Ignite Creation Date:
2025-12-24 @ 3:20 PM
Ignite Modification Date:
2026-02-06 @ 3:06 AM
Study NCT ID:
NCT02109692
Status:
UNKNOWN
Last Update Posted:
2018-05-15
First Post:
2014-04-02
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001800', 'term': 'Blood Specimen Collection'}], 'ancestors': [{'id': 'D013048', 'term': 'Specimen Handling'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D011677', 'term': 'Punctures'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'HEALTH_SERVICES_RESEARCH', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 186}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2014-05-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-05', 'completionDateStruct': {'date': '2019-11', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2018-05-09', 'studyFirstSubmitDate': '2014-04-02', 'studyFirstSubmitQcDate': '2014-04-07', 'lastUpdatePostDateStruct': {'date': '2018-05-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-04-10', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Quantity of serum muscle-derived microRNAs of DMD patients', 'timeFrame': 'up to 12 months', 'description': 'To validate the use of serum muscle-derived microRNAs as biomarkers of DMD patients (compared with healthy subjects)'}], 'secondaryOutcomes': [{'measure': 'severity of the dystrophinopathy', 'timeFrame': 'up to 36 months', 'description': 'to investigate the relationship between circulating levels of these miRNAs and the severity of the dystrophinopathy'}, {'measure': 'progression of the disease', 'timeFrame': 'up to 36 months', 'description': 'to investigate the relationship between circulating levels of these miRNAs and the progression of the disease'}, {'measure': 'specificitiy of miRNA for distrophinopathy', 'timeFrame': 'up to 36 months', 'description': 'to assess the specificity of these markers for dystrophinopathy (comparison with other patients with muscular dystrophy)'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['muscle dystrophies', 'dystromirs', 'longitudinal study', 'miRNA'], 'conditions': ['Muscular Dystrophies', 'Becker Muscular Dystrophy', 'Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': '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 .\n\nThe 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.\n\nClinical data and samples will be recorded at each regular consultation. miRNA levels will be quantified using Real Time Quantitative RT-PCR.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Months', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patient suffers from dystrophinopathy or other muscle dystrophy,\n* Healthy volunteers\n* signed informed consent\n* social insurance\n\nExclusion Criteria:\n\n* patients or parents have not signed the informed consent,'}, 'identificationModule': {'nctId': 'NCT02109692', 'acronym': 'biodystromirs', 'briefTitle': 'Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies', 'organization': {'class': 'OTHER', 'fullName': 'University Hospital, Montpellier'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': '9184'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'cohort', 'description': 'blood sample : doage of miRNA', 'interventionNames': ['Other: blood sample']}], 'interventions': [{'name': 'blood sample', 'type': 'OTHER', 'description': 'dosage of miRNA', 'armGroupLabels': ['cohort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '34395', 'city': 'Montpellier', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Francois Rivier, Professor', 'role': 'CONTACT'}], 'facility': 'Montpellier Hospital', 'geoPoint': {'lat': 43.61093, 'lon': 3.87635}}], 'centralContacts': [{'name': 'Mireille Cossee, MD-PhD', 'role': 'CONTACT', 'email': 'mireille.cosse@inserm.com', 'phone': '0033 4 11 75 98 79'}], 'overallOfficials': [{'name': 'Francois Rivier, PU-PH', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University Hospital, Montpellier'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Hospital, Montpellier', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}