Gene therapy for cystic fibrosis with specific CFTR mutations
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This study looks at correcting “stop” mistakes (nonsense mutations) in the CFTR gene, which can cause cystic fibrosis. If your CFTR test shows the exact type of mutation the study needs, you may be able to take part.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have cystic fibrosis (CF)
- Your CFTR genetic test shows a nonsense (stop) mutation on BOTH copies of the CFTR gene
- Your CFTR gene has been fully tested (sequenced) on BOTH gene copies, and results are known
- You are age 8 or older (including minors), and you/your parent or guardian can give consent
- You can take part while not being pregnant and not breastfeeding
- You are not under guardianship/curatorship
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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