Last updated on January 2019

Study Assessing the Safety Tolerability Pharmacokinetics Food Effect and Drug-Drug Interactions of PTI-801 in Healthy Volunteers and Safety Tolerability and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis


Brief description of study

This trial will consist of two parts: Part 1 and Part 2. Part 1 will enroll adult healthy volunteers (HV) into four treatment groups. The first group will enroll HV into a single ascending dose (SAD) treatment group consisting of three cohorts. The second group will enroll HV into a multiple ascending dose (MAD) treatment group consisting of three cohorts. The third group will enroll HV into a food effect (FE) treatment group consisting of one cohort. The fourth group will enroll HV into a drug-drug interactions (DDI) treatment group consisting of one cohort. Part 2 Cohorts 1 through 3 will enroll adult subjects with cystic fibrosis (CF) currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months into a MAD treatment group consisting of three cohorts. Part 2 Cohort 4 will enroll adult subjects with CF not currently receiving or have received cystic fibrosis conductance regulator (CFTR) modulator therapy within 30 days prior to Day 1. Approximately 125 subjects will be enrolled.

Detailed Study Description

PART 1 The SAD treatment group is comprised of three cohorts where HV will be randomized to either PTI-801 or placebo. Following the conclusion of at least three SAD cohorts, a set of HV will participate in an assigned MAD treatment group. The MAD treatment group is comprised of three cohorts where subjects will be randomized to receive either PTI-801 or placebo once daily (QD) for a total of 7 days. Following the conclusion of at least three SAD cohorts, a set of HV will participate in a FE treatment group. The FE treatment group is comprised of one cohort where subjects will be randomized to receive an initial single dose of PTI-801 either after an overnight fast of at least 10 hours (fasted group) or after an overnight fast of at least 10 hours followed by the consumption of a high fat high and high calorie meal (fed group). After a 10 day washout period, subjects will cross over to the opposite group and receive a second dose of PTI-801. Following the conclusion of at least two MAD cohorts, a set of HV will participate in a DDI treatment group. The DDI treatment group is comprised of one cohort where subjects will receive a 3-drug cocktail consisting of caffeine, bupropion, and midazolam on Day 1. On Day 4, subjects will be randomized to receive either PTI-801 or placebo QD for a total of 12 days. On Day 17, subjects will receive the 3-drug cocktail in combination with PTI-801 or placebo.

PART 2 Part 2 is comprised of a MAD treatment group with three cohorts and a co-administration group with one cohort. Following conclusion of the complementary HV MAD cohort in Part 1, a set of adult subjects diagnosed with CF currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months will participate in the Part 2 complementary CF MAD cohort. Subjects will be randomized to receive either PTI-801 or placebo QD for a total of 14 days. Following conclusion of CF MAD Cohort 1 in Part 2, a set of adult subjects diagnosed with CF not currently receiving or have received background CFTR modulator therapy for a minimum of 30 days prior to Day 1 will participate in the Part 2 CF PTI-801 and PTI-808 co-administration cohort. Subjects will be randomized to receive either PTI-801 co-administered with PTI-808 or placebos QD for a total of 14 days.

Clinical Study Identifier: NCT03140527

Contact Investigators or Research Sites near you

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Ashley Warden

Medical University of South Carolina
Charleston, SC United States
5.28miles
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Recruitment Status: Open


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