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

Qualifiers

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

Treatments tested in this trial

  • Treatment taken

Treatment groups

142 Participants
are divided into 2 treatment groups