PTI-428-01 - Proteostasis Therapeutics - Cystic Fibrosis
Research type
Research Study
Full title
A Phase I/II, Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis
IRAS ID
210591
Contact name
Jane Davies
Contact email
Sponsor organisation
Proteostasis Therapeutics
Eudract number
2016-001214-24
Duration of Study in the UK
0 years, 8 months, 20 days
Research summary
This is a Phase 1/2, Multi-center,randomised, placebo-controlled designed trial. There are three parts to this research study (parts A, B & C).
Part A will consist of two phases (Single Assending Dose (SAD) and Multiple Ascending Dose (MAD). The SAD cohort of Part A has now completed and for the UK on Part A (MAD) will be open to recruit patients. Up to 44 participants in Part A, 60 participants in Part B and 40 participants in Part C, aged between 18-55, will take part in this study. This is a multicentre study and will take place in different research centres around the world.
Part B will consist of two Phase 2 cohorts. Both cohorts will enrol participants who have been treated with a minimum of 3 months of ORKAMBI® into a combination treatment group with either PTI-428 or placebo at two different doses. These cohorts will run one at a time and will include 20 participants each.
Part C will begin once Part B is completed and will consist of three Phase 2 cohorts. Each cohort will enrol participants at the same time. The first cohort will enrol participants who are eligible for ORKAMBI®, but are not actively treated with it into a combination treatment group of ORKAMBI® with either PTI-428 or placebo. The second cohort will enrol participants who have been treated with a minimum of 3 months of KALYDECO® into a combination treatment group with either PTI-428 or placebo. The third cohort will enrol participants who are not receiving either ORKAMBI® and KALYDECO® and who have residual pancreatic activity into a monotherapy treatment group of either PTI-428 or placebo. Each cohort in Part C will include 20 participants
REC name
London - Central Research Ethics Committee
REC reference
17/LO/0661
Date of REC Opinion
24 Jul 2017
REC opinion
Further Information Favourable Opinion