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NCT Number: NCT07415837

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

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.

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Key information

Conditions

Duchenne / Becker Muscular Dystrophy Congenital Myopathies Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dystrophia Myotonica 1 Genetic Diseases, Inborn Genetic Diseases, X-Linked Healthy Participants Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Myotonia Congenita Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

CHU de Clermont-Ferrand

Clermont-Ferrand, France

Location status: Recruiting

Location contact

Catherine Sarret

PRINCIPAL_INVESTIGATOR

Lise Laclautre

CONTACT

[email protected]

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

dosage of blood biomarker miR1

Diagnostic Test

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

Primary outcomes

  1. blood expression level of micro-RNA miR-1

    Time frame: at a unique time of enrollment

    The main goal is to evaluate the interest of miR-1 as a blood biomarker for neuromuscular diseases, specifically muscular dystrophies and congenital myopathies.

Secondary outcomes

  1. Demographic Correlations

    Time frame: at a unique time of enrollment

    The study will analyze the correlation between blood expression levels of miR-1 and the age and sex of the participants

  2. Severity correlation

    Time frame: at a unique time of enrollment

    the study will analyse the correlation between miR-1 levels and the clinical severity of the neuromuscular condition.

    Severity of Myotonic Dystrophy Type 1 (DM1): miR-1 level relation to the severity of the condition, looking at:

    • The clinical phenotype (based on the age of disease onset).
    • The number of CTG nucleotide repeats in the DMPK gene,.
    • The presence of cardiac involvement,.
    • Severity of Duchenne and Becker Muscular Dystrophies (DMD/DMB): miR-1 levels based on:
    • The presence of cardiac involvement,
    • The loss of ambulation (whether the patient has lost the ability to walk)

Study contacts

Contact information is provided by the study sponsor or research team.

Lise Laclautre, PhD

CONTACT

[email protected]

+33473750750

Sponsors and collaborators

Lead sponsor

University Hospital, Clermont-Ferrand

Other

Collaborators

  • iGreD, Université Clermont Auvergne

Registry information

Acronym: Dystro-miR1

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Feb 17, 2026
Registry last updated
Feb 25, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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