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

Completed · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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.

Key facts

Study ID

NCT03591094

Run by

Proteostasis Therapeutics, Inc.

People needed

40

Starts

2018-08-21

Expected to finish

2019-02-18

Last updated by the study team

2020-02-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• University of Arizona — Tucson, Arizona, United States
• Stanford University — Stanford, California, United States
• National Jewish Health — Denver, Colorado, United States
• Central Florida Pulmonary Group — Altamonte Springs, Florida, United States
• Northwestern University — Chicago, Illinois, United States
• Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center — Peoria, Illinois, United States
• University of Iowa, Roy J and Lucille A Carver College of Medicine — Iowa City, Iowa, United States
• Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit — Louisville, Kentucky, United States
• Maine Medical Center — Portland, Maine, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Michigan Medicine, University of Michigan — Ann Arbor, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Dartmouth Hitchcock Medical Center — Manchester, New Hampshire, United States
• Mount Sinai Beth Israel — New York, New York, United States
• Columbia University Medical Center — New York, New York, United States
• New York Medical College — Valhalla, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• The University of Texas Health Science Center at Tyler - Center for Clinical Research — Tyler, Texas, United States

Full record on ClinicalTrials.gov

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