NCT01380964

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
220

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jun 2011

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2011

Completed
22 days until next milestone

First Submitted

Initial submission to the registry

June 23, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 27, 2011

Completed
4.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2015

Completed
Last Updated

August 17, 2016

Status Verified

August 1, 2016

Enrollment Period

4.5 years

First QC Date

June 23, 2011

Last Update Submit

August 16, 2016

Conditions

Keywords

Duchenne Muscular Dystrophybiomarkerstranscriptomicsmironicsproteomics

Outcome Measures

Primary Outcomes (1)

  • 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

Study Arms (2)

DMD patients

DMD Patients

Control patients

Control patients

Eligibility Criteria

Age3 Years - 20 Years
Sexmale
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

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

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Institute of Myology

Paris, France

Location

Biospecimen

Retention: SAMPLES WITH DNA

DNA, serum, t lymphocytes, urine

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Laurent SERVAIS, MD

    Myology Institute

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 23, 2011

First Posted

June 27, 2011

Study Start

June 1, 2011

Primary Completion

December 1, 2015

Study Completion

December 1, 2015

Last Updated

August 17, 2016

Record last verified: 2016-08

Locations