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Duchenne Muscular Dystrophy (DMD)Phase 1/2NCT07642635

Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy

A Phase 1/2 study testing Semaglutide (Rybelsus®) in people with Duchenne Muscular Dystrophy (DMD), run by Vanderbilt University Medical Center at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

Duchenne Muscular Dystrophy (DMD) is a rare, genetic disease that leads to muscle weakness, breathing difficulties, heart disease, and early death. Approximately half of individuals with DMD have elevated body mass indices (BMIs) in the overweight or obesity range. Read more about this study

Who is it for?
Ages 18 years and older, men only
Conditions the study is for
Duchenne Muscular Dystrophy (DMD)
What is being tested?
Semaglutide (Rybelsus®)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
30 participants
Where
1 siteUnited States
Study dates
1 Sep 2026to Dec 2030
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • Men only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Jaclyn Tamaroff, MD contact

615-875-7853

Jaclyn.tamaroff@vumc.org

Jonathan Soslow, MD contact

United States1 site

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

Other trials in United States

About this study

Duchenne Muscular Dystrophy (DMD) is a rare, genetic disease that leads to muscle weakness, breathing difficulties, heart disease, and early death. Approximately half of individuals with DMD have elevated body mass indices (BMIs) in the overweight or obesity range. High BMI is due to a combination of factors including limited mobility and steroid medications, which are used to treat DMD.

There are new medications, glucagon-like peptide-1 receptor agonists (GLP-1 RAs) that promote weight loss in the general population. GLP-1 RAs are approved for weight loss in children and adults and have beneficial effects on the heart. There is a concern that these medications could have unwanted side effects in individuals with DMD, specifically decreasing their muscle mass. While it is important to consider the use weight-loss medications in DMD, the investigators want to ensure that they are safe and well-tolerated. Therefore, this study will systematically evaluate whether the use of GLP-1 RAs in adolescents and young adults with DMD affects muscle mass.

Read more

The overall goal of this study is to assess the safety and tolerability of GLP1-RAs in individuals with both DMD and obesity. The primary focus will be on muscle health, but the study will also evaluate activity levels, mood, gastrointestinal symptoms, and quality of life. Secondary goals will be to understand the impact of GLP1-RAs on weight, fat mass, glucose and insulin levels, and heart and lung function in individuals with DMD. The investigators hypothesize that GLP1-RAs will be well-tolerated and will decrease fat mass, without a large decrease in muscle mass.

Participants will:

  • Take oral semaglutide or a placebo every day for 24 weeks (randomized controlled trial)
  • Then take oral semaglutide every day for 40 weeks (open label extension)
  • Complete in-person study visits at 3 timepoints
  • Study visits may include: an MRI of the body to evaluate muscle and fat tissue, laboratory testing, a mixed meal tolerance test, questionnaires, an MRI of the heart, pulmonary function tests, and additional measures
  • Calls with the study team between visits (monthly or every other month)
Being tested
Semaglutide (Rybelsus®), Placebo
Keywords
Duchenne muscular dystrophy, Obesity, Glucagon-Like Peptide-1 Receptor Agonists
Medical subject headings
Muscular Dystrophy, Duchenne, Obesity

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Semaglutide (Rybelsus®)Drug

    Also called Wegovy

    Oral semaglutide will be provided as part of the RCT and subsequent open label extension. This will be taken as a daily medication following approved dose titration schedule.

  • PlaceboOther

    oral placebo

Study groups in NCT07642635
Study groupRoleWhat they receive
Semaglutide (RCT)Experimentaloral semaglutide (24 week RCT)
Receives: Drug: Semaglutide (Rybelsus®)
Placebo (RCT)Placebo comparatororal placebo (24 week RCT)
Receives: Other: Placebo
Semaglutide (OLE)Experimentaloral semaglutide (40 week open label extension)
Receives: Drug: Semaglutide (Rybelsus®)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Muscle Volume Index (MVI)

    Assessment of muscle mass via skeletal muscle MRI. Change in MVI from baseline to week 24 will be compared in those on placebo compared to those on semaglutide.

    Measured: Week 0 (baseline), week 24 (end of RCT), week 64 (end of study)

Secondary outcomes (6)

  1. Visceral adiposity

    Visceral adiposity will be measured on whole-body MRI. Change in visceral fat area from baseline to week 24 will be compared in those on placebo compared to those on semaglutide..

    Measured: Week 0 (baseline), week 24 (end of RCT), week 64 (end of study)

  2. Weight

    Weight will be measured in kg. Change in weight from baseline to week 24 will be compared in those on placebo compared to those on semaglutide.

    Measured: Week 0 (baseline), week 24 (end of RCT), week 64 (end of study)

  3. Insulin sensitivity

    Insulin sensitivity will be calculated from the mixed meal tolerance test. Change in insulin sensitivity from baseline to week 24 will be compared in those on placebo compared to those on semaglutide.

    Measured: Week 0 (baseline), week 24 (end of RCT), week 64 (end of study)

  4. LVEF

    LVEF will be assessed via cardiac MRI. Change in LVEF over the course of the study will be calculated.

    Measured: Week 0 (baseline) and week 64 (end of study)

  5. Late gadolinium enhancement (LGE)

    LGE, a measurement of myocardial fibrosis, will be assessed on cardiac MRI. Change in LGE over the course of the study will be calculated.

    Measured: Week 0 (baseline) and week 64 (end of study).

  6. FVC%

    Percent predicted forced vital capacity will be evaluated with pulmonary function tests. Change in FVC% over the course of the study will be calculated.

    Measured: Week 0 (baseline) and week 64 (end of study).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Jun 2026
  2. Study start1 Sep 2026 (estimated)
  3. Primary completionSep 2030 (estimated)
  4. Study completionDec 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Randomized controlled trial (24 weeks) and subsequent open label extension (40 weeks).
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07642635
Sponsor's protocol ID
260611
Status last verified
Jun 2026
First submitted
4 Jun 2026
Last updated
11 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: IPD may be available with appropriate regulatory approvals.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Semaglutide (Rybelsus®) in people with Duchenne Muscular Dystrophy (DMD), run by Vanderbilt University Medical Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Jaclyn Tamaroff, MD (615-875-7853, Jaclyn.tamaroff@vumc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Muscle Volume Index (MVI) (time frame: Week 0 (baseline), week 24 (end of RCT), week 64 (end of study)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07642635.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026; AzTrials last refreshed its copy on 7 Nov 2026.

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