Duchenne / Becker Muscular DystrophyDystro-miR1NCT07415837
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
A study testing dosage of blood biomarker miR1 in people with Duchenne / Becker Muscular Dystrophy and Dystrophia Myotonica 1, run by University Hospital, Clermont-Ferrand at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and olderHealthy volunteers can take part
- Conditions the study is for
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies
- What is being tested?
- dosage of blood biomarker miR1
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 104 participants
- Where
- 1 siteFrance
- Study dates
- 11 Feb 2026to Mar 2029
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
CHU de Clermont-Ferrand
Clermont-Ferrand
Lise Laclautre, promo_interne_drci@chu-clermontferrand.fr
Catherine Sarret
About this study
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.
Read more
The main goal is to compare the blood levels of miR-1 between four different groups at different ages and severities:
- Patients with Duchenne or Becker muscular dystrophy (DMD/DMB).
- Patients with Myotonic Dystrophy Type 1 (Steinert's disease).
- Patients with congenital myopathies.
- 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
- Being tested
- dosage of blood biomarker miR1
- Keywords
- biomarkers, neuromuscular diseases, micro-RNAs
- Medical subject headings
- Muscular Dystrophy, Duchenne, Myotonic Dystrophy, Myotonia Congenita, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dosage of blood biomarker miR1Diagnostic test
Patients and controls will be asked to provide blood samples to evaluate their blood level of miR1 biomarker on a unique time participation.
| Study group | Role | What they receive |
|---|---|---|
| study group | Experimental | Not described Receives: Diagnostic Test: dosage of blood biomarker miR1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
blood expression level of micro-RNA miR-1
The main goal is to evaluate the interest of miR-1 as a blood biomarker for neuromuscular diseases, specifically muscular dystrophies and congenital myopathies.
Measured: at a unique time of enrollment
Secondary outcomes (2)
Demographic Correlations
The study will analyze the correlation between blood expression levels of miR-1 and the age and sex of the participants
Measured: at a unique time of enrollment
Severity correlation
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)
Measured: at a unique time of enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2026
- Study start11 Feb 2026
- Primary completionMar 2029 (estimated)
- Study completionMar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for Duchenne / Becker Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University Hospital, Clermont-FerrandThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 104 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Clermont-Ferrand
- Sponsor type
- University, hospital or other
- Collaborators
- Université d'Auvergne
- Responsible party
- Sponsor
- Investigators
- Catherine Sarret, MD, PhD, Prof, Principal investigator, University Hospital, Clermont-Ferrand
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07415837
- Sponsor's protocol ID
- RBHP 2025 SARRET (Dystro-miR1)
- Other IDs
- 2025-A01149-40
- Acronym
- Dystro-miR1
- Status last verified
- Feb 2026
- First submitted
- 10 Feb 2026
- Last updated
- 25 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lopez MA, Si Y, Hu X, Williams V, Qushair F, Carlyle J, Alesce L, Conklin M, Gilbert S, Bamman MM, Alexander MS, King PH. Smad8 Is Increased in Duchenne Muscular Dystrophy and Suppresses miR-1, miR-133a, and miR-133b. Int J Mol Sci. 2022 Jul 7;23(14):7515. doi: 10.3390/ijms23147515.
PubMed 35886863Souidi A, Zmojdzian M, Jagla K. Dissecting Pathogenetic Mechanisms and Therapeutic Strategies in Drosophila Models of Myotonic Dystrophy Type 1. Int J Mol Sci. 2018 Dec 18;19(12):4104. doi: 10.3390/ijms19124104.
PubMed 30567354Vignier N, Amor F, Fogel P, Duvallet A, Poupiot J, Charrier S, Arock M, Montus M, Nelson I, Richard I, Carrier L, Servais L, Voit T, Bonne G, Israeli D. Distinctive serum miRNA profile in mouse models of striated muscular pathologies. PLoS One. 2013;8(2):e55281. doi: 10.1371/journal.pone.0055281. Epub 2013 Feb 13.
