This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
22
Study Site II
Berlin, Germany
Study Site V
Cologne, Germany
Study Site X
Dresden, Germany
Study Site III
Essen, Germany
Change from baseline in sweat chloride concentration compared to placebo
To assess Change from baseline in sweat chloride concentration compared to placebo.
Time frame: Between day 1 pre-morning dose and Day 28.
Change versus placebo in the proportion of subjects with adverse events.
To assess safety and tolerability by the number and percentage of subjects with adverse events.
Time frame: Between Day 1 and 3 weeks after the last dose.
Change from baseline in sweat chloride concentration.
To assess the change from baseline in sweat chloride concentration.
Time frame: From baseline (pre-morning dose on Day 1) through 28 days.
Change in percent predicted forced expiratory volume in 1 second (FEV1).
To assess the change from baseline in percent predicted forced expiratory volume in 1 second (FEV1).
Time frame: From baseline (pre-morning dose on Day 1) through 28 days.
Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).
To assess the change from baseline in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).
Time frame: From baseline (pre-morning dose on Day 1) through 28 days.
Maximum observed plasma concentration of GLPG2737 (Cmax)
To characterize the PK of GLPG2737 and its active metabolite, ivacaftor, and lumacaftor.
Time frame: Between day 1 pre-dose and day 14.
Area under the plasma concentration-time curve from time zero until 8 hours (AUC0-8h) post-dose calculated by the linear up - logarithmic down trapezoidal rule (on Day 14)
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Study Site IV
Frankfurt, Germany
Study Site I
Heidelberg, Germany
Study Site VI
München, Germany
Study Site IX
Stuttgart, Germany
Study Site VIII
Tübingen, Germany
To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.
Time frame: Between day 1 pre-dose and day 14.
Trough plasma concentration observed at the end of the dosing interval (Ctrough).
To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.
Time frame: Between day 1 pre-dose and day 28.