Steinert's DiseasePhase 3METFORMYONCT05532813
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
A Phase 3 study testing Treatment taken in people with Steinert's Disease and Myotonic Dystrophy 1, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Steinert's Disease, Myotonic Dystrophy 1, Metformin
- What is being tested?
- Treatment taken
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 142 participants
- Where
- 1 siteFrance
- Study dates
- 29 Nov 2024to Dec 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
- Women of childbearing potential under efficient contraception during treatment
- Patient able to consent
- All patients who have completed and signed the specific information and informed consent form
- Affiliation to a social security system
Who cannot join
- 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)
- Respiratory:
- Patient requiring tracheotomy or
- Patient requiring non-invasive-ventilation: - more than 12 hours per day; - insufficiently ventilated
Show all 13Show fewer
- Creatinine clearance inferior to 50 ml/min
- Cardiac:
- Left ventricular ejection fraction below 35%
- 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 performed over the past 5 years
- Third-degree or Second degree type II atrioventricular block without a pacemaker or an implantable defibrillator
- Sustained ventricular tachycardia
- Acute disease that may lead to tissue hypoxia
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Neurology Department, Raymond-Poincaré hospital - APHP
Garches, 92380
About this study
Official title: Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study
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.
Read more
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:
- The hand-grip strength;
- The thumb-index pinch strength;
- The locomotor function;
- The respiratory function;
- The cardiac function;
- The quality of life;
- The daily and social activity.
Read the detailed description
This is a multicenter, national, comparative study comparing the efficacy and safety of metformin and placebo in patients (1:1 ratio between the 2 groups) with DM1.
Population of study participants: patients with biochemically and/or genetically confirmed DM1 disease already followed in the referral and competence departments, as well as new patients.
All patients will be included by a neuromuscular specialist from French centers participating in the research.
Enrolled patients were randomly assigned (71 patients per group with 1:1 ratio) to either metformin therapy or a placebo, using a centralized randomization procedure.
Metformin or placebo will be administered orally and titrated as recommended in diabetic patients. Initial digestive effects (nausea, vomiting and constipation) of metformin that can be observed in the first days. 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. In case of bad digestive tolerance, the dosage should be decrease and the maximum tolerated dosage of metformin should be used. The evaluations of muscle function, walking test, respiratory and cardiac function, quality of life, and tolerance will be assessed at M6 and M12, in the neuromuscular centers. With the estimated effect size, we believe that the inclusion capacities evaluated at 8 to 12 patients per center over one year (18 reference centers involved) will allow to determine a significant difference of MFM score 12 months after inclusion. Dose titration, monitoring of side effects and dose adjustments will be assessed at each visit according to the site endocrinologist advice, if necessary.
Statistical analysis: The difference between the score at 12 months and baseline will be compared between treatment groups using the Student T-test.
Secondary efficacy endpoints evaluating the evolution of symptoms will be analyzed using either a GMM or a GEE for continuous and categorical variables, respectively.
Other quantitative variables will be compared using the Student t-test (or a non-parametric test if the distribution remains skewed following transformation), while categorical variables will be analyzed using either the Chi-squared or the Fisher-exact tests.
All efficacy endpoints will be analysed on an intention-to- treat basis and safety endpoints on a per-protocol basis.
All statistical tests will be performed with a level of significance of 5%. No interim analysis will be performed.
- Conditions
- Steinert's Disease, Myotonic Dystrophy 1, Metformin
- Being tested
- Treatment taken
- Keywords
- Steinert's Disease, Myotonic Dystrophy 1, Metformin, Muscle function
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Treatment takenDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Metformin arm | Experimental | Patients randomized in Metformin arm will take metformin orally. Receives: Drug: Treatment taken |
| Placebo receivers | Placebo comparator | Patients randomized in placebo arm will take placebo orally in the same procedure as metformin taken. Receives: Drug: Treatment taken |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: at baseline and 12 months
Secondary outcomes (9)
Safety endpoint
Any serious adverse event, especially lactic acidosis.
Measured: through study completion, an average of 30 month
Change of muscle function between baseline and 6 months
By the MFM (Motor Function Measure) scale.
Measured: at baseline and 6 months
The hand-grip strength
The hand-grip strength defined by the scores obtained using a manual dynamometry standardized (MyoGrip)
Measured: baseline, 6 and 12 months
The thumb-index pinch strength
The thumb-index pinch strength defined by the scores obtained using a standardized manual dynamometry (MyoPinch)
Measured: baseline, 6 and 12 months
The locomotor function
The locomotor function defined by the scores obtained using the 6 Minutes Walking Test.
Measured: baseline, 6 and 12 months
The respiratory function
The respiratory function, using pulmonary function tests, defined by the Supine Vital Capacity (VC).
Measured: baseline, 6 and 12 months
The cardiac function
The cardiac function, using transthoracic echocardiography, defined by the left ventricular ejection fraction.
Measured: baseline, 6 and 12 months
Quality of life assessement
The quality of life defined by the scores obtained using the quality of life in genetic neuromuscular disease questionnaire (QoLgNMD).
Measured: baseline, 6 and 12 months
The difference between DM1-ActivC at baseline visit, the visit at 6 months and final visit
The activity defined by the scores obtained using the DM1 activity and participation scale for clinical use (DM1-ActivC).
