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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-28

Study Completion Date

2018-10-03

Brief Summary

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The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.

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Detailed Description

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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.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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PTI-428

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Group Type ACTIVE_COMPARATOR

PTI-428

Intervention Type DRUG

PTI-428 or placebo will be given QD for 14 days.

Placebo

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

PTI-428 or placebo will be given QD for 14 days.

Interventions

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PTI-428

PTI-428 or placebo will be given QD for 14 days.

Intervention Type DRUG

Placebo

PTI-428 or placebo will be given QD for 14 days.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of CF.
* Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
* Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
* Non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.

Exclusion Criteria

* Participation in another clinical trial or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.
* History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).
* History of organ transplantation.
* Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1.
* History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
* Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.
* Pregnant or nursing women.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No