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Could a popular Weight-Loss drug help—or Harm—Those with duchenne muscular dystrophy?

NCT ID NCT07642635

Disease control Sponsor: Vanderbilt University Medical Center 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 This study
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 Results may not be published yet.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)
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This status has not been confirmed recently, so it may be out of date.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 study tests whether semaglutide (a GLP-1 drug used for weight loss) is safe for adolescents and young adults with Duchenne muscular dystrophy (DMD) who are also overweight or obese. The main concern is that the drug might reduce muscle mass, which is already weak in DMD. Participants will take either semaglutide or a placebo for 24 weeks, then all will receive semaglutide for another 40 weeks. The study monitors muscle mass, fat, heart and lung function, and quality of life.

What this could mean

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

Active substance
semaglutide (Rybelsus)
What this could lead to
If safe, this could offer a weight-loss option for people with Duchenne muscular dystrophy who struggle with obesity, potentially improving heart and metabolic health.
What could go wrong
This is an early-phase trial with only 30 participants. The main concern is that semaglutide might reduce muscle mass, which is already compromised in DMD. Results may not apply to all patients.

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.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Dec 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

18 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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: * Male * Age ≥18years * BMI ≥ 30kg/m2 or BMI ≥ 27kg/m2 with at least one weight-related comorbid condition (e.g., hypertension, T2D, or dyslipidemia). * Clinical phenotype of DMD confirmed with muscle biopsy or genotype Exclusion Criteria: * Type 1 diabetes, uncontrolled type 2 diabetes (HbA1c \>8%) or type 2 diabetes requiring the use of insulin or sulfonylurea. * History of pancreatitis * Personal or family history of medullary thyroid carcinoma or multiple endocrine neoplasia type 2 * History of allergic reaction to semaglutide or medication components * Contraindication to MRI. If unable to tolerate whole body/cardiac MRI but able to undergo lower extremity MRI, the participant may be invited to complete extremity MRI (Aim 1) and not complete MRI for Aim 2/3 (secondary Aims/outcomes) * Uncontrolled major depressive disorder, lifetime history of suicide attempt, history of other severe psychiatric disorders (e.g., schizophrenia, bipolar disorder), PHQ-9 score ≥15 or suicidal ideation type 4 or 5 (C-SSRS) * Unable to comply with study procedures or unsafe to complete the study in the opinion of the investigator

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy Obesity

As listed by the trial registrant

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

Duchenne muscular dystrophy (DMD)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

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