Trial Number
647-26
Condition
Cystic Fibrosis
Participant Age Range
18 years and older
Participant Gender
Any
Enrolling Participants
Yes
Overview
“A Phase 2a, Randomized, Placebo-Controlled, Double-Blind Multiple Ascending Dose Study in Patients with Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84”
Researchers are studying an investigational inhaled treatment called SPL84 for people with cystic fibrosis who carry the 3849+10Kb C>T genetic mutation. SPL84 is designed to help the body produce more functional CFTR protein, which may improve lung function and respiratory symptoms. Participants will receive either SPL84 or placebo by inhalation and will undergo regular lung function testing, health assessments, and safety monitoring to evaluate the treatment's safety, tolerability, and potential effectiveness.