EAP of Apitegromab for Patients With Spinal Muscular Atrophy

N/ANo Longer AvailableNCT06877689

Scholar Rock, Inc.

Overview

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.

Study Type

EXPANDED_ACCESS

Conditions

SMA

Interventions

ApitegromabDRUG

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.

Eligibility

Sex: ALLMin age: 2 Years

Medical Language ↔ Plain English

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.

Data from ClinicalTrials.gov

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