Gene therapy trial for OPMD with swallowing problems
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests an experimental gene therapy (BB-301) for people with OPMD who have trouble swallowing. It involves a one-time injection into the throat muscles during a short surgery, and researchers will check if it is safe and improves swallowing.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a specific genetic type of OPMD (either one or two copies of certain GCN repeats).
- You have moderate trouble swallowing (dysphagia), confirmed by a special X-ray test.
- You haven't had prior gene therapy or AAV treatment, and your antibodies against AAV9 are low.
- You are not pregnant, and you or your partner agrees to use effective birth control for a year after treatment.
- You haven't had certain throat procedures (like cricopharyngeal myotomy or Botox) or major throat surgery.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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