FindTrial
Sign In

EAP of Apitegromab for Patients With Spinal Muscular Atrophy

Expanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy

Status
NO_LONGER_AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT06877689
Enrollment
Unknown
Registered
2025-03-14
Start date
Unknown
Completion date
Unknown
Last updated
2026-04-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

SMA

Keywords

Expanded Access, Pre-Approval Access, Compassionate Use

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.

Interventions

DRUGApitegromab

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.

Sponsors

Scholar Rock, Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
2 Years to No maximum

Inclusion criteria

1. ≥2 years of age. 2. Documented diagnosis of 5q SMA.

Exclusion criteria

1. 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. 2. Enrolled in a clinical study for any investigational drug. 3. Pregnant or breastfeeding.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 29, 2026