Study to assess the safety, tolerability & PK of PTI-808/801/428 in CF

• Research type

  Research Study

• Full title

  A Phase 1/2, Randomized, Double-Blind, Placebo-Controlled Study Designed to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects with Cystic Fibrosis

• IRAS ID

  237404

• Contact name

  Geoffrey Gilmartin

• Contact email

  geoffrey.gilmartin@proteostasis.com

• Sponsor organisation

  Proteostasis Therapeutics Inc

• Eudract number

  2017-004229-33

• Duration of Study in the UK

  0 years, 6 months, 0 days

• Research summary

  This is a multicenter study to be performed in approximately 32 Cystic Fibrosis patients, who are homozygous for the F508del mutation. Subjects will be randomized with 8 subjects per cohort receiving active study drug or placebo at a 3:1 randomization ratio, where safety, tolerability and blood levels of PTI-428, PTI-808 & PTI-801 will be assessed. Individual participation in the trial will be up to 49 days and will be mainly performed as an outpatient study with day visits to the clinical trial unit.

• REC name

  HSC REC A

• REC reference

  17/NI/0230

• Date of REC Opinion

  4 Dec 2017

• REC opinion

  Favourable Opinion