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Restore CFTR Protein Completed with Results
Phase 2 study of PTI-428 drug in people with CF ages 18 and older who have two copies of the F508del CFTR mutation (Proteostasis PTI-428-06)
This study evaluated the safety and tolerability of the drug PTI-428. This study was for people with CF ages 18 and older who have two copies of the F508del CFTR mutation and were already taking tezacaftor/ivacaftor (Symdeko®). A key goal of the study was to determine if PTI-428 could increase the amount of CFTR protein produced in people who were already taking tezacaftor/ivacaftor (Symdeko®).
All participants in this study had been taking tezacaftor/ivacaftor (TEZ/IVA) for at least one month before their first dose of PTI-428. Participants were randomly assigned to receive one of two doses of PTI-428 plus TEZ/IVA treatment or placebo plus TEZ/IVA for 28 days.
Eligibility
See other primary eligibility criteria for more information.
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Age:
18 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Subjects must be currently receiving stable background treatment with tezacaftor/ivacaftor for a minimum of 1 month.
Study Results
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What We Learned:
PTI-428 was generally well tolerated at both dose levels. PTI-428 did not have a significant impact on lung function in participants already taking TEZ/IVA but did increase CFTR protein expression in nasal mucosa by approximately 50% as measured by ELISA (a lab test that detects proteins in your blood).
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Primary Findings:
Effectiveness:
Safety:
This study was conducted between August 2018 and February 2019. The study enrolled 40 participants.
PTI-428 was generally well tolerated at both dose levels. Although PTI-428 did not have a significant impact on lung function in participants already taking TEZ/IVA, it did result in an increase in CFTR protein production of approximately 50% as measured by ELISA (a lab test that detects proteins in your blood).
These results have been provided from an NACFC abstract and have not been peer reviewed.
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Citation:
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
74 days -
Number of Study Visits:
10
Additional Information
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Phase: ?more info
Phase Two -
Study Sponsor: ?more info
Proteostasis -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Subjects must be currently receiving stable background treatment with tezacaftor/ivacaftor for a minimum of 1 month.

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