Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study
Trial ID: NCT05532813
Interventional - Phase 3 - Recruiting
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
- Steinert's Disease
- Myotonic Dystrophy 1
- Metformin
Interventions
Eligibility
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
Sponsor
Assistance Publique - Hôpitaux de Paris
Source: ClinicalTrials.gov
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