Measured: baseline, 6 and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2022
- Study start29 Nov 2024
- Primary completionNov 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Steinert's DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 142 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Pascal LAFORÊT, MD, PhD, Principal investigator, Neurology Department, Raymond Poincaré Hospital, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05532813
- Sponsor's protocol ID
- APHP220832
- Other IDs
- 2023-507660-39-00, 2023
- Acronym
- METFORMYO
- Status last verified
- Nov 2025
- First submitted
- 6 Sep 2022
- Last updated
- 24 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Berard C, Payan C, Hodgkinson I, Fermanian J; MFM Collaborative Study Group. A motor function measure for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005 Jul;15(7):463-70. doi: 10.1016/j.nmd.2005.03.004.
PubMed 16106528Landfeldt E, Nikolenko N, Jimenez-Moreno C, Cumming S, Monckton DG, Faber CG, Merkies ISJ, Gorman G, Turner C, Lochmuller H. Change over time in ability to perform activities of daily living in myotonic dystrophy type 1. J Neurol. 2020 Nov;267(11):3235-3242. doi: 10.1007/s00415-020-09970-6. Epub 2020 Jun 15.
PubMed 32542526
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study
- ClinicalTrials.gov ID
- NCT05532813
- Acronym / sponsor ID
- METFORMYO / APHP220832
- Conditions
- Steinert's Disease, Myotonic Dystrophy 1, Metformin
- Keywords
- Steinert's Disease, Myotonic Dystrophy 1, Metformin, Muscle function
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 142 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 29 Nov 2024 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 8 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- The hand-grip strength;
- The thumb-index pinch strength;
- The locomotor function;
- The respiratory function;
- The cardiac function;
- The quality of life;
- The daily and social activity.
Detailed description
This is a multicenter, national, comparative study comparing the efficacy and safety of metformin and placebo in patients (1:1 ratio between the 2 groups) with DM1.
Population of study participants: patients with biochemically and/or genetically confirmed DM1 disease already followed in the referral and competence departments, as well as new patients.
All patients will be included by a neuromuscular specialist from French centers participating in the research.
Enrolled patients were randomly assigned (71 patients per group with 1:1 ratio) to either metformin therapy or a placebo, using a centralized randomization procedure.
Metformin or placebo will be administered orally and titrated as recommended in diabetic patients. Initial digestive effects (nausea, vomiting and constipation) of metformin that can be observed in the first days. 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. In case of bad digestive tolerance, the dosage should be decrease and the maximum tolerated dosage of metformin should be used. The evaluations of muscle function, walking test, respiratory and cardiac function, quality of life, and tolerance will be assessed at M6 and M12, in the neuromuscular centers. With the estimated effect size, we believe that the inclusion capacities evaluated at 8 to 12 patients per center over one year (18 reference centers involved) will allow to determine a significant difference of MFM score 12 months after inclusion. Dose titration, monitoring of side effects and dose adjustments will be assessed at each visit according to the site endocrinologist advice, if necessary.
Statistical analysis: The difference between the score at 12 months and baseline will be compared between treatment groups using the Student T-test.
Secondary efficacy endpoints evaluating the evolution of symptoms will be analyzed using either a GMM or a GEE for continuous and categorical variables, respectively.
Other quantitative variables will be compared using the Student t-test (or a non-parametric test if the distribution remains skewed following transformation), while categorical variables will be analyzed using either the Chi-squared or the Fisher-exact tests.
All efficacy endpoints will be analysed on an intention-to- treat basis and safety endpoints on a per-protocol basis.
All statistical tests will be performed with a level of significance of 5%. No interim analysis will be performed.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Metformin arm | Experimental | Drug: Treatment takenPatients randomized in Metformin arm will take metformin orally. |
| Placebo receivers | Placebo comparator | Drug: Treatment takenPatients randomized in placebo arm will take placebo orally in the same procedure as metformin taken. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
- Women of childbearing potential under efficient contraception during treatment
Key exclusion criteria (13 listed)
- 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)
- Respiratory:
- Creatinine clearance inferior to 50 ml/min
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Treatment takenTreatment (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. | Drug | Metformin arm, Placebo receivers |
Primary outcome
Change of muscle function
Time frame: at baseline and 12 months
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.
9 secondary outcomes
Safety endpoint
Time frame: through study completion, an average of 30 month
Change of muscle function between baseline and 6 months
Time frame: at baseline and 6 months
The hand-grip strength
Time frame: baseline, 6 and 12 months
The thumb-index pinch strength
Time frame: baseline, 6 and 12 months
The locomotor function
Time frame: baseline, 6 and 12 months
The respiratory function
Time frame: baseline, 6 and 12 months
The cardiac function
Time frame: baseline, 6 and 12 months
Quality of life assessement
Time frame: baseline, 6 and 12 months
The difference between DM1-ActivC at baseline visit, the visit at 6 months and final visit
Time frame: baseline, 6 and 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2025.
- Background: PubMed 16106528Berard C, Payan C, Hodgkinson I, Fermanian J; MFM Collaborative Study Group. A motor function measure for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005 Jul;15(7):463-70. doi: 10.1016/j.nmd.2005.03.004.
- Background: PubMed 32542526Landfeldt E, Nikolenko N, Jimenez-Moreno C, Cumming S, Monckton DG, Faber CG, Merkies ISJ, Gorman G, Turner C, Lochmuller H. Change over time in ability to perform activities of daily living in myotonic dystrophy type 1. J Neurol. 2020 Nov;267(11):3235-3242. doi: 10.1007/s00415-020-09970-6. Epub 2020 Jun 15.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Pascal LAFORÊT, MD, PhD principal investigator
Neurology Department, Raymond Poincaré Hospital, APHP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Treatment taken in people with Steinert's Disease and Myotonic Dystrophy 1, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pascal LAFORÊT, MD, PhD (+33 (0)1 47 10 77 36, pascal.laforet@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of muscle function (time frame: at baseline and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05532813, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Nov 2025.