EAP of Apitegromab for Patients With Spinal Muscular Atrophy

NCT06877689 | Unknown

• 1 other identifier: SRK-015-008
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency . A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.

Conditions

SMA

Keywords

Expanded Access; Pre-Approval Access; Compassionate Use

Interventions

Apitegromab DRUG

Apitegromab (SRK-015) is a fully human anti-proMyostatin monoclonal antibody (mAb) that specifically binds to human pro/latent myostatin, inhibiting myostatin activation. Apitegromab will be administered every 4 weeks by intravenous (IV) infusion.

Also known as: SRK-015

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥2 years of age.
• Documented diagnosis of 5q SMA.

You may not qualify if:

• Previous history of a hypersensitivity reaction to a monoclonal antibody or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab.
• Enrolled in a clinical study for any investigational drug.
• Pregnant or breastfeeding.

Sponsors & Collaborators

• Scholar Rock, Inc.: lead

MeSH Terms

Interventions

apitegromab

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2025
• First Posted: March 14, 2025
• Last Updated: April 28, 2026

Record last verified: 2026-04