Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
Recruiting
Phase 3
Interventional Study
Steinert's Disease
Myotonic Dystrophy 1
Metformin
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 70
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 142 (estimated)
- Sponsor
- Assistance Publique - Hôpitaux de Paris · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
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 com…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion 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
* …
Contacts