Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis

NCT ID: NCT03591094

Last Updated: 2020-02-27

Study Results

Results pending

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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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-08-21

Study Completion Date

2019-02-18

Brief Summary

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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

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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PTI-428 dose level 1

Group Type ACTIVE_COMPARATOR

PTI-428

Intervention Type DRUG

Active

PTI-428 dose level 2

Group Type ACTIVE_COMPARATOR

PTI-428

Intervention Type DRUG

Active

Placebo PTI-428

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo

Interventions

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PTI-428

Active

Intervention Type DRUG

Placebo

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Proteostasis Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of Arizona

Tucson, Arizona, United States

Site Status

Stanford University

Stanford, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

Central Florida Pulmonary Group

Altamonte Springs, Florida, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center

Peoria, Illinois, United States

Site Status

University of Iowa, Roy J and Lucille A Carver College of Medicine

Iowa City, Iowa, United States

Site Status

Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, United States

Site Status

Maine Medical Center

Portland, Maine, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Michigan Medicine, University of Michigan

Ann Arbor, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Dartmouth Hitchcock Medical Center

Manchester, New Hampshire, United States

Site Status

Mount Sinai Beth Israel

New York, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

New York Medical College

Valhalla, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Akron Children's Hospital

Akron, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

The University of Texas Health Science Center at Tyler - Center for Clinical Research

Tyler, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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PTI-428-06

Identifier Type: -

Identifier Source: org_study_id

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