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

Phase 2CompletedNCT03119649

Lakefront Biotherapeutics NV59 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Cystic Fibrosis

Interventions

GLPG2222 50 mgDRUG

Oral tablet(s) containing GLPG2222

GLPG2222 100 mgDRUG

Oral tablet(s) containing GLPG2222

PlaceboDRUG

Matching oral tablet(s) containing placebo

GLPG2222 200 mg

Eligibility

Sex: ALLMin age: 18 YearsMax age: 99 Years

Medical Language ↔ Plain English

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.

Locations (23)

Child Health Research Unit at UAB

Chatom, Alabama, United States

University of Arkansas for medical Sciences

Outcomes

Primary Outcomes

Number of Participants With Treatment-Emergent Adverse Events

Number of participants with any treatment-emergent adverse events (TEAEs) and serious or treatment-related TEAEs, as well as number of patients with TEAEs by worst intensity reported (mild, moderate, or severe).

Time frame: First administration (Day 1) through Follow-up (Day 43)

Secondary Outcomes

Mean Change From Baseline in Sweat Chloride Concentration at Day 29

Two sweat collections, one from each arm, were obtained. Mean sweat chloride concentration was determined from both arms and measured as millimoles per liter (mmol/L). Baseline was defined as the predose value on Day 1 (or the last non-missing predose measurement).

Time frame: Prior to dosing on Days 1 and 29, or at early discontinuation

Mean Change From Baseline in Percent (%) Predicted FEV1 (%FEV1) at Day 29

Percent predicted FEV1 for age, gender, and height was determined from standardized spirometry assessments and estimated using the 2012 Global Lungs Initiative equation. Baseline was defined as the last non-missing predose assessment on Day 1.

Time frame: Predose and between 1 and 2 hours postdose on Days 1 and 29, or at early discontinuation

Mean Change From Baseline in the Respiratory Domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at Day 29

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. The respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), derived from Questions 40, 41, 42, 45, and 46 if at least 50% of the questions had non-missing data. The scale score ranged from 0-100; higher scores indicated fewer symptoms and better health-related quality of life with a negative change indicating a worsening of symptoms. A change of 4 is considered clinically relevant.

Time frame: Prior to dosing on Days 1 and 29, or at early discontinuation

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.