A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis

Galapagos

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: GLPG2222 200 mg; Drug: GLPG2222 400 mg; Drug: Placebo; Drug: GLPG2222 100 mg; Drug: GLPG2222 50 mg

Study type: Interventional

Funder types: Industry

Identifiers

NCT03119649

GLPG2222-CL-202

Details and patient eligibility

About

This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate 4 different doses of GLPG2222 administered for 4 weeks to adult subjects with a confirmed diagnosis of CF and homozygous for the F508del Cystic Fibrosis Transmembrane conductance Regulator (CFTR) mutation.

Enrollment: 59 patients

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

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 and homozygous for the F508del CFTR mutation
3. Weight ≥ 40 kg.
4. Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline
5. Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted normal for age, gender and height at screening

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 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. Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to the first study drug administration.
5. History of hepatic cirrhosis with portal hypertension.
6. Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/ or alkaline phosphatase and/or gamma-glutamyl transferase (GGT) ≥ 3x the upper limit of normal (ULN); and/or total bilirubin (>1.5 times ULN)
7. Estimated creatinine clearance < 60 mL/min using the Cockcroft-Gault formula at screening.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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59 participants in 6 patient groups, including a placebo group

Cohort A: GLPG2222 50 mg once daily (QD)

Experimental group

Description:

Participants received a single GLPG2222 50 mg tablet and two matching placebo tablets orally, QD for 29 days.

Treatment:

Drug: Placebo

Drug: GLPG2222 50 mg

Cohort A: GLPG2222 100 mg QD

Experimental group

Description:

Participants received a single GLPG2222 100 mg tablet and two matching placebo tablets orally, QD for 29 days.

Treatment:

Drug: GLPG2222 100 mg

Drug: Placebo

Cohort B: GLPG2222 200 mg QD

Experimental group

Description:

Participants received two GLPG2222 100 mg tablets and one matching placebo tablet orally, QD for 29 days.

Treatment:

Drug: GLPG2222 200 mg

Drug: Placebo

Cohort B: GLPG2222 400 mg QD

Experimental group

Description:

Participants received two GLPG2222 150 mg tablets and one GLPG2222 100 mg tablet orally, QD for 29 days.

Treatment:

Drug: GLPG2222 400 mg

Drug: Placebo

Cohort A Placebo

Placebo Comparator group

Description:

Participants received three matching placebo tablets, orally, QD for 29 days.

Treatment:

Drug: Placebo

Cohort B Placebo

Placebo Comparator group

Description:

Participants received three matching placebo tablets, orally, QD for 29 days.

Treatment:

Drug: Placebo