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.
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
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.
Jonathan Soslow, MD contact
United States1 site
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
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)
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- 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 group | Role | What they receive |
|---|---|---|
| Semaglutide (RCT) | Experimental | oral semaglutide (24 week RCT) Receives: Drug: Semaglutide (Rybelsus®) |
| Placebo (RCT) | Placebo comparator | oral placebo (24 week RCT) Receives: Other: Placebo |
| Semaglutide (OLE) | Experimental | oral 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.
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)
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)
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)
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)
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)
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).
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.
- First posted11 Jun 2026
- Study start1 Sep 2026 (estimated)
- Primary completionSep 2030 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jaclyn Tamaroff, Vanderbilt University Medical Center
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.