METFORMIN HYDROCHLORIDE for Myotonic dystrophy type 1 | Steinert disease

Inclusion criteria

• {"criterion_text":"- DM1 disease confirmed by genetic analysis"}
• {"criterion_text":"- Men and women between 18 and 70 years of age"}
• {"criterion_text":"- Preserved walking abilities (stick assistance possible)"}
• {"criterion_text":"- MIRS (Muscular Impairment Rating Scale) 3 or 4"}
• {"criterion_text":"- Women of childbearing potential under efficient contraception during treatment and up to 2 days after the end of the experimental treatment"}
• {"criterion_text":"- Ability to provide signed informed consent"}
• {"criterion_text":"- Capacity of the patient to understand French language or presence of a relative (or the medical team) who can help the patient understand the study."}
• {"criterion_text":"- Affiliation to a social security system"}

Exclusion criteria

• {"criterion_text":"- Diabetic patient"}
• {"criterion_text":"- Patient already taking metformin"}
• {"criterion_text":"- Pregnant or breast-feeding women"}
• {"criterion_text":"- Men with an intention to conceive a child during the time of the study"}
• {"criterion_text":"- Contraindications to Metformin: o All types of acute metabolic acidosis o Acute conditions that may impair renal function, such as: dehydration, severe infection, shock o Hepatocellular insufficiency, acute alcohol intoxication, alcoholism o Hypersensitivity to metformin or to one of the excipients o Acute disease that may lead to tissue hypoxia such as decompensated heart failure, acute respiratory failure, recent myocardial infarction. - Respiratory contraindications: o Patient requiring tracheotomy or o Patient requiring non-invasive-ventilation: - more than 12 hours per day - insufficiently ventilated"}
• {"criterion_text":"- Creatinine clearance inferior to 60 ml/min (MDRD)."}
• {"criterion_text":"- Cardiac contraindications: o Left ventricular ejection fraction below 35% o 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 has been performed o Third-degree or Second degree type II atrioventricular block without a pacemaker or an implantable defibrillator o Sustained ventricular tachycardia o Acute cardiac failure o Myocardial infarction of less than three months"}
• {"criterion_text":"- Patient participating in another interventional clinical trial"}
• {"criterion_text":"- Patient under legal protection (curatorship, tutorship)"}

Primary endpoints

• {"endpoint_text":"- The primary endpoint is the evolution of the MFM-32 (Motor Function Measure) scale scoring between the baseline visit and the final visit at 12 months.","definition_or_measurement_approach":"Evolution of the MFM-32 (Motor Function Measure) score between baseline and the final visit at 12 months."}

Secondary endpoints

• {"endpoint_text":"- To evaluate the safety of metformin in DM1 patient using the collection of serious adverse event during the 12 months of treatment.","definition_or_measurement_approach":"Collection and evaluation of serious adverse events occurring during the 12 months of treatment."}
• {"endpoint_text":"- To evaluate the efficacy on metformin on muscle function at 6 months using the evolution of MFM-32 scale scoring between the baseline visit and the visit at 6 months as endpoint.","definition_or_measurement_approach":"Evolution of MFM-32 score between baseline and 6-month visit."}
• {"endpoint_text":"- To evaluate the efficacy of metformin on locomotor function using the evolution of six minutes walking test scoring between the baseline visit, the visit at 6 months and the final visit at 12 months as endpoints.","definition_or_measurement_approach":"Change in six-minute walk test score between baseline, 6 months and 12 months."}
• {"endpoint_text":"- To evaluate the efficacy of metformin on hand-grip strength using the evolution of MyoGrip scoring between the baseline visit, the visit at 6 months and the final visit at 12 months as endpoints.","definition_or_measurement_approach":"Evolution of MyoGrip score between baseline, 6 months and 12 months."}
• {"endpoint_text":"- To evaluate the efficacy of metformin on thumb-index pinch strength using the evolution of MyoPinch scoring between the baseline visit, the visit at 6 months and the final visit at 12 months as endpoints.","definition_or_measurement_approach":"Evolution of MyoPinch score between baseline, 6 months and 12 months."}
• {"endpoint_text":"- To evaluate the efficacy of metformin on respiratory function using the evolution of supine vital capacity between the baseline visit, the visit at 6 months and the final visit at 12 months as endpoints.","definition_or_measurement_approach":"Change in supine vital capacity between baseline, 6 months and 12 months."}
• {"endpoint_text":"- To evaluate the efficacy of metformin on quality of life using the evolution of QoLgNMD scoring between the baseline visit, the visit at 6 months and the final visit at 12 months as endpoints","definition_or_measurement_approach":"Evolution of QoLgNMD questionnaire score between baseline, 6 months and 12 months."}
• {"endpoint_text":"- To evaluate the efficacy of metformin on cardiac function using the evolution of left ventricular ejection fraction between the baseline visit and the final visit at 12 months as endpoints.","definition_or_measurement_approach":"Change in left ventricular ejection fraction between baseline and 12 months."}

France

Earliest CTIS Part Ii Submission Date

24-05-2024

Latest Decision Or Authorization Date

07-08-2025

Processing Time Days

440

Number Of Sites

15

Number Of Participants

203

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France