PubMed 23418438Matsuzaka Y, Kishi S, Aoki Y, Komaki H, Oya Y, Takeda S, Hashido K. Three novel serum biomarkers, miR-1, miR-133a, and miR-206 for Limb-girdle muscular dystrophy, Facioscapulohumeral muscular dystrophy, and Becker muscular dystrophy. Environ Health Prev Med. 2014 Nov;19(6):452-8. doi: 10.1007/s12199-014-0405-7. Epub 2014 Aug 24.
PubMed 25150707Zaharieva IT, Calissano M, Scoto M, Preston M, Cirak S, Feng L, Collins J, Kole R, Guglieri M, Straub V, Bushby K, Ferlini A, Morgan JE, Muntoni F. Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy. PLoS One. 2013 Nov 25;8(11):e80263. doi: 10.1371/journal.pone.0080263. eCollection 2013.
PubMed 24282529Kirby TJ, Chaillou T, McCarthy JJ. The role of microRNAs in skeletal muscle health and disease. Front Biosci (Landmark Ed). 2015 Jan 1;20(1):37-77. doi: 10.2741/4298.
PubMed 25553440Wan X, Wang W, Liu J, Tong T. Estimating the sample mean and standard deviation from the sample size, median, range and/or interquartile range. BMC Med Res Methodol. 2014 Dec 19;14:135. doi: 10.1186/1471-2288-14-135.
PubMed 25524443Croce CM. Causes and consequences of microRNA dysregulation in cancer. Nat Rev Genet. 2009 Oct;10(10):704-14. doi: 10.1038/nrg2634.
PubMed 19763153Lagos-Quintana M, Rauhut R, Yalcin A, Meyer J, Lendeckel W, Tuschl T. Identification of tissue-specific microRNAs from mouse. Curr Biol. 2002 Apr 30;12(9):735-9. doi: 10.1016/s0960-9822(02)00809-6.
PubMed 12007417Rau F, Freyermuth F, Fugier C, Villemin JP, Fischer MC, Jost B, Dembele D, Gourdon G, Nicole A, Duboc D, Wahbi K, Day JW, Fujimura H, Takahashi MP, Auboeuf D, Dreumont N, Furling D, Charlet-Berguerand N. Misregulation of miR-1 processing is associated with heart defects in myotonic dystrophy. Nat Struct Mol Biol. 2011 Jun 19;18(7):840-5. doi: 10.1038/nsmb.2067.
PubMed 21685920Mendell JT. MicroRNAs: critical regulators of development, cellular physiology and malignancy. Cell Cycle. 2005 Sep;4(9):1179-84. doi: 10.4161/cc.4.9.2032. Epub 2005 Sep 15.
PubMed 16096373
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
- ClinicalTrials.gov ID
- NCT07415837
- Acronym / sponsor ID
- Dystro-miR1 / RBHP 2025 SARRET (Dystro-miR1)
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Keywords
- biomarkers, neuromuscular diseases, micro-RNAs
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 104 participants (estimated)
- Lead sponsor
- University Hospital, Clermont-FerrandUniversity, hospital or other
- Collaborators
- Université d'Auvergne
- Study start
- 11 Feb 2026 (actual)
- Primary completion
- Mar 2029 (estimated)
- Study completion
- Mar 2029 (estimated)
- First posted
- 17 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Patients with Duchenne or Becker muscular dystrophy (DMD/DMB).
- Patients with Myotonic Dystrophy Type 1 (Steinert's disease).
- Patients with congenital myopathies.
- Healthy volunteers (control group). The main objective is to assess if miR-1 levels can accurately show how a muscular disease is progressing.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| study group | Experimental | Diagnostic Test: dosage of blood biomarker miR1 |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| dosage of blood biomarker miR1Patients and controls will be asked to provide blood samples to evaluate their blood level of miR1 biomarker on a unique time participation. | Diagnostic test | study group |
Primary outcome
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.
2 secondary outcomes
Demographic Correlations
Time frame: at a unique time of enrollment
Severity correlation
Time frame: at a unique time of enrollment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2026.
- Background: PubMed 35886863Lopez MA, Si Y, Hu X, Williams V, Qushair F, Carlyle J, Alesce L, Conklin M, Gilbert S, Bamman MM, Alexander MS, King PH. Smad8 Is Increased in Duchenne Muscular Dystrophy and Suppresses miR-1, miR-133a, and miR-133b. Int J Mol Sci. 2022 Jul 7;23(14):7515. doi: 10.3390/ijms23147515.
