A Phase I study to evaluate PTI-808 in Adults with Cystic Fibrosis

• Research type

  Research Study

• Full title

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

• IRAS ID

  244849

• Contact name

  Damian Downey

• Contact email

  Damian.Downey@belfasttrust.hscni.net

• Sponsor organisation

  Proteostasis Therapeutics Inc.

• Eudract number

  2017-003319-21

• Duration of Study in the UK

  1 years, 0 months, 30 days

• Research summary

  The purpose of the study is to gain information on the safety of an experimental drug called PTI-808 when used to treat Cystic Fibrosis. Cystic fibrosis is a disease that changes how the body creates mucus and sweat, it affects how well the lungs and digestive system work. The experimental drug PTI-808 will be given alone and in combination with two other experimental drugs, PTI-801 and PTI-428 or a placebo only will be administered. These drugs are being developed to treat the underlying cause of cystic fibrosis.
  This is a Phase I study and it is planned that at least 45 patients aged 18 or older will be receiving either study drug [7 days of just PTI-808 followed by 14 days of PTI-808 + PTI-801 + PTI-428] or placebo. The study drug is capsules that will be taken orally once a day. Participants will be in the study for approximately 57 days and there will be up to 10 site visits.

  Participants will be randomly assigned to receive either the study drug or placebo. There is a 4 in 5 (80% chance) of being assigned study drug. This will make sure neither the participant or the study doctor will know if the study drug or placebo is being administered.
  During the study participants will need to undergo a physical examination and have blood, urine, sweat chloride testing, nasal sampling and lung function tests.

  Participants may or may not receive direct medical benefit from being in this study. It is hoped that this study treatment may provide relief of, or lessening of, the signs and symptoms of cystic fibrosis; however, such a benefit cannot be guaranteed. The possible benefit to other people with this condition includes learning more information about treatment in patients diagnosed with cystic fibrosis.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  18/NE/0146

• Date of REC Opinion

  4 Jun 2018

• REC opinion

  Further Information Favourable Opinion