Metformin to improve muscle strength in myotonic dystrophy type 1

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, randomized, placebo-controlled Phase 3 trial in France compares oral metformin versus placebo in adults with genetically confirmed myotonic dystrophy type 1 and preserved walking ability. Participants are randomized 1:1 (about 71 per group) and receive metformin titrated up to a maximum of 1000 mg three times daily or matching placebo, with dose adjustments for gastrointestinal tolerance. Muscle function, hand-grip and pinch strength, walking tests, respiratory and cardiac function, quality of life, and safety are assessed at 6 and 12 months. The trial aims to determine whether metformin leads to measurable improvements in motor function and daily activity compared with placebo.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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's running

• Neurology Department, Raymond-Poincaré hospital - APHP
  Garches, France Recruiting

Study contacts

• Principal investigator: Pascal LAFORÊT, MD, PhD — Neurology Department, Raymond Poincaré Hospital, APHP
• Study coordinator: Pascal LAFORÊT, MD, PhD
• Email: pascal.laforet@aphp.fr
• Phone: +33 (0)1 47 10 77 36

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Online registry for myotonic dystrophy patients

  This study is creating an online registry for people with myotonic dystrophy to share their experiences and help researchers find better treatments.

  Recruiting Myotonic Dystrophy Foundation · Oakland, California · NCT02398786

• Understanding long-term progression in myotonic dystrophy type 1 (DM1)

  This study will follow adults with DM1 for at least four years to see how walking, hand function, muscle strength, heart rhythms, and lung function change over time.

  Recruiting Virginia Commonwealth University · Richmond, Virginia · NCT07700225

• Spanish National Registry for Myotonic Dystrophy Type 1

  This registry gathers health, clinical, and genetic information from people in Spain with DM1 to see how the condition changes over time and help match patients to future trials.

  Recruiting at 8 sites Fundació Institut Germans Trias i Pujol · Multiple Locations, Andalusia and 7 other locations · NCT07385443

• Long-term PGN-EDODM1 extension for people with myotonic dystrophy type 1

  This open-label extension will try continued PGN-EDODM1 in people with myotonic dystrophy type 1 who completed a prior PGN-EDODM1 study to see how safe and tolerable it is over time.

  Recruiting at 3 sites Phase 2 PepGen Inc · Calgary, Alberta and 2 other locations · NCT07220603

• Recognizing dapagliflozin overdose in ICU patients admitted for metformin poisoning.

  This project will see if clinical signs and routine lab tests can help doctors tell whether adults in the ICU for metformin poisoning also took too much dapagliflozin.

  Recruiting Centre Hospitalier de Saint-Denis · Saint-Denis, France · NCT07193069

• Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders

  This project uses a wrist/ankle wearable to continuously monitor walking and upper-limb movement in ambulant adults with neuromuscular diseases or obesity to see if digital measures like stride speed track real-life motor function.

  Recruiting Centre Hospitalier Universitaire de Liege · Liège · NCT07136844

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.