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Steinert's Disease clinical trials

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NCT ID: NCT05532813 Not yet recruiting - Metformin Clinical Trials

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

METFORMYO
Start date: March 2024
Phase: Phase 3
Study type: Interventional

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: 1. The hand-grip strength; 2. The thumb-index pinch strength; 3. The locomotor function; 4. The respiratory function; 5. The cardiac function; 6. The quality of life; 7. The daily and social activity.

NCT ID: NCT02930408 Terminated - Steinert's Disease Clinical Trials

Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects

Start date: January 2014
Phase: N/A
Study type: Interventional

Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in subjects suffering from dystrophy would detect patients at risk of sudden death. The first stage of work is to validate the tools to detect early stage of infringement systolic function in a population of patients with myotonic dystrophy.

NCT ID: NCT02398786 Recruiting - Clinical trials for Myotonic Dystrophy 1

Myotonic Dystrophy Family Registry

MDFR
Start date: February 2013
Phase:
Study type: Observational [Patient Registry]

The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.

NCT ID: NCT00082108 Recruiting - Muscular Dystrophy Clinical Trials

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

Start date: September 2000
Phase:
Study type: Observational

Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue. The purpose of this registry is to connect people with DM or FSHD with researchers studying these diseases. The registry will offer individuals with DM and FSHD an opportunity to participate in research that focuses of their diseases. The registry will also help scientists to accomplish research on DM and FSHD and to distribute their findings to patients and care providers.