Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis
NCT ID: NCT02718495
Last Updated: 2019-03-21
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
56 participants
INTERVENTIONAL
2016-07-19
2017-11-28
Brief Summary
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Detailed Description
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PART B Following the conclusion of MAD Cohort 1, a set of adult subjects diagnosed with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months will participate in Part B. The Part B Phase II treatment group is comprised of 2 cohorts where subjects will be randomized to either PTI-428 or placebo. Each dose will be administered QD for a total of 28 days.
PART C Following the conclusion of Part B Phase II, a set of adult subjects diagnosed with CF will participate in Part C. The Part C Phase II treatment group is comprised of 3 cohorts. Part C Cohort 1 will enroll adult subjects with CF who are eligible to take, but not currently taking, ORKAMBI® in accordance with the approved label. Part C Cohort 2 will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Part C Cohort 3 will enroll adult subjects with CF who are not currently on any background therapies and are pancreatic sufficient. Each PTI-428 or placebo dose will be administered QD for a total of 28 days.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part A
Part A consists of two treatment groups, SAD and MAD. Both treatment groups will consist of 3 cohorts. In SAD, subjects will receive a single dose of PTI-428 or placebo. In MAD, subjects will receive once daily dosing of PTI-428 or placebo for 7 days.
PTI-428
Placebo
Part B
Part B will consist of 2 cohorts. Subjects will receive once daily dosing of PTI-428 or placebo for 28 days.
PTI-428
Placebo
Part C
Part C will consist of 3 cohorts. Subjects will receive once daily dosing of PTI-428 or placebo for 28 days.
PTI-428
Placebo
Interventions
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PTI-428
Placebo
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
ALL
No
Sponsors
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Proteostasis Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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Stanford University Medical Center
Stanford, California, United States
Central Florida Pulmonary Group
Altamonte Springs, Florida, United States
University of Florida College of Medicine
Gainesville, Florida, United States
St. Luke's Cystic Fibrosis Center of Idaho
Boise, Idaho, United States
Northwestern University Memorial Hospital
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center Research Institute, Inc.
Kansas City, Kansas, United States
Quintiles Overland Park Phase 1 Unit
Overland Park, Kansas, United States
University of Louisville
Louisville, Kentucky, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Childrens Hospital Boston
Boston, Massachusetts, United States
Universiy of Michigan Health System
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Children's Lung Specialists
Las Vegas, Nevada, United States
Duke University Health System
Durham, North Carolina, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Drexel University College of Medicine
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
St. Paul's Hospital Pacific Lung Research Center
Vancouver, British Columbia, Canada
St. Michael's Hospital
Toronto, Ontario, Canada
Institut de Recherches Cliniques de Montreal
Montreal, Quebec, Canada
Institut Universitaire de Cardiologie et de Pneumologie de Quebec
Québec, Quebec, Canada
University of Copenhagen Rigshospitalet
Copenhagen, , Denmark
Groupe Hospitalier Pellegrin - Hôpital des Enfants
Bordeaux, , France
Hôpital Cochin
Paris, , France
Charite - Campus Virchow-Klinikum
Berlin, , Germany
Universitaetsklinikum Frankfurt-Zentrum der Inneren Medizin
Frankfurt, , Germany
Countries
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Other Identifiers
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PTI-428-01
Identifier Type: -
Identifier Source: org_study_id
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