A study looking at the safety and tolerability of the drug GLPG2222 in patients with cystic fibrosis with the F508del CFTR mutation and a second gating (class III) mutatio

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 19.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2016-002837-31-GB
• Lead Sponsor: Galapagos NV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-10-17
• Study Completion: 2017-08-24
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 37

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

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified