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Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Adults With Cystic Fibrosis


Therapeutic category
the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise

Restore CFTR Function

Trial status

Closed to recruitment - in follow up

Participating Centres
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use

Phase II

Full title

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults With Cystic Fibrosis

In this study, the investigational CFTR modulator medicine PTI-808 will be given in combination with one or two other investigational modulator medicines, PTI-801 and PTI-428. PTI-428, PTI-801, and PTI-808 are considered cystic fibrosis transmembrane conductance regulator (CFTR) modulators being developed to treat the underlying cause of cystic fibrosis. PTI-428 is a CFTR amplifier that increases the amount of CFTR protein in the cell, PTI-801 is a CFTR corrector that improves the processing of CFTR protein in the cell, and PTI-808 is a CFTR potentiator to enhance the activity of CFTR at the cell surface. Modulators on their own have been shown to have limited activity, and it is predicted that combining modulators will be necessary to address CFTR defects. There is a screening visit up to 4 weeks before the active portion of the study, which lasts 4 weeks. In the active portion of the study, participants will take one of the following: PTI-808 by mouth in combination with PTI-801 and PTI-428, PTI-808 in combination with PTI-801 and placebo, or all placebos for 4 weeks. The study medication is taken once a day at breakfast. Two weeks after stopping the study medication, another visit will occur.

Trial Reference Number


Trial type


the name of the treatment or therapy being researched

CFTR Modulators

Last edited date

31 January 2020

CF sponsor

Proteostasis Therapeutics, Inc.

CF sponsor type


Who can take part?



Top inclusion criteria
  • Homozygous for the F508del mutation (have 2 copies of F508del) or who are heterozygous for the F508del mutation (have only 1 copy of F508del)
  • FEV1 between 40-90%
Top exclusion criteria
  • Participation in another clinical trial or treatment with an investigational agent
  • History of organ transplantation.

CF centres running this trial


Belfast City Hospital


51 Lisburn Road Belfast BT9 7AB


Downey, Damian

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Heartlands Hospital


Bordesley Green East Birmingham B9 5SS


Whitehouse, Joanna

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King's College Hospital NHS Foundation Trust


Denmark Hill London Greater London SE5 9RS



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Southampton General Hospital


Tremona Road Southampton Hampshire SO16 6YD


Carroll, Mary

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Show all participating centres