GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis (PELICAN)

Galapagos

Status and phase

Completed

Phase 2

Conditions

Cystic Fibrosis

Treatments

Drug: GLPG2737

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT03474042

GLPG2737-CL-202

2017-002181-42 (EudraCT Number)

Details and patient eligibility

About

This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.

Enrollment

22 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.
Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study.
FEV1 ≥40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
Sweat chloride concentration ≥60 mmol/L at screening.

Exclusion criteria

History of serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug.
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.
History of hepatic cirrhosis with portal hypertension (e.g.,signs/symptoms of splenomegaly, esophageal varices, etc.).
Abnormal liver function test at screening, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gammaglutamyl transferase (GGT) ≥3 x the upper limit of normal (ULN), and/or total bilirubin ≥1.5 x the ULN at screening.