GLPG1837-CL-201 in subjects with CF and G551D mutation
Research type
Research Study
Full title
A phase IIa, open-label study of multiple doses of GLPG1837 in subjects with cystic fibrosis and the G551D mutation.
IRAS ID
193346
Contact name
Gordon MacGregor
Contact email
Sponsor organisation
Galapagos NV
Eudract number
2015-003291-77
Duration of Study in the UK
0 years, 7 months, 1 days
Research summary
This is a Phase IIa, open-label, multi-centre, ascending-dose study to evaluate multiple oral doses of GLPG1837 in subjects with CF and the G551D mutation.
CF is a recessive genetic disease caused by mutations in the gene encoding for the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein. Overall, the broad spectrum of mutations can be grouped into six classes based on their effect on CFTR expression or function. G551D is the most common mutation among class III or ‘gating’ mutations accounting for ~4% of total CF patient population, leading to a CFTR protein that is correctly positioned on the cell surface but fails to open in response to intracellular signals (no function).
At least 12 subjects will be included in to the study for a maximum of 8-9 weeks. All patients will be at least 18 years of age and have cystic fibrosis with gating G551D CFTR mutation to take part in this study. The study consists of 3 periods; screening, treatment and follow-up.
The following countries are taking part in this study:
Australia, Czech Republic, Germany and the UK.
Enrollment timelines: March 2016 to August 2016.REC name
East of England - Essex Research Ethics Committee
REC reference
15/EE/0451
Date of REC Opinion
2 Feb 2016
REC opinion
Further Information Favourable Opinion