Cystic Fibrosis MedDRA version: 19.0 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: Oral suspension
INN or Proposed INN: Not Applicable
Current Sponsor code: G957389
Other descriptive name: GLPG2222
Concentration unit:
Sponsors
Galapagos NV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Male or female subject = 18 years of age, on the day of signing the Informed Consent Form (ICF). 2.A confirmed clinical diagnosis of CF. 3.One F508del mutation on one allele in the CFTR gene, a gating (class III) mutation (one of the following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) on the 2nd allele in the CFTR gene (documented in the subject’s medical record or CF registry). 4.Weight = 40 kg. 5.Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline (including physician prescribed ivacaftor (Kalydeco®) 150 mg b.i.d.). 6.Forced expiratory volume in 1 second (FEV1) = 40% of predicted normal for age, gender and height at screening (pre- or post-bronchodilator). 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 34 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1.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. 2.Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks of baseline. 3.Need for supplemental oxygen during the day, and >2 liters per minute (LPM) while sleeping. 4.History of hepatic cirrhosis with portal hypertension (e.g., signs/symptoms of splenomegaly, esophageal varices, etc). 5.Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or total bilirubin (>1.5 times ULN (CTCAE Grade 2) and/or gamma-glutamyl transferase (GGT) = 3x the upper limit of normal (ULN), and/or total bilirubin (>1.5 times ULN (CTCAE Grade 2). 6.Estimated creatinine clearance < 60mL/min using the Cockroft-Gault formula at screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of two doses of orally administered GLPG2222 in ivacaftor-treated adult subjects with CF harbouring one F508del CFTR mutation and a second gating (class III) mutation;Secondary Objective: •To assess changes in sweat chloride as biomarker of CFTR ion channel function •To assess changes in pulmonary function (FEV1) •To assess changes in Cystic Fibrosis Questionnaire – Revised (CFQ-R) ;Primary end point(s): Safety and tolerability will be assessed through: Adverse events (AEs) Oxygen saturation by pulse oximetry Physical examinations Vital signs 12-lead ECG Spirometry Safety laboratory assessments ;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): Efficacy will be assessed through: sweat Chloride Concentration Testing Spirometry Cystic Fibrosis Questionnaire – Revised (CFQ-R);Timepoint(s) of evaluation of this end point: Various time points throughout the trial as specified in the protocol | — |
Countries
Australia, Belgium, Czech Republic, Germany, Ireland, United Kingdom
Contacts
Public ContactClinical trial information desk
Galapagos NV
Outcome results
None listed