NCT07415837

Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 104 (estimated)
• Sponsor: University Hospital, Clermont-Ferrand · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Accepted

Summary

The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases. MicroRNAs (miRs) are small messengers that help control how cells grow and stay healthy. Some of these, like miR-1, are specifically found in muscles and the heart. Research shows that levels of miR-1 are often abnormal in people with muscle-wasting conditions, but more information are needed to understand how this relates to the severity of the disease. The main goal is to compare the blood levels of miR-1 between four different groups at different ages and severities: 1. Patients with Duchenne or Becker muscular dystrophy (DMD/DMB). 2. Patients with Myotonic Dystrophy Type 1 (Steinert's disease). 3. Patients with congenital myopathies. 4. Healthy volunteers (control group). The main objective is to assess if miR-1 levels can accurately show how a muscular disease is progressing.

Conditions

• Duchenne / Becker Muscular Dystrophy
• Dystrophia Myotonica 1
• Congenital Myopathies
• Healthy Participants

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	dosage of blood biomarker miR1	Patients and controls will be asked to provide blood samples to evaluate their blood level of miR1 biomarker on a unique time participation.

Timeline

• Start date: 2026-02-11
• Primary completion: 2029-03-01
• Completion: 2029-03-01
• First posted: 2026-02-17
• Last updated: 2026-02-25

Locations

1 site across 1 country: France