Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects With Cystic Fibrosis
1 other identifier
interventional
40
1 country
26
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Aug 2018
Shorter than P25 for phase_2
26 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 6, 2018
CompletedFirst Posted
Study publicly available on registry
July 18, 2018
CompletedStudy Start
First participant enrolled
August 21, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 18, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
February 18, 2019
CompletedFebruary 27, 2020
February 1, 2020
6 months
July 6, 2018
February 25, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Number of subjects with treatment-emergent adverse events (TEAEs)
Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
Baseline through Day 42
Secondary Outcomes (5)
Maximum plasma concentration (Cmax)
28 days
Time of Cmax (Tmax)
28 days
Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t)
28 days
Change in FEV1 over time
Baseline through Day 42
Change in sweat chloride over time
Baseline through Day 42
Other Outcomes (5)
Change in nasal epithelial CFTR mRNA and protein expression over time
Baseline through Day 42
Change in CFQ-R over time
Baseline through Day 42
Cmax of PTI-428 metabolites, if applicable
28 days
- +2 more other outcomes
Study Arms (3)
PTI-428 dose level 1
ACTIVE COMPARATORPTI-428 dose level 2
ACTIVE COMPARATORPlacebo PTI-428
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (26)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
University of Arizona
Tucson, Arizona, 85724, United States
Stanford University
Stanford, California, 94305, United States
National Jewish Health
Denver, Colorado, 80206, United States
Central Florida Pulmonary Group
Altamonte Springs, Florida, 32701, United States
Northwestern University
Chicago, Illinois, 60611, United States
Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center
Peoria, Illinois, 61637, United States
University of Iowa, Roy J and Lucille A Carver College of Medicine
Iowa City, Iowa, 52242, United States
Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
Maine Medical Center
Portland, Maine, 04102, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Michigan Medicine, University of Michigan
Ann Arbor, Michigan, 48109, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Dartmouth Hitchcock Medical Center
Manchester, New Hampshire, 03104, United States
Mount Sinai Beth Israel
New York, New York, 10003, United States
Columbia University Medical Center
New York, New York, 10032, United States
New York Medical College
Valhalla, New York, 10595, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Akron Children's Hospital
Akron, Ohio, 44308, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
The University of Texas Health Science Center at Tyler - Center for Clinical Research
Tyler, Texas, 75708, United States
University of Utah
Salt Lake City, Utah, 84132, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 6, 2018
First Posted
July 18, 2018
Study Start
August 21, 2018
Primary Completion
February 18, 2019
Study Completion
February 18, 2019
Last Updated
February 27, 2020
Record last verified: 2020-02
Data Sharing
- IPD Sharing
- Will not share