Duchenne Muscular Dystrophy Clinical Trial
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.
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.
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Observational Model: Cohort, Time Perspective: Prospective
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
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|
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|
N/A | |
| Active, not recruiting |
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|
Phase 1/Phase 2 | |
| Active, not recruiting |
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Phase 1/Phase 2 | |
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Phase 1/Phase 2 | |
| Completed |
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|
Phase 1 | |
| Terminated |
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Phase 2 | |
| Completed |
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|
Phase 2 | |
| Completed |
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||
| Recruiting |
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| Recruiting |
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| Recruiting |
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| Completed |
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||
| Terminated |
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|
N/A | |
| Completed |
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|
Phase 1 | |
| Terminated |
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|
Phase 4 | |
| Withdrawn |
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|
Phase 3 |