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

What this Trial Is About

Researchers are evaluating the effects of metformin compared to a placebo in adults with Myotonic Dystrophy type 1 (DM1), also known as Steinert's Disease. The study aims to see if metformin can improve muscle function, especially motor deficits, which are key features of DM1. The trial also looks at safety and other aspects such as hand-grip strength, respiratory and cardiac function, quality of life, and daily activity. This is a Phase III, multicenter, randomized, double-blind controlled study involving patients with genetically confirmed DM1. Participants are randomly assigned to receive either metformin or a placebo in equal groups. The treatment is taken orally and gradually increased from 500 mg twice daily to a maximum of 1000 mg three times daily over a few weeks if tolerated. Those who experience digestive side effects may have their dose adjusted. The study includes regular assessments at 6 and 12 months, including muscle function tests, walking tests, respiratory and cardiac evaluations, quality of life surveys, and monitoring for side effects. During the study, participants will visit neuromuscular centers for evaluations and dose adjustments as needed. Researchers will collect data at baseline, 6 months, and 12 months to assess changes in muscle function and other health measures. Safety is monitored throughout the study, which may last about 30 months on average per participant. The main outcome measured is the change in muscle function after 12 months, with other secondary outcomes assessed over time to evaluate the overall impact of metformin on disease symptoms and patient well-being.

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