Actively Recruiting
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2025-11-24
142
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 must use effective contraception during treatment
- Patient must be able to give informed consent
- Signed informed consent form
- Affiliation to a social security system
You will not qualify if you...
- Pregnant or breastfeeding women
- Men planning to conceive a child during the study
- Known hypersensitivity to metformin or its components
- Patients requiring tracheotomy
- Patients requiring non-invasive ventilation more than 12 hours per day or insufficiently ventilated
- Creatinine clearance less than 50 ml/min
- Left ventricular ejection fraction below 35%
- Conduction system disease with PR interval >200 ms or QRS duration >110 ms without pacemaker or implantable defibrillator
- Second or third-degree atrioventricular block without pacemaker or implantable defibrillator
- Sustained ventricular tachycardia
- Acute diseases that may cause tissue hypoxia
AI-Screening
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Trial Site Locations
Total: 1 location
1
Neurology Department, Raymond-Poincaré hospital - APHP
Garches, France, 92380
Actively Recruiting
Research Team
P
Pascal LAFORÊT, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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