Research of Biomarkers in Duchenne Muscular Dystrophy Patients (IBISD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2011 by Genethon
Sponsor:
Collaborator:
Institute of Myology
Information provided by (Responsible Party):
Genethon
ClinicalTrials.gov Identifier:
NCT01380964
First received: June 23, 2011
Last updated: June 10, 2013
Last verified: June 2011
  Purpose

The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.


Condition
Duchenne Muscular Dystrophy (DMD)

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Research of Biomarkers for Disease Diagnosis, Disease Monitoring and Therapeutic Treatment Response in Duchenne Muscular Dystrophy Patients

Resource links provided by NLM:


Further study details as provided by Genethon:

Primary Outcome Measures:
  • IBiSD aims to identify and validate new and disease-specific biomarkers. [ Time Frame: End of study ] [ Designated as safety issue: No ]
    This study will establish the relevance of urinary and blood biomarkers for the diagnosis, follow-up and assessment of treatment response in patients with DMD (IBiSD1, 2 and 4). IBiSD will also attempt to establish the seroprevalence to the different strains of AAV in patients with DMD (IBiSD3).


Biospecimen Retention:   Samples With DNA

DNA, serum, t lymphocytes, urine


Estimated Enrollment: 220
Study Start Date: June 2011
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts
DMD patients
DMD Patients
Control patients
Control patients

  Eligibility

Ages Eligible for Study:   3 Years to 20 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population

patients with Duchenne Muscular Dystrophy controls (healthy or patients with a non-related disease)

Criteria

Inclusion Criteria:

  • FOR PATIENTS:
  • Diagnosis of DMD confirmed by genetic testing
  • Age over 3 years
  • Weight over 15 kg
  • Informed consent signed
  • FOR CONTROLS:
  • Age over 3 years
  • Male gender
  • Weight over 15 kg
  • Subjects with national health insurance coverage
  • Informed consent signed
  • Nonacute or chronic muscular, allergic, infectious, endocrine or inflammatory disorder in the 3 weeks preceding inclusion

Exclusion Criteria:

  • FOR PATIENTS:
  • Concomitant chronic or acute muscular, endocrine, infectious, allergic or inflammatory disorder in the three weeks preceding the blood test
  • Intake of medicines other than angiotensin-converting enzyme inhibitors, beta blockers, dietary supplements, vitamins, alendronate and methylphenidate. Steroids (and medicines prescribed with them such as calcium supplements and proton pump inhibitors) will be discussed
  • Mental retardation or autism
  • Vaccination or treatment with immunoglobulins within the three months preceding inclusion
  • FOR CONTROLS:
  • Concomitant chronic or acute muscular, neurological (including mental retardation and autism), infectious or inflammatory disorder in the three weeks preceding the blood test
  • Vaccination or treatment with immunoglobulins within the three months preceding inclusion
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01380964

Locations
France
Institute of Myology Recruiting
Paris, France
Contact: Laurent Servais, MD    +33(0)142169966    l.servais@institut-myologie.org   
Principal Investigator: Laurent Servais, MD         
Sponsors and Collaborators
Genethon
Institute of Myology
Investigators
Principal Investigator: Laurent SERVAIS, MD Myology Institute
  More Information

No publications provided

Responsible Party: Genethon
ClinicalTrials.gov Identifier: NCT01380964     History of Changes
Other Study ID Numbers: GEE006.10
Study First Received: June 23, 2011
Last Updated: June 10, 2013
Health Authority: France: Committee for the Protection of Personnes

Keywords provided by Genethon:
Duchenne Muscular Dystrophy
biomarkers
transcriptomics
mironics
proteomics

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on September 22, 2014