Phase IIB study of GS-5745 in participants with Cystic Fibrosis
Research type
Research Study
Full title
A Phase 2b, Dose-Ranging Study of the Effect of GS-5745 on FEV1 in Adult Subjects with Cystic Fibrosis
IRAS ID
201131
Contact name
Mary Carroll
Contact email
Sponsor organisation
Gilead Sciences, Inc.
Eudract number
2015-002192-23
Clinicaltrials.gov Identifier
120100, IND number
Duration of Study in the UK
2 years, 8 months, 16 days
Research summary
Cystic fibrosis (CF) affects approximately 100,000 people worldwide. CF is the most common life-shortening genetic disorder in Caucasians, with an average age of death of 27.5 years in the US and 28.0 years in the EU. Despite many advances in CF therapies, the majority of CF patients experience progressive loss of lung function and recurrent exacerbations. \n\nCompared with healthy individuals, patients with CF have very high concentrations of a substance called Matrix Metalloproteinase 9 (MMP9) in their sputum (coughed up material from the lower airways) which has been associated with reduced lung function and exacerbations in CF patients. GS-5745 is an experimental medication developed by Gilead Sciences, Inc. to block the function of MMP9. Therefore treatment of CF patients with GS-5745 may reduce airway MMP9 activity and result in reduced airway inflammation and improved lung function. \n\nThis clinical study will provide information about the benefits GS-5745 may have in the CF population and potentially provide an additional therapy to improve the health and life-expectancy of patients living with this life-limiting condition. The main objective of this study is to see how 8 weeks of treatment with GS-5745 changes the amount of air participants can forcefully blow out of their lungs in one second, before bronchodilator use. \n\nIt is expected that 150 adult CF participants will be recruited onto this study globally with approximately 12 participants in the UK. Throughout the study, participants will continue their stable, routine CF approved study medications. In Part 1 and 2, the study will test different doses (600mg, 300mg and 150mg) of GS-5745 given by weekly injections, against placebo control groups, for 8 weeks. Participants will then have the option to take part in an open-label extension treatment period, where they will receive weekly GS-5745 injections, for 16 weeks. \n
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
16/EE/0216
Date of REC Opinion
11 Jul 2016
REC opinion
Further Information Favourable Opinion