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Not yet recruitingPhase 1 / Phase 2

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
Male only
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether semaglutide, a weight-loss medicine, is safe for adult males with Duchenne muscular dystrophy (DMD) and a high body weight. DMD is a genetic disease that weakens muscles, so the study will focus on whether the medicine causes muscle loss. Participants take semaglutide or a placebo (a pill with no study medicine) for 24 weeks, then semaglutide for 40 weeks.

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)

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Male and at least 18 years old.
  • Have a body mass index (BMI, a measure of weight compared with height) of 30 or higher, or 27 or higher with a weight-related health problem, such as high blood pressure, type 2 diabetes, or unhealthy blood fat levels.
  • Have signs of DMD, confirmed by a muscle tissue test or genetic testing.

Who can't join

  • Have type 1 diabetes, type 2 diabetes with an HbA1c above 8% (a measure of average blood sugar), or type 2 diabetes that needs insulin or a sulfonylurea medicine.
  • Have had pancreatitis, which is swelling of the pancreas.
  • You or a family member has had medullary thyroid cancer or multiple endocrine neoplasia type 2, a condition that causes tumors in hormone-making glands.
  • Have had an allergic reaction to semaglutide or its ingredients.
  • Cannot safely have an MRI scan. If you cannot handle a whole-body or heart MRI but can have a leg MRI, you may be able to join only the leg MRI part.
  • Have uncontrolled major depression, any past suicide attempt, or a history of another severe mental health condition, such as schizophrenia or bipolar disorder. Certain scores on depression or suicide-risk checks also rule out joining.
  • Cannot follow the study steps, or the study doctor thinks taking part would be unsafe.

Simplified for readability. The official criteria below are what the study team uses.

Official 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

Study sites (1)

TN