Research of Biomarkers for Disease Diagnosis, Disease Monitoring and Therapeutic Treatment Response in Duchenne Muscular Dystrophy Patients

Genethon1 site in 1 country220 target enrollmentJune 2011

ConditionsDuchenne Muscular Dystrophy (DMD)

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Duchenne Muscular Dystrophy (DMD)
Sponsor: Genethon
Enrollment: 220
Locations: 1
Primary Endpoint: IBiSD aims to identify and validate new and disease-specific biomarkers.
Status: Completed
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

June 2011

End Date

December 2015

Last Updated

9 years ago

Study Type

Observational

Sex

Male

Investigators

Sponsor

Genethon

Responsible Party

Sponsor

Eligibility 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

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

Outcomes

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).