PTI-808-01 Study

Principal Investigator: 

Damian Downey

Name of the Study:

A Phase ½ Study to Evaluate the Safety, tolerability , and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in adults with Cystic Fibrosis

Short name: PTI-808-01

Why is this important?

Previous studies have shown that altering CF at the cell level (CFTR) can significantly reduce symptoms and improve lung function.

What is the Research question/aim?

This trial is exploring the safety and tolerability of the co-administration of PTI-808 (CFTR potentiator), PTU-808 (CFTR corrector) + PTI-428 (CFTR amplifier) in patients with Cystic Fibrosis with the F508del/F508del in genotype.

This study is placebo controlled, meaning that some participants will receive the study drug, and others will receive a placebo. Both the participants and study team will be blinded to whether the participant is receiving the study drug or placebo.

What the Study involves: 

It involved 10 visits over approximately 2 months

Who can take part in the Study?

Patients with cystic fibrosis, aged over 18 years, with the F508del/F508del genotype

Contact Information:

Principal Investigator:

Dr Damian Downey

BHSCT/QUB

Respiratory Research Office

Ground Floor

Tower Block

Belfast City Hospital

02895043902

damian.downey@beflasttrust.hscni.net

Study Coordinator:

Esther Ramsay

Respiratory Research Office

Ground Floor

Tower Block

Belfast City Hospital

02895043902

esther.ramsay@belfasttrut.hscni.net

‌