Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
1 other identifier
observational
53
2 countries
9
Brief Summary
We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the dystrophin gene that could be treated by antisense oligonucleotide (AO) drugs.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Sep 2012
Longer than P75 for all trials
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2012
CompletedFirst Submitted
Initial submission to the registry
January 17, 2013
CompletedFirst Posted
Study publicly available on registry
January 21, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2016
CompletedJuly 28, 2015
July 1, 2015
3.9 years
January 17, 2013
July 27, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Tissue Collection
Collection of blood, skin and optional muscle samples
1 day
Study Arms (1)
Duchenne muscular dystrophy
Eligibility Criteria
Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by mutations in the dystrophin gene. DMD participants over 4 years of age with known mutations that could be targeted by exon skipping therapies will be recruited for this study.
You may qualify if:
- Age 4 and above
- Diagnosis of DMD with a confirmed out-of-frame dystrophin gene deletions that could be corrected by skipping exon 45, 51, or 53 based on past genetic testing.
You may not qualify if:
- Investigator assessment of inability to comply with blood and skin sample collection
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
University of California Davis
Sacramento, California, United States
Stanford University Medical Center
Stanford, California, United States
Children's National Health System
Washington D.C., District of Columbia, United States
Johns Hopkins University School of Medicine, Kennedy Krieger
Baltimore, Maryland, United States
Carolinas Medical Center
Charlotte, North Carolina, United States
Duke Children's Hospital and Health Center
Durham, North Carolina, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Tennessee
Memphis, Tennessee, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
Biospecimen
Blood samples with DNA Skin samples Muscle samples (optional)
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 17, 2013
First Posted
January 21, 2013
Study Start
September 1, 2012
Primary Completion
August 1, 2016
Study Completion
August 1, 2016
Last Updated
July 28, 2015
Record last verified: 2015-07