Medication Targets Underlying Mechanism of Cystic Fibrosis

Trial Objective

The purpose of this study is to test the effectiveness of a new investigational medication, PTI-801, in treating cystic fibrosis (CF). The results of PTI-801 will be compared to those of a placebo, a capsule that looks like the investigational medication but does not contain an active drug substance. PTI-801 has not been approved by the Food and Drug Administration (FDA). Although it has been given to healthy adults, this is the first time it will be given to people with CF.

Medication Targets Underlying Mechanism of Cystic Fibrosis

The majority of CF therapeutics are aimed at controlling symptoms and complications of CF. PTI-801 is being developed to treat the underlying mechanism of the disease. In laboratory tests, PTI-801 has been shown to work like one of the components of Orkambi®, a medication currently used to treat CF. In laboratory tests, PTI-801 has appeared to have an effect on cells from CF patients, adding to the effect of Orkambi®. 


This trial is active and currently recruiting.

How to Participate

For more information, contact Connie St. Clair at 303.270.2517 or complete the form below.

Who Can Participate

Cystic fibrosis patients who are taking Orkambi® may be eligible to participate. Study participants should have a body mass index (BMI) greater than 18 and less than 30, and be tobacco-free for a minimum of 30 days prior to screening and during the study. Females of child-bearing potential and males capable of fathering a child must meet contraception requirements.

Estimated Time Commitment

Up to 8 visits totaling up to 35 hours

Visits on Day 2 and Day 14 are about 13 hours long.

Trial Location

National Jewish Main Campus, Denver, CO



Trial Sponsors

Proteostasis Therapeutics, Inc.




Any Gender

Principal Investigators

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