An observational study in Becker Muscular Dystrophy, sponsored by Cooperative International Neuromuscular Research Group. Status unknown at 16 sites in 4 countries. Open to male participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2018-06-15.
Sponsored by Cooperative International Neuromuscular Research Group · Observational
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. The investigators will characterize the Becker muscular dystrophy phenotype, and correlate specific abnormal dystrophin proteins with the range of clinical outcomes.
We will utilize the Cooperative International Neuromuscular Research group (CINRG) network to collect cohorts of Becker muscular dystrophy (BMD) patients with in-frame deletions in the dystrophin gene. We will collect clinical data across multiple body systems and correlate these findings to the high-resolution deletion break-point mapping performed from the tissue samples. We will investigate the observed variability to deepen our understanding of molecular mechanisms relevant to the optimization of exon skipping therapeutic approaches.
548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.
This study's enrollment of 85 is above 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.
BMD participants over 4 years of age with in-frame deletions in the dystrophin gene.
Exclusion Criteria:
BMD participants over 4 years of age with in-frame deletions in the dystrophin gene. .
Strength and function
Time frame: Annual
Quality of life
These questionnaires include: * Pediatric Quality of Life Inventory (PedsQL) * Pediatrics and Adult Neuromuscular module Quality of Life (NeuroQOL)
Time frame: Annual
Medical history assessment - ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests
Time frame: Annual
This study is status unknown, as verified in Jun 2018. You cannot join it, but the record below documents what was studied.
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Cooperative International Neuromuscular Research Group