Metformin Clinical Trial
— METFORMYOOfficial title:
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
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.
Status | Not yet recruiting |
Enrollment | 142 |
Est. completion date | October 2026 |
Est. primary completion date | October 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 70 Years |
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 |
Country | Name | City | State |
---|---|---|---|
France | Neurology Department, Raymond-Poincaré hospital - APHP | Garches |
Lead Sponsor | Collaborator |
---|---|
Assistance Publique - Hôpitaux de Paris |
France,
Berard C, Payan C, Hodgkinson I, Fermanian J; MFM Collaborative Study Group. A motor function measure for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005 Jul;15(7):463-70. doi: 10.1016/j.nmd.2005.03.004. — View Citation
Landfeldt E, Nikolenko N, Jimenez-Moreno C, Cumming S, Monckton DG, Faber CG, Merkies ISJ, Gorman G, Turner C, Lochmuller H. Change over time in ability to perform activities of daily living in myotonic dystrophy type 1. J Neurol. 2020 Nov;267(11):3235-3242. doi: 10.1007/s00415-020-09970-6. Epub 2020 Jun 15. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change of muscle function | By MFM (Motor Function Measure) scale. The MFM-32 is a widely used sensitive and reliable quantitative functional motor scale, validated for use in various neuromuscular disorders (BĂ©rard et al. 2005) and presenting the advantage to measure precisely, not only the muscle strength but motor function which is the main concern for DM1 patients. | at baseline and 12 months | |
Secondary | Safety endpoint | Any serious adverse event, especially lactic acidosis. | through study completion, an average of 30 month | |
Secondary | Change of muscle function between baseline and 6 months | By the MFM (Motor Function Measure) scale. | at baseline and 6 months | |
Secondary | The hand-grip strength | The hand-grip strength defined by the scores obtained using a manual dynamometry standardized (MyoGrip) | baseline, 6 and 12 months | |
Secondary | The thumb-index pinch strength | The thumb-index pinch strength defined by the scores obtained using a standardized manual dynamometry (MyoPinch) | baseline, 6 and 12 months | |
Secondary | The locomotor function | The locomotor function defined by the scores obtained using the 6 Minutes Walking Test. | baseline, 6 and 12 months | |
Secondary | The respiratory function | The respiratory function, using pulmonary function tests, defined by the Supine Vital Capacity (VC). | baseline, 6 and 12 months | |
Secondary | The cardiac function | The cardiac function, using transthoracic echocardiography, defined by the left ventricular ejection fraction. | baseline, 6 and 12 months | |
Secondary | Quality of life assessement | The quality of life defined by the scores obtained using the quality of life in genetic neuromuscular disease questionnaire (QoLgNMD). | baseline, 6 and 12 months | |
Secondary | The difference between DM1-ActivC at baseline visit, the visit at 6 months and final visit | The activity defined by the scores obtained using the DM1 activity and participation scale for clinical use (DM1-ActivC). | baseline, 6 and 12 months |
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