Cystic Fibrosis
Conditions
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.
Interventions
DRUGPTI-428
PTI-428 or placebo will be given QD for 14 days.
DRUGPlacebo
PTI-428 or placebo will be given QD for 14 days.
Sponsors
Proteostasis Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs | Baseline through Day 21 |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
Other
| Measure | Time frame |
|---|---|
| Change in sweat chloride over time | Baseline through Day 21 |
| Change in FEV1 over time | Baseline through Day 21 |
| Change in weight over time | Baseline through Day 21 |
| Change in nasal epithelial mRNA and protein expression over time | Baseline through Day 21 |
Countries
United Kingdom
Outcome results
None listed