Cystic fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: GLPG2737
Product Code: G1117337
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Not applicable
Current Sponsor code: G1117337
Other descriptive name: GLPG2737
Concentration unit:
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. • Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study. • FEV1 =40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator). • Sweat chloride concentration =60 mmol/L at screening. Reference is made to the protocol for a complete overview of the inclusion criteria. 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 17 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 serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug. • 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. • History of hepatic cirrhosis with portal hypertension (e.g., signs/symptoms of splenomegaly, esophageal varices, etc.). • Abnormal liver function test at screening, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gammaglutamyl transferase (GGT) =3 x the upper limit of normal (ULN), and/or total bilirubin =1.5 x the ULN at screening. Reference is made to the protocol for a complete overview of the exclusion criteria.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess changes in sweat chloride concentration as a biomarker of CFTR ion channel function after administration of repeated doses of GLPG2737 compared to placebo in Orkambi-treated adult subjects with CF homozygous for the F508del mutation on Day 28;Secondary Objective: - To evaluate safety and tolerability of GLPG2737 - To assess changes in sweat chloride concentration - To assess changes in pulmonary function - To assess changes in respiratory symptoms - To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor;Primary end point(s): Change from baseline (pre-dose on Day 1) in sweat chloride concentration on Day 28 compared to placebo.;Timepoint(s) of evaluation of this end point: Day 28 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Safety and tolerability, assessed by the number of subjects with adverse events (AEs) - Change from baseline (pre-dose on Day 1) in sweat chloride concentration through 28 days. - Change from baseline (pre-dose on Day 1) in percent predicted forced expiratory volume in 1 second (FEV1) through 28 days. - Change from baseline (pre-dose on Day 1) in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R) on Day 28. - PK parameters (including Cmax, AUCt on Day 14 and Ctrough through 28 days) of GLPG2737, its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.;Timepoint(s) of evaluation of this end point: Various timepoints during the trial as specified in the protocol | — |
Countries
Germany
Contacts
Public ContactClinical Trial Information Desk
Galapagos NV
Outcome results
None listed