Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
- Registration Number
- NCT03591094
- Lead Sponsor
- Proteostasis Therapeutics, Inc.
- Brief Summary
The study population is comprised of adult subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation and are currently receiving background treatment with tezacaftor/ivacaftor for a minimum of 1 month prior to Day 1. The planned sample size is approximately 40 subjects. 20 subjects will be assigned to PTI-428 dose level 1 or placebo and 20 subjects will be assigned to PTI-428 dose level 2 or placebo. At each dose level, subjects will be randomized at a 3:1 randomization ratio. Subjects will receive once daily oral doses of PTI-428 or placebo for 28 days, while the subjects continue to receive background treatment with tezacaftor/ivacaftor per product label. The study drug administration period will be followed by a 14-day safety follow-up period.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 40
- Confirmed diagnosis of CF with the F508del/F508del genotype on record
- On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a minimum of 1 month on Day 1
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
- Clinically stable with no significant changes in health status within 14 days of Day 1
- Non-smoker and non-tobacco user for a minimum of 28 days prior to screening and for the duration of the study
- Participation in another clinical trial or treatment with an investigational agent within 28 days or 5 half-lives, whichever is longer, prior to Study Day 1
- History of cancer within the past 5 years (excluding cervical cancer in situ with curative therapy for at least one year prior to screening and non-melanoma skin cancer)
- History of organ transplantation
- Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness (as determined by the investigator) requiring an increase or addition of medication, such as antibiotics or corticosteroids, within 14 days of Day 1
- Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline, azithromycin, Pulmozyme®, Cayston®, TOBI®)) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to Day 1
- History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
- Pregnant or nursing women
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description PTI-428 dose level 2 PTI-428 - Placebo PTI-428 Placebo - PTI-428 dose level 1 PTI-428 -
- Primary Outcome Measures
Name Time Method Number of subjects with treatment-emergent adverse events (TEAEs) Baseline through Day 42 Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
- Secondary Outcome Measures
Name Time Method Time of Cmax (Tmax) 28 days Change in FEV1 over time Baseline through Day 42 Change in sweat chloride over time Baseline through Day 42 Maximum plasma concentration (Cmax) 28 days Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t) 28 days
Trial Locations
- Locations (26)
New York Medical College
🇺🇸Valhalla, New York, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
University of Arizona
🇺🇸Tucson, Arizona, United States
University of Iowa, Roy J and Lucille A Carver College of Medicine
🇺🇸Iowa City, Iowa, United States
Stanford University
🇺🇸Stanford, California, United States
Maine Medical Center
🇺🇸Portland, Maine, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit
🇺🇸Louisville, Kentucky, United States
Mount Sinai Beth Israel
🇺🇸New York, New York, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center
🇺🇸Peoria, Illinois, United States
Central Florida Pulmonary Group
🇺🇸Altamonte Springs, Florida, United States
Dartmouth Hitchcock Medical Center
🇺🇸Manchester, New Hampshire, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
The University of Texas Health Science Center at Tyler - Center for Clinical Research
🇺🇸Tyler, Texas, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Michigan Medicine, University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States