A study to assess the safety, tolerability and PK of PTI-428 in CF.
Research type
Research Study
Full title
A Phase 1, Randomised, Placebo-Controlled Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis.
IRAS ID
228243
Contact name
Geoffrey Gilmartin
Contact email
Sponsor organisation
Proteostasis Therapeutics Inc
Eudract number
2017-001627-50
Duration of Study in the UK
0 years, 5 months, 30 days
Research summary
This is a multi-center, study to be performed in Cystic fibrosis patients who are currently on stable background treatment with KALYDECO®. 16 enrolled patients will concurrently administer PTI-428 or placebo for 14 consecutive days during which the safety, tolerability and blood levels of PTI-428 and KALYDECO® will be assessed. individual participation time in this study will be up to 59 days, and will be mainly performed as an outpatient study with day visits to the Clinical Trials Units.
REC name
HSC REC B
REC reference
17/NI/0086
Date of REC Opinion
1 Jun 2017
REC opinion
Further Information Favourable Opinion