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completed

Research of Biomarkers in Duchenne Muscular Dystrophy Patients

key information

study id #: NCT01380964

condition: Duchenne Muscular Dystrophy (DMD)

status: completed

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.

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT01380964

last updated: November 21, 2018

study details

start date: June 2011

estimated completion: December 2015

size / enrollment: 220

primary outcomes:

  • IBiSD aims to identify and validate new and disease-specific biomarkers. [ Time Frame: End of study ]
    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).

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

study contacts

sponsor: Genethon

investigators: Laurent SERVAIS, MD

locations: France

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