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

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 19.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2016-004477-40-BE
• Lead Sponsor: Galapagos NV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-02-21
• Study Completion: 2017-10-19
• Last Update Posted: 5 January 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 59

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

• 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