miR-1 as a blood biomarker for muscular dystrophies and congenital myopathies
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
This project will see if levels of a blood molecule called miR-1 can track disease status in people with Duchenne/Becker muscular dystrophy, myotonic dystrophy type 1, congenital myopathies, and in healthy volunteers.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 104 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | University Hospital, Clermont-Ferrand Academic / other |
| Locations | 1 site (Clermont-Ferrand) |
| Trial ID | NCT07415837 on ClinicalTrials.gov |
What this trial studies
The study measures miR-1 levels in blood samples from four groups: Duchenne/Becker muscular dystrophy, myotonic dystrophy type 1, congenital myopathies, and healthy volunteers, across ages and disease severities. Investigators will compare miR-1 concentrations between groups and correlate levels with clinical severity measures. Participants provide blood samples at the CHU de Clermont-Ferrand and data are analyzed to determine whether miR-1 reliably reflects muscle disease activity. The protocol excludes recent intense exercise and treatments that could alter systemic, muscular, or cardiac biology to reduce confounding.
Who should consider this trial
Good fit: Ideal candidates are people older than 2 years with a confirmed diagnosis of Duchenne or Becker muscular dystrophy, myotonic dystrophy type 1, or a congenital myopathy, as well as healthy volunteers who are affiliated with the French social security system and provide consent.
Not a fit: Patients currently on systemic, muscular, or cardiac treatments that affect biological measurements, those who recently did intense exercise, or people without the eligible diagnoses are unlikely to benefit from this protocol.
Why it matters
Potential benefit: If successful, this could give patients and clinicians a simple blood test to monitor disease progression and treatment response.
How similar studies have performed: Prior research has reported abnormal miR-1 levels in various neuromuscular diseases, providing supportive but not yet definitive evidence that miR-1 can serve as a clinical biomarker.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age: Participants must be older than 2 years of age * Consent: Participants (or their legal guardians) must provide free and informed consent,. For children, the consent is oral for those under 6 years old and written for those over 6,. * Social Security: Every participant must be affiliated with the French social security system. * Participants must have a diagnosed neuromuscular pathology : the eligible pathologies are Myotonic Dystrophy Type 1 (DM1 or Steinert's disease), Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (DMB), or congenital myopathies or are healthy participants. Exclusion Criteria: * Refusal to participate expressed by the subject or their parental authority. * Engaging in intense and unusual physical effort within 10 days before the blood draw. * Current use of any treatment with systemic, muscular, or cardiac effects that could interfere with the study's biological results. * Subjects or their legal guardians who are under tutelage, curatorship, deprived of liberty, or under judicial protection. * Women who are pregnant or breastfeeding. * The presence of an additional pathology that, in the judgment of the clinician, could interfere with the biological findings
Where this trial is running
Clermont-Ferrand
- CHU de Clermont-Ferrand — Clermont-Ferrand, France (Recruiting)
Study contacts
- Principal investigator: Catherine Sarret, MD, PhD, Prof — University Hospital, Clermont-Ferrand
- Study coordinator: Lise Laclautre, PhD
- Email: promo_interne_drci@chu-clermontferrand.fr
- Phone: +33473750750
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.