Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy


Therapeutic category
Restore CFTR Function
Trial status
Completed Participating Centres
Phase I

Full title

A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.

The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.
Trial Reference Number
Trial type
Length of participation
21 weeks
CFTR Modulator
Recruitment target
Last edited date
CF sponsor
Proteostasis Therapeutics, Inc.
CF sponsor type

Who can take part?

Top inclusion criteria
  • Stable on Kalydeco dosing for minimum of 3 months prior to study
  • Non-smoker and non-tobacco user for a minimum of 30 days prior to screening
Top exclusion criteria
  • Participation in another clinical trial or treatment with an investigational agent within 30 days.
  • History of organ transplantation.

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