NielsjeB schreef op 21 september 2016 19:02:
GLPG1837 IN SUBJECTS WITH CYSTIC FIBROSIS (CF) AND THE S1251N MUTATION: RESULTS FROM A PHASE IIA STUDY (SAPHIRA2)
De Boeck, C.1; Van Braeckel, E.2; van der Ent, C.K.3;
Verhulst, S.4; Weersink, E.J.5; Conrath, K.6; Kanters, D.6;
Namour, F.7; de Kock, H.6; Van de Steen, O.6 1. Department
of Pediatrics, University of Leuven, Leuven, Belgium;
2. Department of Respiratory Medicine, Ghent University
and Hospital, Ghent, Belgium; 3. Cystic Fibrosis Center and
Department of Pediatric Respiratory Medicine, Wilhelmina
Children’s Hospital, University Medical Center Utrecht, Utrecht,
Netherlands; 4. Cystic Fibrosis Center, University Hospital
Antwerp, Antwerpen, Belgium; 5. Department of Respiratory
Medicine, Academic Medical Center, Amsterdam, Belgium;
6. Galapagos NV, Mechelen, Belgium; 7. Galapagos SASU,
Romainville, France
Background: GLPG1837 is a novel cystic fibrosis transmembrane
conductance regulator (CFTR) potentiator in clinical development for the
treatment of cystic fibrosis. GLPG1837 has shown to be safe and well
tolerated after 2 weeks of dosing up to 800 mg orally twice daily in healthy
subjects. In vitro assays on S1251N CFTR showed GLPG1837 to open this
dysfunctional channel with a high potency. Together, these results suggest
GLPG1837 could be beneficial to CF patients harbouring the S1251N
mutation.
Methods and Objectives: SAPHIRA2 is a phase IIa, open-label, multicentre
study designed to evaluate two doses of GLPG1837 (two weeks
of dose 1, followed by two weeks of dose 2). Primary study objective is
to evaluate safety and tolerability. Secondary outcome parameters include
sweat chloride concentration as pharmacodynamic biomarker and pulmonary
function. In addition, we explore the correlation of biomarker change
and GLPG1837 plasma exposure. Main study inclusion criteria are: CF and
S1251N mutation on one allele; age = 18 years; FEV1 = 40% of predicted
normal. For subjects treated with ivacaftor, a 7-day washout prior to start
of first dose of GLPG1837 is required. At least 6 subjects will be included
in the study.
Results: At the time of submission of the abstract, the study is still
ongoing: 7 subjects have been screened, 5 subjects have been enrolled and
4 subjects have completed the study. To date, no unexpected safety signals
have been detected and the study is continuing as planned.
The current
projection is to complete enrolment by end of June 2016. Consequently, we
expect top-line results to become available by September 2016.
Conclusions: Pending results.