- Background: PubMed 30567354Souidi A, Zmojdzian M, Jagla K. Dissecting Pathogenetic Mechanisms and Therapeutic Strategies in Drosophila Models of Myotonic Dystrophy Type 1. Int J Mol Sci. 2018 Dec 18;19(12):4104. doi: 10.3390/ijms19124104.
- Background: PubMed 23418438Vignier N, Amor F, Fogel P, Duvallet A, Poupiot J, Charrier S, Arock M, Montus M, Nelson I, Richard I, Carrier L, Servais L, Voit T, Bonne G, Israeli D. Distinctive serum miRNA profile in mouse models of striated muscular pathologies. PLoS One. 2013;8(2):e55281. doi: 10.1371/journal.pone.0055281. Epub 2013 Feb 13.
- Background: PubMed 25150707Matsuzaka Y, Kishi S, Aoki Y, Komaki H, Oya Y, Takeda S, Hashido K. Three novel serum biomarkers, miR-1, miR-133a, and miR-206 for Limb-girdle muscular dystrophy, Facioscapulohumeral muscular dystrophy, and Becker muscular dystrophy. Environ Health Prev Med. 2014 Nov;19(6):452-8. doi: 10.1007/s12199-014-0405-7. Epub 2014 Aug 24.
- Background: PubMed 24282529Zaharieva IT, Calissano M, Scoto M, Preston M, Cirak S, Feng L, Collins J, Kole R, Guglieri M, Straub V, Bushby K, Ferlini A, Morgan JE, Muntoni F. Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy. PLoS One. 2013 Nov 25;8(11):e80263. doi: 10.1371/journal.pone.0080263. eCollection 2013.
- Background: PubMed 25553440Kirby TJ, Chaillou T, McCarthy JJ. The role of microRNAs in skeletal muscle health and disease. Front Biosci (Landmark Ed). 2015 Jan 1;20(1):37-77. doi: 10.2741/4298.
- Background: PubMed 25524443Wan X, Wang W, Liu J, Tong T. Estimating the sample mean and standard deviation from the sample size, median, range and/or interquartile range. BMC Med Res Methodol. 2014 Dec 19;14:135. doi: 10.1186/1471-2288-14-135.
- Background: PubMed 19763153Croce CM. Causes and consequences of microRNA dysregulation in cancer. Nat Rev Genet. 2009 Oct;10(10):704-14. doi: 10.1038/nrg2634.
- Background: PubMed 12007417Lagos-Quintana M, Rauhut R, Yalcin A, Meyer J, Lendeckel W, Tuschl T. Identification of tissue-specific microRNAs from mouse. Curr Biol. 2002 Apr 30;12(9):735-9. doi: 10.1016/s0960-9822(02)00809-6.
- Background: PubMed 21685920Rau F, Freyermuth F, Fugier C, Villemin JP, Fischer MC, Jost B, Dembele D, Gourdon G, Nicole A, Duboc D, Wahbi K, Day JW, Fujimura H, Takahashi MP, Auboeuf D, Dreumont N, Furling D, Charlet-Berguerand N. Misregulation of miR-1 processing is associated with heart defects in myotonic dystrophy. Nat Struct Mol Biol. 2011 Jun 19;18(7):840-5. doi: 10.1038/nsmb.2067.
- Background: PubMed 16096373Mendell JT. MicroRNAs: critical regulators of development, cellular physiology and malignancy. Cell Cycle. 2005 Sep;4(9):1179-84. doi: 10.4161/cc.4.9.2032. Epub 2005 Sep 15.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Catherine Sarret, MD, PhD, Prof principal investigator
University Hospital, Clermont-Ferrand
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing dosage of blood biomarker miR1 in people with Duchenne / Becker Muscular Dystrophy and Dystrophia Myotonica 1, run by University Hospital, Clermont-Ferrand at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lise Laclautre, PhD (+33473750750, promo_interne_drci@chu-clermontferrand.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
blood expression level of micro-RNA miR-1 (time frame: at a unique time of enrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07415837, which also lists 11 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2026.