A study looking at the safety, tolerability and efficacy of the study drug GLPG2222 in patients with cystic fibrosis who have the F508del CFTR mutation on both alleles
- Conditions
- Cystic FibrosisMedDRA version: 19.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Body processes [G] - Genetic Phenomena [G05]
- Registration Number
- EUCTR2016-004477-40-ES
- Lead Sponsor
- Galapagos NV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 50
• 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
• 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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.<br>To assess changes in respiratory symptoms.<br>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 Outcome Measures
Name Time Method Secondary end point(s): • Change from baseline in sweat chloride concentration <br>• Change from baseline in percent predicted FEV1 <br>• Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R)<br>• 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<br>• PK parameters of GLPG2222 at various time points throughout the trial as specified in the protocol