Research of Biomarkers in Duchenne Muscular Dystrophy Patients

Duchenne Muscular Dystrophy (DMD) Clinical Trial

— IBISD  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01380964
• Other study ID #: GEE006.10
• Status: Completed
• Phase: N/A
• First received: June 23, 2011
• Last updated: August 16, 2016
• Start date: June 2011
• Est. completion date: December 2015

Study information

• Verified date: August 2016
• Source: Genethon
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Committee for the Protection of Personnes
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 220
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Male
• Age group: 3 Years to 20 Years

Eligibility

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

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy (DMD)
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
France	Institute of Myology	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Genethon	Institute of Myology

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	IBiSD aims to identify and validate new and disease-specific biomarkers.	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).	End of study	No