Research of Biomarkers in Duchenne Muscular Dystrophy Pat... | NCT01380964

Studies
Research of Biomarkers in Duchenne Muscular Dystrophy Patients

NCT01380964

Sponsor

Genethon

Status

Completed

Last Update Posted

Aug 17, 2016Estimated

Enrollment

220Actual

Phase

Not provided

Conditions

Duchenne Muscular Dystrophy (DMD)

Interventions

Not provided

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2016

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2011

Primary Completion Date

Dec 2015Actual

Completion Date

Dec 2015Actual

First Submitted Date

Jun 23, 2011

First Submission Date that Met QC Criteria

Jun 23, 2011

First Posted Date

Jun 27, 2011Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 16, 2016

Last Update Posted Date

Aug 17, 2016Estimated

Name	Class
Institute of Myology	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

220Actual

Label	Type	Description	Intervention Names
DMD patients		DMD Patients
Control patients		Control patients

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

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
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

Accepts Healthy Volunteers

Yes

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

3 Years

Maximum Age

20 Years

Standard Ages

ChildAdult

Study Population

patients with Duchenne Muscular Dystrophy controls (healthy or patients with a non-related disease)

Sampling Method

Probability Sample

Name	Affiliation	Role
Laurent SERVAIS, MD	Myology Institute	Principal Investigator

Condition Browse Module

MeSH Terms

ID	Term
D020388	Muscular Dystrophy, Duchenne

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

Not provided

Browse Branches

Not provided