NCT05532813 Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
| NCT ID | NCT05532813 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Condition | Steinert's Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 142 participants |
| Start Date | 2024-11-29 |
| Primary Completion | 2026-11 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 142 participants in total. It began in 2024-11-29 with a primary completion date of 2026-11.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
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
Contact & Investigator
Pascal LAFORÊT, MD, PhD
PRINCIPAL INVESTIGATOR
Neurology Department, Raymond Poincaré Hospital, APHP
Frequently Asked Questions
Who can join the NCT05532813 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 70 Years, studying Steinert's Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05532813 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 142 participants.
Is NCT05532813 currently recruiting?
Yes, NCT05532813 is actively recruiting participants. Contact the research team at pascal.laforet@aphp.fr for enrollment information.
Where is the NCT05532813 trial being conducted?
This trial is being conducted at Garches, France.
Who is sponsoring the NCT05532813 clinical trial?
NCT05532813 is sponsored by Assistance Publique - Hôpitaux de Paris. The principal investigator is Pascal LAFORÊT, MD, PhD at Neurology Department, Raymond Poincaré Hospital, APHP. The trial plans to enroll 142 participants.