Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
Status:
Completed
Trial end date:
2019-02-18
Target enrollment:
Participant gender:
Summary
The study population is comprised of adult subjects with cystic fibrosis (CF) who are
homozygous for the F508del mutation and are currently receiving background treatment with
tezacaftor/ivacaftor for a minimum of 1 month prior to Day 1. The planned sample size is
approximately 40 subjects. 20 subjects will be assigned to PTI-428 dose level 1 or placebo
and 20 subjects will be assigned to PTI-428 dose level 2 or placebo. At each dose level,
subjects will be randomized at a 3:1 randomization ratio. Subjects will receive once daily
oral doses of PTI-428 or placebo for 28 days, while the subjects continue to receive
background treatment with tezacaftor/ivacaftor per product label. The study drug
administration period will be followed by a 14-day safety follow-up period.