Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Early access to experimental SMA drug apitegromab offered before approval

NCT ID NCT06877689

Disease control Sponsor: Scholar Rock, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended) This study
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed diagnosis of 5q SMA. The program aims to offer treatment to those who cannot join other clinical trials and for whom the potential benefit outweighs the risks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
apitegromab (a monoclonal antibody that blocks myostatin activation to help maintain muscle strength)
What this could lead to
If successful, this program could provide early access to a treatment that helps preserve muscle function in people with spinal muscular atrophy.
What could go wrong
This is an expanded access program, not a full clinical trial, so data on safety and effectiveness are still limited. Apitegromab is investigational and may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for 5q spinal muscular atrophy are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

proximal spinal muscular atrophy spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SMA

More trials for these conditions

Other studies related to the condition(s) this trial covers.