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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Registration Number: EUCTR2016-002837-31-DE

Lead Sponsor: Galapagos NV

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 35

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety and tolerability of two doses of orally administered GLPG2222 in ivacaftor-treated adult subjects with CF harbouring one F508del CFTR mutation and a second gating (class III) mutation;Secondary Objective: •To assess changes in sweat chloride as biomarker of CFTR ion channel function<br>•To assess changes in pulmonary function (FEV1)<br>•To assess changes in Cystic Fibrosis Questionnaire – Revised (CFQ-R)<br><br><br>;Primary end point(s): Safety and tolerability will be assessed through:<br>Adverse events (AEs)<br>Oxygen saturation by pulse oximetry<br>Physical examinations<br>Vital signs <br>12-lead ECG<br>Spirometry<br>Safety laboratory assessments ;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): Efficacy will be assessed through:<br>sweat Chloride Concentration Testing<br>Spirometry<br>Cystic Fibrosis Questionnaire – Revised (CFQ-R);Timepoint(s) of evaluation of this end point: Various time points throughout the trial as specified in the protocol

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