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.
Similar studies
Other trials that look related to this one.
This study looks at the mouth health and saliva of people with OPMD and their household members. By understanding saliva changes, researchers hope to learn more about the disease and find ways to help manage symptoms.
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
This study tests a new gene therapy called BBM-D101 for boys with Duchenne muscular dystrophy. The goal is to see if it is safe and can help improve muscle function. It is for boys aged 4 to 8 who can still walk.
This trial tests a new gene editing treatment called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD) whose genetic mutation falls within a specific range of the dystrophin gene (exons 45-55). The goal is to see if it is safe and can help improve muscle function.
This study is building a national registry (a database) for people with Oculo-Pharyngeal Muscular Dystrophy (OPMD) in Israel. Joining will help researchers understand how OPMD progresses over time and may lead to better care.
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
Hear when a new Oculopharyngeal Muscular Dystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.