NCT02718495.

Trial name or title	Study Assessing PTI‐428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis
Methods	Quadruple‐blind, placebo‐controlled, parallel design 3‐arm Phase 2 RCT. Multicentre: 29 centres Sample size: expected to enrol 136 participants. Duration: 28 days of treatment. Part A has 2 groups: the 1st group will enrol adults with CF into a single ascending dose treatment group; the 2nd group will enrol adults with CF, including those on background treatment with ORKAMBI® and those not on a CFTR modulator into a multiple ascending dose treatment group. Part B will enrol adults with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months into a Phase II treatment group consisting of 2 cohorts. Part C will enrol adults with CF, including those on background treatment with KALYDECO® and those not on a CFTR modulator, into a Phase II treatment group consisting of 3 cohorts.
Participants	Age: 18 years and older. Mutation: not specified. Lung function: FEV1 40% ‐ 90% predicted.
Interventions	PTI‐428 versus placebo.
Outcomes	Primary outcome Safety and tolerability as assessed by adverse events, pulmonary function tests, safety labs (haematology, chemistry, and urinalysis, ECGs, physical examinations, and vital signs) Secondary outcomes PK and pharmacodynamic parameters Change in FEV1 Change in sweat chloride Change in weight Change in CFQ‐R Change in nasal epithelial CFTR mRNA and protein expression
Starting date	November 2017.
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