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

Phase 1

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: PTI-428; Drug: Placebo

• Registration Number: NCT03258424
• Lead Sponsor: Proteostasis Therapeutics, Inc.

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2017-07-28
• Study First Submitted: 2017-08-18
• Study First Submitted QC: 2017-08-18
• Study First Posted: 2017-08-23
• Primary Completion: 2018-10-03
• Study Completion: 2018-10-03
• Status Verified: 2020-03
• Last Update Submitted: 2020-03-24
• Last Update Posted: 2020-03-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs	Baseline through Day 21

Secondary Outcome Measures

Name	Time	Method
t1/2 of multiple oral doses	Baseline through Day 14
Tmax of multiple oral doses	Baseline through Day 14
Cmax of multiple oral doses	Baseline through Day 14
AUC0-t of multiple oral doses	Baseline through Day 14

Trial Locations

Locations (2)

Celerion; 🇬🇧 Belfast, United Kingdom

Medicines Evaluation Unit; 🇬🇧 Manchester, United Kingdom