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Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies (biodystromirs)

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ClinicalTrials.gov Identifier: NCT02109692
Recruitment Status : Recruiting
First Posted : April 10, 2014
Last Update Posted : January 9, 2015
Sponsor:
Information provided by (Responsible Party):
University Hospital, Montpellier

April 2, 2014
April 10, 2014
January 9, 2015
May 2014
November 2017   (Final data collection date for primary outcome measure)
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)
Same as current
Complete list of historical versions of study NCT02109692 on ClinicalTrials.gov Archive Site
  • severity of the dystrophinopathy [ Time Frame: up to 36 months ]
    to investigate the relationship between circulating levels of these miRNAs and the severity of the dystrophinopathy
  • progression of the disease [ Time Frame: up to 36 months ]
    to investigate the relationship between circulating levels of these miRNAs and the progression of the disease
  • specificitiy of miRNA for distrophinopathy [ Time Frame: up to 36 months ]
    to assess the specificity of these markers for dystrophinopathy (comparison with other patients with muscular dystrophy)
Same as current
Not Provided
Not Provided
 
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
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.

Not Provided
Interventional
Not Applicable
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Health Services Research
  • Dystrophinopathies,
  • Muscular Dystrophies
Other: blood sample
dosage of miRNA
cohort
blood sample : doage of miRNA
Intervention: Other: blood sample
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
186
Same as current
May 2018
November 2017   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patient suffers from dystrophinopathy or other muscle dystrophy,
  • Healthy volunteers
  • signed informed consent
  • social insurance

Exclusion Criteria:

  • patients or parents have not signed the informed consent,
Sexes Eligible for Study: All
18 Months to 80 Years   (Child, Adult, Senior)
Yes
Contact: Mireille Cossee, MD-PhD 0033 4 11 75 98 79 mireille.cosse@inserm.com
France
 
 
NCT02109692
9184
Yes
Not Provided
Not Provided
University Hospital, Montpellier
University Hospital, Montpellier
Not Provided
Principal Investigator: Francois Rivier, PU-PH University Hospital, Montpellier
University Hospital, Montpellier
January 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP