Phase 2
Completed N=59
A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis
Source: ClinicalTrials.gov NCT03119649 ↗Enrolled (actual)
59
Serious AEs
5.1%
Results posted
Nov 2018
Primary outcomePrimary: Number of Participants With Treatment-Emergent Adverse Events — 9; 8; 10; 11 Participants
Summary
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events |
9; 8; 10; 11; 9; 2 | — |
| SECONDARY Mean Change From Baseline in Sweat Chloride Concentration at Day 29 |
-2.5; -5.8; -6.6; -18.3; -8.8 | 0.4291 |
| SECONDARY Mean Change From Baseline in Percent (%) Predicted FEV1 (%FEV1) at Day 29 |
-1.0; 0.1; -0.3; 0.0; 1.3 | 0.5940 |
| SECONDARY Mean Change From Baseline in the Respiratory Domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at Day 29 |
-2.4; 0.4; -0.7; 4.5; -0.8 | 0.5749 |
| SECONDARY Mean Maximum Observed Plasma Concentration (Cmax; Nanograms Per Milliliter [mg/mL]) of GLPG2222 |
478; 1170; 2490; 5330 | — |
| SECONDARY Mean GLPG2222 Plasma Concentration Observed at Predose (Ctrough; ng/mL) |
48.1; 132; 343; 677 | — |
| SECONDARY Median Time to Occurrence of GLPG2222 Cmax (Tmax; Hours [h]) |
2.0; 2.0; 3.0; 2.0 | — |
| SECONDARY Mean Area Under the Concentration-Time Curve From Time 0 up to 24 Hours Following Multiple Dosing (AUC[0-t]; ng.h/mL) of GLPG2222 |
3850; 9670; 22900; 46400 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female subject ≥ 18 years of age, on the day of signing the Informed Consent Form (ICF).
- A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation
- Weight ≥ 40 kg.
- Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline
- Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted normal for age, gender and height at screening
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 requiring a change in therapy within 4 weeks of baseline.
- Need for supplemental oxygen during the day, and >2 liters per minute (LPM) while sleeping.
- Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to the first study drug administration.
- History of hepatic cirrhosis with portal hypertension.
- 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)
- Estimated creatinine clearance < 60 mL/min using the Cockcroft-Gault formula at screening.
Data sourced from ClinicalTrials.gov (NCT03119649). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.