CClinicalTrials.gg
No longer availableNCT06877689Updated Apr 28, 2026

EAP of Apitegromab for Patients With Spinal Muscular Atrophy

An expanded access record providing Apitegromab in SMA, sponsored by Scholar Rock, Inc.. No longer available. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-04-28.

Sponsored by Scholar Rock, Inc. · Expanded access

Study type
Expanded access
Access type
Intermediate-size population
Ages
2 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • SMA

Keywords

  • Expanded Access
  • Pre-Approval Access
  • Compassionate Use
03

Who can participate

Ages eligible
2 Years and older
Sexes eligible
All

Inclusion criteria

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

Exclusion criteria

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.
04

Access details

Study type
Expanded access
Access type
Intermediate-size population

Available treatment

  • 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.

    Also known as: SRK-015

05

Where to request access

No study locations are listed for this record.

06

Registry details

Key details

Study ID
NCT06877689
Lead sponsor
Scholar Rock, Inc.
Responsible party
Sponsor
First posted
Mar 14, 2025
Last update
Apr 28, 2026
View the source record on ClinicalTrials.gov ↗

Requesting access

Expanded access is arranged between your doctor and the company. Ask your care team to contact the provider listed on this record.

No contact was published for this record. The registry link below has the sponsor’s details.

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Discussion

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