Research of Biomarkers in Duchenne Muscular Dystrophy Patients

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01380964

Acronym

IBISD

Enrollment

220

Registered

2011-06-27

Start date

2011-06-30

Completion date

2015-12-31

Last updated

2016-08-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy, biomarkers, transcriptomics, mironics, proteomics

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

3 Years to 20 Years

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

Design outcomes

Primary

Measure	Time frame	Description
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).

Countries

France

Outcome results