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

What Was Tested

This registered phase 1, phase 2 trial tested Semaglutide.

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.

It had three arms (groups): Semaglutide (RCT) (experimental), Placebo (RCT) (placebo comparator) and Semaglutide (OLE) (experimental).

Oral semaglutide (24 week RCT).

Oral placebo (24 week RCT).

Oral semaglutide (40 week open label extension).

The registry classes the interventions as drug and other.

Who Took Part

30 planned participants were the target.

It was open to men only aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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.

The full record lists 4 inclusion and 7 exclusion criteria; among the exclusions: 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.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the Semaglutide group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Blinding on both sides kept the participants and researchers unaware of who received Semaglutide.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2026-09-01 with completion listed for 2030-09.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Muscle Volume Index (MVI) (time frame: Week 0 (baseline), week 24 (end of RCT), week 64 (end of study)).

It also tracked 6 secondary outcomes, for example Visceral adiposity, Weight, Insulin sensitivity, LVEF, Late gadolinium enhancement (LGE) and FVC%.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, randomized trial of Semaglutide in 30 people.

Its size is small, so on its own it can only give an early look.

Its status is not yet recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Semaglutide compound page, all human trials.