NCT07642635 Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy
| NCT ID | NCT07642635 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Vanderbilt University Medical Center |
| Condition | Duchenne Muscular Dystrophy (DMD) |
| Study Type | INTERVENTIONAL |
| Enrollment | 30 participants |
| Start Date | 2026-12 |
| Primary Completion | 2030-09 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 30 participants in total. It began in 2026-12 with a primary completion date of 2030-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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. 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. 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)
Eligibility Criteria
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07642635 clinical trial?
This trial is open to male participants only, aged 18 Years or older, studying Duchenne Muscular Dystrophy (DMD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07642635 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07642635 currently recruiting?
Yes, NCT07642635 is actively recruiting participants. Contact the research team at Jaclyn.tamaroff@vumc.org for enrollment information.
Where is the NCT07642635 trial being conducted?
This trial is being conducted at Nashville, United States.
Who is sponsoring the NCT07642635 clinical trial?
NCT07642635 is sponsored by Vanderbilt University Medical Center. The trial plans to enroll 30 participants.