Overview

This trial is active, not recruiting.

Condition duchenne muscular dystrophy
Sponsor Cooperative International Neuromuscular Research Group
Start date September 2012
End date August 2016
Trial size 53 participants
Trial identifier NCT01772043, CHAR0312

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm

Primary Outcomes

Measure
Tissue Collection
time frame: 1 day

Eligibility Criteria

Male participants at least 4 years old.

Inclusion Criteria: - 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. Exclusion Criteria: - Investigator assessment of inability to comply with blood and skin sample collection

Additional Information

Description The purpose of this tissue bank is to collect blood and skin samples from participants who are diagnosed with Duchenne muscular dystrophy (DMD) and carry one of nine specific changes in the dystrophin gene. The specific dystrophin changes that we are interested in studying are those that would work with exon-skipping therapies in patients with DMD, specifically deletions of the follow exons: 10-52, 13-50, 29-50, 43-52, 44, 43-50, 45-50, 45-52, 46, 46-47, 46-48, 46-49, 46-51, 46-53, 46-55, 46-60, 47-50, 47-52, 48-50, 49-50, 50, 52, 52-63, 48-52, 49-52, 50-52. These blood and skin samples will be held in a tissue bank at Carolinas Medical Center for future DMD research.
Trial information was received from ClinicalTrials.gov and was last updated in July 2015.
Information provided to ClinicalTrials.gov by Cooperative International Neuromuscular Research Group.