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Tests treatment safety and results for Steinert's Disease
Official title Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
ClinicalTrials.gov ID: NCT05532813
What this study is testing
What is Treatment taken?
Treatment taken is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for steinert's disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 70
You may be able to join if
- 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
You likely can't join if
- 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
See the full eligibility criteria
- 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
- 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
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Garches, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Garches, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.