A Phase IIa, randomized, double-blind, placebo-controlled study to evaluate multiple doses of GLPG2222 in subjects with Cystic Fibrosis who are homozygous for the F508del mutatio
- Conditions
- mucoviscidosisthick mucus disease10083624
- Registration Number
- NL-OMON45460
- Lead Sponsor
- Galapagos NV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 16
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).
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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety and tolerability, assessed by the incidence of adverse events (AEs), as<br /><br>well as changes over time in weight, vital signs, oxygen<br /><br>saturation by pulse oximetry, 12-lead ECG, spirometry, and clinical safety<br /><br>laboratory data.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Change from baseline in sweat chloride concentration<br /><br>• Change from baseline in percent predicted FEV1<br /><br>• Change from baseline in the respiratory domain of the Cystic Fibrosis<br /><br>Questionnaire-Revised (CFQ-R)<br /><br>• PK parameters of GLPG2222</p><br>