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Study on the Effects of Metformin for Improving Muscle Function in Patients with Myotonic Dystrophy Type 1

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called metformin on a condition known as Myotonic Dystrophy Type 1, also referred to as Steinert's Disease. This is a genetic disorder that affects muscle function, leading to muscle weakness and other symptoms. The study will compare the effects of metformin with a placebo to see if metformin can improve muscle function in people with this condition.

The purpose of the study is to evaluate how well metformin works in improving muscle function in patients with Myotonic Dystrophy Type 1. Participants in the study will be randomly assigned to receive either metformin or a placebo. The study will last for about 12 months, during which participants will have regular check-ups to monitor their muscle function and overall health. The study will also assess the safety of metformin by keeping track of any serious side effects that may occur during the treatment period.

Throughout the study, various aspects of muscle function will be measured, including walking ability, hand-grip strength, and other physical capabilities. The study will also look at how metformin affects heart and lung function, as well as the overall quality of life for participants. By the end of the study, researchers hope to determine whether metformin can be a beneficial treatment for improving muscle function in people with Myotonic Dystrophy Type 1.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed through genetic analysis for myotonic dystrophy type 1 (DM1).

    Participants must be between 18 and 70 years old, with preserved walking abilities, and meet specific health criteria.

  2. Step 2

    Baseline visit

    The initial assessment includes a Motor Function Measure (MFM-32) scale evaluation to establish a baseline for muscle function.

    Additional tests may include assessments of walking, hand-grip strength, pinch strength, cardiac function, respiratory function, and quality of life.

  3. Step 3

    Treatment phase

    Participants receive either Metformin Viatris 500 mg or a placebo, administered orally as a dispersible tablet.

    The medication is taken regularly over a period of 12 months.

  4. Step 4

    Interim assessment

    At 6 months, an interim evaluation is conducted to assess changes in muscle function using the MFM-32 scale.

    Additional assessments may include walking tests, hand-grip strength, pinch strength, and other health indicators.

  5. Step 5

    Final assessment

    At 12 months, a final evaluation is conducted to measure the evolution of muscle function and other health indicators.

    The primary endpoint is the change in the MFM-32 scale score from the baseline to the final visit.

  6. Step 6

    Safety monitoring

    Throughout the study, safety is monitored by collecting data on any serious adverse events.

    The safety of metformin is evaluated continuously during the 12-month treatment period.

Who can join the trial?

8 criteria

  • Diagnosis of Myotonic dystrophy type 1 (DM1) confirmed by a genetic test.
  • Men and women who are between 18 and 70 years old.
  • Ability to walk, even if a walking stick is needed for assistance.
  • A Muscular Impairment Rating Scale (MIRS) score of 3 or 4, which is a way to measure muscle weakness.
  • Women who can have children must use effective birth control during the study and for 2 days after the treatment ends.
  • Ability to understand and sign a consent form, which means agreeing to participate in the study.
  • Understanding of the French language, or having a family member or medical team member who can help explain the study.
  • Being part of a social security system.

Who cannot join the trial?

4 criteria

  • Patients who are not diagnosed with Myotonic dystrophy type 1 (DM1) cannot participate. This is a genetic disorder that affects muscle function.
  • Individuals who are not within the specified age range for the study are excluded. The study is open to certain age groups only.
  • Participants who are part of a vulnerable population, meaning those who might be at higher risk of harm or exploitation, are not eligible.
  • Both males and females can participate, but anyone not fitting the gender criteria specified for the study will be excluded.
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Investigated drugs

Metformin is a medication commonly used to treat type 2 diabetes. In this clinical trial, it is being tested to see if it can help improve muscle function in patients with Myotonic Dystrophy Type I, also known as Steinert’s Disease. The study aims to determine if metformin can be effective in managing symptoms related to this condition.

What is already known about the treatment

Metformin – Metformin is typically administered orally in tablet form. It is currently being studied in a Phase III clinical trial to assess its effectiveness and safety for treating myotonic dystrophy type 1 (DM1), also known as Steinert's disease. The main therapeutic indication being explored is the improvement of muscle function in patients with DM1. Metformin works by decreasing glucose production in the liver and improving insulin sensitivity, which may have beneficial effects on muscle function. It is classified as a biguanide, a type of medication commonly used to manage blood sugar levels in type 2 diabetes.

Investigated diseases

Myotonic Dystrophy Type 1 (DM1) – This is a genetic disorder characterized by progressive muscle wasting and weakness. It often begins in adulthood and can affect various body systems, including the heart, eyes, and endocrine system. Individuals with DM1 may experience prolonged muscle contractions and have difficulty relaxing certain muscles after use. Over time, muscle weakness can lead to mobility issues and challenges with daily activities. The severity and progression of symptoms can vary widely among individuals. It is considered a rare disease and is caused by a specific genetic mutation.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507660-39-00Protocol codeAPHP220832Estimated enrolment203 patientsSponsorAssistance Publique Hopitaux De Paris

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