

Source: ClinicalTrials.gov record NCT07047144. Inclusion in this directory is not an endorsement.