SPL84 for cystic fibrosis with 3849+10kb mutation
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This trial tests a new drug called SPL84 for people with cystic fibrosis who have a specific gene mutation (3849+10kb C->T). It aims to see if the drug is safe and can improve lung function. The study has two paths: one for people not taking certain CF drugs, and one for those who are already on Trikafta or Alyftrek.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have cystic fibrosis and a specific genetic mutation called 3849+10kb C->T in at least one of your CFTR genes.
- Your body mass index (BMI) must be at least 17.
- Your lung function (FEV1) must be between 40% and 90% of normal (or 40% to 80% if you are in Cohort 4).
- You must not have smoked or vaped for at least 6 months before the study starts.
- You cannot take CFTR modulator drugs like Trikafta, Orkambi, or Symdeko for 30 days before the study (unless you are in Cohort 4, where you must be on a stable dose of Trikafta or Alyftrek).
- You must be stable — no recent lung infections, changes in CF medications, or serious coughing up of blood.
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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