A study of FDL169 in subjects with Cystic Fibrosis
Research type
Research Study
Full title
A Randomized, Double-Blind Placebo-Controlled, Parallel Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of FDL169 in Cystic Fibrosis Subjects Homozygous for the F508del-CFTR mutation
IRAS ID
218578
Contact name
Alexander Horsley
Contact email
Sponsor organisation
Flatley Discovery Labs, LLC
Eudract number
2016-004098-42
Duration of Study in the UK
1 years, 4 months, 0 days
Research summary
This is a phase Ib, multicenter, randomised double-blind, placebo-controlled dose-escalation and parallel-arm, dose-ranging study in participants with Cystic Fibrosis (CF).
The purpose of the study is to determine how safe and effective FDL169 is in participants with CF. Cystic fibrosis is an autosomal recessive chronic progressive disorder which affects approximately 70,000 patients worldwide.
FDL169 has not yet been approved by any regulatory authority for marketing in any country. This means that FDL169 is an investigational (experimental) drug that can only be used in a clinical research study.
In this study there will be three different doses of FDL169 or placebo (a tablet that looks identical to FDL169 but contains no FDL169 or other active ingredient) that will be given for 28 days. The three different dose levels are 1200 mg /day, 1800 mg/day and 2400 mg/day.
Participants would be asked to sign a consent form and would be screened to determine their eligibility to take part in the study. Only patients who have CF and meet other inclusion criteria would be eligible for this study.
A total of approximately 24 patients will participate in this study at approximately 14 sites throughout Australia, Germany, Czech Rep and United Kingdom. Approximately 10 participants will be enrolled in the UK.
Some of the assessments that the participants would undergo include:
• Questions about any current or previous illnesses, including details of any medication currently being taking will be asked
• Blood and urine samples will be collected for safety tests
• Spirometry- the measurement of participant’s ability to breathe
It is possible that participants taking part in this study may find no benefit to them, but the information obtained from this study will help in future to improve the treatment of people with Cystic Fibrosis.Flatley Discovery Laboratory is sponsor of the study.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
17/EE/0115
Date of REC Opinion
5 May 2017
REC opinion
Further Information Favourable Opinion