mucoviscidosis thick mucus disease
Conditions
Interventions
Treatment with the study drug GLPG2222/placebo:
Cohort A: 50 mg GLPG2222, 100 mg GLPG2222 or placebo q.d. for 29 days.
Cohort B: 200 mg GLPG2222, 400 mg GLPG2222 or placebo q.d. for 29 days.
Cystic Fibrosis
Safety
Tolerability
Sponsors
Galapagos NV
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: Male or female subject >= 18 years of age on the day of signing the ICF. • A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation (documented in the subject's medical record or CF registry). • Weight >= 40 kg during the screening period. • Stable concomitant medication regimen for at least 4 weeks prior to the first study drug administration and continuing the same regimen for the duration of the study. • FEV1 >= 40% of predicted normal for age, gender and height at screening (pre- or ostbronchodilator).
Exclusion criteria
Exclusion criteria: History of clinically meaningful unstable or uncontrolled chronic disease that makes the subject unsuitable for inclusion in the study in the opinion of the investigator. • Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks prior to the first study drug administration. • Need for supplemental oxygen during the day, and > 2 L/minute while sleeping. • History of hepatic cirrhosis with portal hypertension (e.g. signs/symptoms of splenomegaly, esophageal varices, etc.). • Concomitant use of any strong inhibitor(s) or inducer(s) of CYP3A4 within 4 weeks prior to the first study drug administration. • Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to the first study drug administration. • Concomitant use of CYP2C8 substrates within 4 weeks prior the first study drug administration. • Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or ALT and/or alkaline phosphatase and/or gamma-glutamyl transferase (GGT) >= 3 x the upper limit of normal (ULN); and/or total bilirubin >= 1.5 x the ULN. • Estimated creatinine clearance
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability, assessed by the incidence of adverse events (AEs), as well as changes over time in weight, vital signs, oxygen saturation by pulse oximetry, 12-lead ECG, spirometry, and clinical safety laboratory data. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in sweat chloride concentration • Change from baseline in percent predicted FEV1 • Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) • PK parameters of GLPG2222 | — |
Countries
The Netherlands
Outcome results
None listed