IBISD: Research of Biomarkers in Duchenne Muscular Dystrophy Patients

Sponsor

Genethon (Other)

Overall Status

Completed

CT.gov ID

NCT01380964

Collaborator

Institute of Myology (Other)

220

Enrollment

Location

Duration (Months)

4.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy (DMD)

Study Design

Study Type:

Observational

Actual Enrollment :

220 participants

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

Study Start Date :

Jun 1, 2011

Actual Primary Completion Date :

Dec 1, 2015

Actual Study Completion Date :

Dec 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
DMD patients DMD Patients
Control patients Control patients

Outcome Measures

Primary Outcome Measures

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).

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 20 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Yes

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