Clinical trial NCT03258424: Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy
Official title: A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cystic Fibrosis
Lead sponsor: Proteostasis Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2017-08-23
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs
timeFrame: Baseline through Day 21
Secondary outcomes:
measure: t1/2 of multiple oral doses
timeFrame: Baseline through Day 14
measure: Tmax of multiple oral doses
timeFrame: Baseline through Day 14
measure: Cmax of multiple oral doses
timeFrame: Baseline through Day 14
measure: AUC0-t of multiple oral doses
timeFrame: Baseline through Day 14
Other outcomes:
measure: Change in nasal epithelial mRNA and protein expression over time
timeFrame: Baseline through Day 21
measure: Change in sweat chloride over time
timeFrame: Baseline through Day 21
measure: Change in FEV1 over time
timeFrame: Baseline through Day 21
measure: Change in weight over time
timeFrame: Baseline through Day 21
Locations:
city: Belfast
country: United Kingdom
facility: Celerion
city: Manchester
country: United Kingdom
facility: Medicines Evaluation Unit
Study officials:
References: