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

Details

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

Open to recruitment

Participating Centres
Phase
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

124674

Trial type

Medication

Intervention
the name of the treatment or therapy being researched

CFTR Modulators

Last edited date

03 October 2019

CF sponsor

Proteostasis Therapeutics, Inc.

CF sponsor type

Commercial

Who can take part?

Age

18+

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 within 28 days or 5 half-lives, whichever is longer, prior to Study Day 1.
  • 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 28 days of Day 1.

CF centres running this trial

Recruiting

Belfast City Hospital

Address

51 Lisburn Road Belfast BT9 7AB

Recruitment ends

November 2019

Contact

Downey, Damian

Get in touch

Recruiting

Heartlands Hospital

Address

Bordesley Green, Birmingham B9 5SS

Recruitment ends

October 2019

Contact

Ed Nash

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Recruiting

King's College Hospital NHS Foundation Trust

Address

Denmark Hill London Greater London SE5 9RS

Recruitment ends

November 2019

Contact

,

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Recruiting

Royal Devon and Exeter Hospital

Address

Royal Devon and Exeter Hospital,Barrack Road,Exeter,Devon EX2 5DW

Recruitment ends

November 2019

Contact

Withers, Nick

Get in touch
Show all participating centres