A Phase 1b Study of FDL169 in Healthy Volunteers and CF Patients.
Research type
Research Study
Full title
A Three-Part Phase 1b Bioavailability and Pharmacokinetics (PK) study of Two Formulations of FDL169 in Healthy Volunteers and Patients with Cystic Fibrosis (CF)
IRAS ID
199663
Contact name
Stuart Elborn
Contact email
Sponsor organisation
Flatley Discovery Lab
Eudract number
2016-000045-32
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 1 months, 14 days
Research summary
Cystic fibrosis (CF) is an autosomal recessive chronic progressive disorder with high\nmorbidity and a shortened life expectancy. It affects approximately 70,000 patients\nworldwide. Cystic fibrosis is caused by a mutation in the gene encoding for the\ncystic fibrosis transmembrane conductance regulator (CFTR) protein, a plasma\nmembrane ion channel that mediates transport of chloride, bicarbonate and other\nanions. The CFTR protein is located principally in secretory epithelia in the lungs,\npancreas, gut, testes and exocrine glands. Abnormal ion transport leads to build-up of thick mucus resulting in airway obstruction, chronic lung infection and bronchiectasis with progressive deterioration. \n\nAt present, there is no cure for CF and most of the available therapies treat CF symptoms rather than the underlying genetic defect.\n\nTherapies that restore the function of the abnormal CFTR protein are known as CFTR\nmodulators. FDL169 is an oral small molecule CFTR corrector and is intended for the chronic treatment of CF.\n\nThe proposed study will be conducted in three parts:\n\nPart 1 will investigate the relative bioavailability of two formulations of FDL169.\nPart 2 will evaluate the PK profile of three different doses of FDL169 tablets.\nPart 3 will determine the PK profile of a single 400 mg dose of FDL169 tablets (test\nformulation) in subjects with CF.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
16/SC/0047
Date of REC Opinion
14 Mar 2016
REC opinion
Further Information Favourable Opinion