An observational study in Duchenne Muscular Dystrophy, sponsored by Cooperative International Neuromuscular Research Group. Status unknown at 9 sites in 2 countries. Open to male participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2015-07-28.
Sponsored by Cooperative International Neuromuscular Research Group · Observational
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.
548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.
This study's enrollment of 53 is below the median of 69 across 179 observational studies indexed under Muscular Dystrophies.
Browse Muscular Dystrophies studies →Cooperative International Neuromuscular Research Group is the lead sponsor of 17 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
Tissue Collection
Collection of blood, skin and optional muscle samples
Time frame: 1 day
This study is status unknown, as verified in Jul 2015. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Cooperative International Neuromuscular Research Group