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

Proteostasis Therapeutics

Status and phase

Completed

Phase 1

Conditions

Cystic Fibrosis

Treatments

Drug: Placebo

Drug: PTI-428

Study type

Interventional

Funder types

Industry

Identifiers

NCT03258424

PTI-428-05

Details and patient eligibility

About

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.

Full 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.

Enrollment

15 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.