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

NCT07047144 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 52

Last updated 2026-05-01

No results posted yet for this study

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

  • Spinal Muscular Atrophy
  • SMA
  • Spinal Muscular Atrophy Type 2
  • Spinal Muscular Atrophy Type 3
  • Neuromuscular Manifestations
  • Anti-myostatin

Interventions

DRUG

Apitegromab

Apitegromab 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.

DRUG

Nusinersen

Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.

DRUG

Risdiplam

Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.

Sponsors & Collaborators

  • Scholar Rock, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-09-15
Primary Completion
2028-11-30
Completion
2029-03-31
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • France
  • Italy
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07047144 on ClinicalTrials.gov