Muscular DystrophiesbiodystromirsNCT02109692
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
A study testing blood sample in people with Muscular Dystrophies and Becker Muscular Dystrophy, run by University Hospital, Montpellier at 1 site in France.
Trial summary
Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child. Although the development is rapidly progressive , there is variability in the severity of the disease between DMD patients that do not correlate with the type of mutations in… Read more about this study
- Who is it for?
- Ages 18 months to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Muscular Dystrophies, Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
- What is being tested?
- blood sample
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 186 participants
- Where
- 1 siteFrance
- Study dates
- 19 May 2014to Nov 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 18 months to 80 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Patient suffers from dystrophinopathy or other muscle dystrophy,
- Healthy volunteers
- signed informed consent
- social insurance
Who cannot join
- patients or parents have not signed the informed consent,
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Montpellier Hospital
Montpellier, 34395
Francois Rivier, Professor
About this study
Official title: Quantification of Muscle Specific microRNAs in the Serum of Patients With Duchenne Muscular Dystrophy (DMD) and Becker (BMD) : Evaluation of the Inters-est of These Biomarkers in Patients Care
Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child. Although the development is rapidly progressive , there is variability in the severity of the disease between DMD patients that do not correlate with the type of mutations in the DMD gene. There are no easily measurable biomarkers for monitoring the DMD or moderate form of the disease, Becker muscular dystrophy (BMD ) . MicroRNAs (miRNAs) are involved in most cellular processes , and their expression pattern is a signature of the state of a cell . They represent a potential class of diagnostic and prognostic biomarkers. Some are specific for the skeletal myogenesis , and changes in their pattern of expression are associated with muscle diseases including muscular dystrophy. The levels of muscle- specific miRNAs are indeed greatly increased in the serum of DMD and BMD compared to control patients .
The main objective of this is to validate the use of serum muscle-derived microRNAs as biomarkers of DMD patients (compared with healthy subjects). Secondary objectives are i) to investigate the relationship between circulating levels of these miRNAs and the severity of the dystrophinopathy (DMD vs BMD) and also the progression of the disease (longitudinal study), ii) to assess the specificity of these markers for dystrophinopathy (comparison with other patients with muscular dystrophy), iii) to test candidate miRNAs recently identified but not yet analyzed in the serum of patients.
Clinical data and samples will be recorded at each regular consultation. miRNA levels will be quantified using Real Time Quantitative RT-PCR.
- Being tested
- blood sample
- Keywords
- muscle dystrophies, dystromirs, longitudinal study, miRNA
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood sampleOther
dosage of miRNA
| Study group | Role | What they receive |
|---|---|---|
| cohort | Other | blood sample : doage of miRNA Receives: Other: blood sample |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantity of serum muscle-derived microRNAs of DMD patients
To validate the use of serum muscle-derived microRNAs as biomarkers of DMD patients (compared with healthy subjects)
Measured: up to 12 months
Secondary outcomes (3)
severity of the dystrophinopathy
to investigate the relationship between circulating levels of these miRNAs and the severity of the dystrophinopathy
Measured: up to 36 months
progression of the disease
to investigate the relationship between circulating levels of these miRNAs and the progression of the disease
Measured: up to 36 months
specificitiy of miRNA for distrophinopathy
to assess the specificity of these markers for dystrophinopathy (comparison with other patients with muscular dystrophy)
Measured: up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2014 (estimated)
- Study start19 May 2014
- Primary completionNov 2018 (estimated)
- Study completionNov 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- More trials run by University Hospital, MontpellierThe 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
- Health services research
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 186 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Montpellier
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Francois Rivier, PU-PH, Principal investigator, University Hospital, Montpellier
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02109692
- Sponsor's protocol ID
- 9184
- Acronym
- biodystromirs
- Status last verified
- May 2018
- First submitted
- 2 Apr 2014
- Last updated
- 15 May 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Quantification of Muscle Specific microRNAs in the Serum of Patients With Duchenne Muscular Dystrophy (DMD) and Becker (BMD) : Evaluation of the Inters-est of These Biomarkers in Patients Care
- ClinicalTrials.gov ID
- NCT02109692
- Acronym / sponsor ID
- biodystromirs / 9184
- Keywords
- muscle dystrophies, dystromirs, longitudinal study, miRNA
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 186 participants (estimated)
- Lead sponsor
- University Hospital, MontpellierUniversity, hospital or other
- Study start
- 19 May 2014 (actual)
- Primary completion
- Nov 2018 (estimated)
- Study completion
- Nov 2019 (estimated)
- First posted
- 10 Apr 2014 (estimated)
- 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.
Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child. Although the development is rapidly progressive , there is variability in the severity of the disease between DMD patients that do not correlate with the type of mutations in the DMD gene. There are no easily measurable biomarkers for monitoring the DMD or moderate form of the disease, Becker muscular dystrophy (BMD ) . MicroRNAs (miRNAs) are involved in most cellular processes , and their expression pattern is a signature of the state of a cell . They represent a potential class of diagnostic and prognostic biomarkers. Some are specific for the skeletal myogenesis , and changes in their pattern of expression are associated with muscle diseases including muscular dystrophy. The levels of muscle- specific miRNAs are indeed greatly increased in the serum of DMD and BMD compared to control patients .
The main objective of this is to validate the use of serum muscle-derived microRNAs as biomarkers of DMD patients (compared with healthy subjects). Secondary objectives are i) to investigate the relationship between circulating levels of these miRNAs and the severity of the dystrophinopathy (DMD vs BMD) and also the progression of the disease (longitudinal study), ii) to assess the specificity of these markers for dystrophinopathy (comparison with other patients with muscular dystrophy), iii) to test candidate miRNAs recently identified but not yet analyzed in the serum of patients.
Clinical data and samples will be recorded at each regular consultation. miRNA levels will be quantified using Real Time Quantitative RT-PCR.
Study design
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| cohort | Other | Other: blood sampleblood sample : doage of miRNA |
Who may qualify
- Ages
- 18 months to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Patient suffers from dystrophinopathy or other muscle dystrophy,
- Healthy volunteers
- signed informed consent
- social insurance
Key exclusion criteria (1 listed)
- patients or parents have not signed the informed consent,
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 |
|---|---|---|
| blood sampledosage of miRNA | Other | cohort |
Primary outcome
Quantity of serum muscle-derived microRNAs of DMD patients
Time frame: up to 12 months
To validate the use of serum muscle-derived microRNAs as biomarkers of DMD patients (compared with healthy subjects)
3 secondary outcomes
severity of the dystrophinopathy
Time frame: up to 36 months
progression of the disease
Time frame: up to 36 months
specificitiy of miRNA for distrophinopathy
Time frame: up to 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2018.
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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. This study is not recruiting at the moment.
Francois Rivier, PU-PH principal investigator
University Hospital, Montpellier
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing blood sample in people with Muscular Dystrophies and Becker Muscular Dystrophy, run by University Hospital, Montpellier 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 1 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: status not confirmed, as of the sponsor's last update on 15 May 2018. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mireille Cossee, MD-PhD (0033 4 11 75 98 79, mireille.cosse@inserm.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quantity of serum muscle-derived microRNAs of DMD patients (time frame: up to 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 May 2018.