Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

Status unconfirmed

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT01772043

Run by

Cooperative International Neuromuscular Research Group

People needed

53

Starts

2012-09-01

Expected to finish

2016-08-01

Last updated by the study team

2015-07-28

Who can join

Age: 4 and older. Sex: male. Healthy volunteers: not accepted.

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

Where it is running

• 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

Full record on ClinicalTrials.gov

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