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
Location:
NI Clinical Research Facility,
U Floor, Belfast City Hospital,
Lisburn Road, Belfast, BT9 7AB
Tel:+44 (0)28 9504 0342
Email:NICRF@qub.ac.uk