Recruiting
Apitegromab Access for Spinal Muscular Atrophy
What is being tested
Data Collection
Who is eligible
Over 2 Years
+5 Eligibility Criteria
How is it accessible
Recruiting
Available upon a request by a licensed MDExpanded Access
Summary
Principal SponsorScholar Rock, Inc.
Study ContactScholar Rock, Inc. Expanded Access
Last updated: January 28, 2026Sourced from a government-validated database.Claim as a partner
This study is an Expanded Access Program (EAP) designed to provide apitegromab, an investigational therapy, to eligible patients diagnosed with Spinal Muscular Atrophy (SMA). SMA is a condition that affects the nervous system and causes muscle weakness. The goal of this program is to make apitegromab available to patients before it receives approval from local regulatory agencies. The decision to participate is made by a medical doctor, who evaluates the potential benefits and risks of the therapy based on the patient's medical history and the eligibility criteria of the program.
Official TitleExpanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy
Principal SponsorScholar Rock, Inc.
Study ContactScholar Rock, Inc. Expanded Access
Last updated: January 28, 2026Sourced from a government-validated database.Claim as a partner
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Criteria
Any sex
Biological sex of participants that are eligible to enroll.Over 2 Years
Range of ages for which participants are eligible to join.Criteria
2 inclusion criteria required to participate
≥2 years of age.
Documented diagnosis of 5q SMA.
3 exclusion criteria prevent from participating
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.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has no location dataSave this study to your profile to know when the location data is available.
Recruiting
No study centers