Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-70
SponsorAssistance Publique - Hôpitaux de Paris
About this trial
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
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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)
Patient requiring tracheotomy or
Trial design
Design model
Parallel
Treatments tested in this trial
Treatment taken
DrugTreatment (Metformin or placebo) will be administered orally and titrated following the same guideline that metformin in diabetic patient: start with a daily dose of 500 mg twice a day, given during or after meals; then increase to 1000 mg twice a day after a week. If digestive tolerance is good, treatment will be increased to a maximum of 1000 mg three times a day i.e. 3000 mg/day after another week.
Treatment groups
142 Participants
are divided into 2 treatment groupsGroup A: Metformin armExperimental treatment 1 intervention
Group B: Placebo receiversPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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 (Bérard 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.
Time frame
at baseline and 12 monthsSecondary outcomes
1
Safety endpoint
Any serious adverse event, especially lactic acidosis.
Time frame
through study completion, an average of 30 month2
Change of muscle function between baseline and 6 months
By the MFM (Motor Function Measure) scale.
Time frame
at baseline and 6 months3
The hand-grip strength
The hand-grip strength defined by the scores obtained using a manual dynamometry standardized (MyoGrip)
Time frame
baseline, 6 and 12 months4
The thumb-index pinch strength
The thumb-index pinch strength defined by the scores obtained using a standardized manual dynamometry (MyoPinch)
Time frame
baseline, 6 and 12 monthsSponsors and contacts
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Source ClinicalTrials.gov