Research of Biomarkers in Duchenne Muscular Dystrophy Patients

Completed

• Conditions: Duchenne Muscular Dystrophy (DMD)

• Registration Number: NCT01380964
• Lead Sponsor: Genethon

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-06
• Study First Submitted: 2011-06-23
• Study First Submitted QC: 2011-06-23
• Study First Posted: 2011-06-27
• Primary Completion: 2015-12
• Study Completion: 2015-12
• Status Verified: 2016-08
• Last Update Submitted: 2016-08-16
• Last Update Posted: 2016-08-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 220

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
IBiSD aims to identify and validate new and disease-specific biomarkers.	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).

Trial Locations

Locations (1)

Institute of Myology; 🇫🇷 Paris, France