NCT03227471.

Trial name or title	A Study of VX‐445 in Healthy Subjects and Subjects With Cystic Fibrosis
Methods	First‐in‐human, proof‐of‐concept RCT. Parallel design. Multicentre: 38 centres Sample size: expected to enrol 224 participants. Duration: 4 weeks of treatment. The study includes 6 parts, of which the first 3 are conducted in healthy participants, and the last 3 in people with CF.
Participants	Inclusion criteria Age: 18 years and older. Mutation: heterozygous for F508del and an MF mutation (F/MF), or Homozygous for F508del (F/F). Lung function: FEV1 value ≥ 40% and ≤ 90% of predicted mean for age, sex, and height.
Interventions	Group 1: VX‐445 in triple combination with tezacaftor and VX‐561 for 4 weeks. Group 2: placebo.
Outcomes	Primary outcomes Safety and tolerability as assessed by number of participants with AEs and serious AEs Absolute change in FEV1 % predicted (Parts D, E, and F only) (from baseline through day 29) Secondary outcomes Absolute change in sweat chloride concentrations (from baseline through day 29) Relative change in FEV1 % predicted (from baseline through day 29) Absolute change in CFQ‐R respiratory domain score (from baseline through day 29)
Starting date	January 2017.
Contact information	Vertex Pharmaceuticals (medicalinfo@vrtx.com).
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