Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Steinert's Disease, Myotonic Dystrophy 1, Metformin

In brief

The study team hypothesize that non-diabetic patients with Myotonic dystrophy type I (DM1) will improve their symptoms, especially their motor deficit which is the main feature of the disease, because of the splicing defect correction by metformin. The primary objective of the study is to evaluate the efficacy of metformin vs placebo, on the improvement of muscle function in patients with DM1 compared to its placebo. As the secondary objectives, the study aims: * To evaluate the safety of metformin on patient with DM1. * To evaluate the efficacy of metformin vs placebo on: 1. The hand-grip strength; 2. The thumb-index pinch strength; 3. The locomotor function; 4. The respiratory function; 5. The cardiac function; 6. The quality of life; 7. The daily and social activity.

Key facts

Study ID: NCT05532813
Run by: Assistance Publique - Hôpitaux de Paris
People needed: 142
Starts: 2024-11-29
Expected to finish: 2026-12-01
Last updated by the study team: 2025-11-24

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

DM1 disease confirmed by genetic analysis
Men and women between 18 and 70 years of age.
Preserved walking abilities (stick assistance possible)
MIRS score 3 or 4
Women of childbearing potential under efficient contraception during treatment
Patient able to consent
All patients who have completed and signed the specific information and informed consent form
Affiliation to a social security system

You may not qualify if…

Pregnant or breast-feeding women
Men with an intention to conceive a child during the time of the study
Contraindications to Metformin (hypersensitivity to metformin or to one of the excipients)
Respiratory:
Patient requiring tracheotomy or
Patient requiring non-invasive-ventilation: - more than 12 hours per day; - insufficiently ventilated
Creatinine clearance inferior to 50 ml/min
Cardiac:
Left ventricular ejection fraction below 35%
Conduction system disease on the electrocardiogram with PR interval >200 ms or QRS duration >110 ms without a pacemaker or an implantable defibrillator or cardiac electrophysiological study performed over the past 5 years
Third-degree or Second degree type II atrioventricular block without a pacemaker or an implantable defibrillator
Sustained ventricular tachycardia
Acute disease that may lead to tissue hypoxia

Where it is running

Neurology Department, Raymond-Poincaré hospital - APHP — Garches, France (enrolling)

Full record on ClinicalTrials.gov

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