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Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)

NCT05532813 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 142

Last updated 2025-11-24

No results posted yet for this study

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.

Conditions

  • Steinert's Disease
  • Myotonic Dystrophy 1
  • Metformin

Interventions

DRUG

Treatment taken

Treatment (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.

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Pascal LAFORÊT, MD, PhD · Neurology Department, Raymond Poincaré Hospital, APHP

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-29
Primary Completion
2026-11-30
Completion
2026-12-31

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05532813 on ClinicalTrials.gov