Cystic Fibrosis MedDRA version: 19.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: GLPG2222
Product Code: G957389
Pharmaceutical Form: Tablet
INN or Proposed INN: Not applicable
Current Sponsor code: G9573
Sponsors
Galapagos NV
Eligibility
Sex/Gender
All
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 postbronchodilator). Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 49 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
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 < 60 mL/minute using Cockcroft-Gault equation at screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of 4 different doses of GLPG2222 administered orally and q.d. for 29 days in adult subjects with CF who are homozygous for the F508del CFTR mutation.; Secondary Objective: To assess changes in biomarkers of CFTR activity. To assess changes in respiratory symptoms. To assess the pharmacokinetics (PK) of GLPG2222. ;Primary end point(s): 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;Timepoint(s) of evaluation of this end point: Various time points throughout the trial as specified in the protocol | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • 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 ; Timepoint(s) of evaluation of this end point: • Sweat chloride concentration, percent predicted FEV1 and the respiratory domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at day 29 • PK parameters of GLPG2222 at various time points throughout the trial as specified in the protocol | — |
Countries
Belgium, Netherlands, Serbia, Spain, United Kingdom, United States
Contacts
Public ContactClinical trial information desk
Galapagos NV
Outcome results
None listed