Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
Public ClinicalTrials.gov record NCT03591094. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03591094
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 40 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 21, 2018 (Actual)
- Primary completion
- Feb 18, 2019 (Actual)
- Completion
- Feb 18, 2019 (Actual)
- Last update posted
- Feb 27, 2020
2018 – 2019
United States locations
- U.S. sites
- 26
- U.S. states
- 21
- U.S. cities
- 24
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | — |
| University of Arizona | Tucson | Arizona | 85724 | — |
| Stanford University | Stanford | California | 94305 | — |
| National Jewish Health | Denver | Colorado | 80206 | — |
| Central Florida Pulmonary Group | Altamonte Springs | Florida | 32701 | — |
| Northwestern University | Chicago | Illinois | 60611 | — |
| Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center | Peoria | Illinois | 61637 | — |
| University of Iowa, Roy J and Lucille A Carver College of Medicine | Iowa City | Iowa | 52242 | — |
| Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit | Louisville | Kentucky | 40202 | — |
| Maine Medical Center | Portland | Maine | 04102 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Michigan Medicine, University of Michigan | Ann Arbor | Michigan | 48109 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | — |
| Dartmouth Hitchcock Medical Center | Manchester | New Hampshire | 03104 | — |
| Mount Sinai Beth Israel | New York | New York | 10003 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| New York Medical College | Valhalla | New York | 10595 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | — |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | — |
| The University of Texas Health Science Center at Tyler - Center for Clinical Research | Tyler | Texas | 75708 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03591094, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 27, 2020 · Synced Oct 24, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03591094 live on ClinicalTrials.gov.