Skip to main content
OpenTrials
Completed

NCT Number: NCT03045523

A Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis

This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable treatment with ivacaftor.

Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed ivacaftor (Kalydeco®) for at least 28 days at the baseline visit. They will be randomized in a 2:2:1 ratio to receive one of two active doses of GLPG2222 (150 mg q.d. or 300 mg q.d.) or placebo q.d. administered for 29 days. Subjects will be in the study for a minimum of 6 weeks and a maximum of 10 weeks, from screening until the follow-up visit.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Prince Charles Hospital, Chermside, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.
  • 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).
  • Weight ≥ 40 kg.
  • Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline (including physician prescribed ivacaftor (Kalydeco®) 150 mg b.i.d.).
  • Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).

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 (including rhinosinusitis) 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.
  • 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 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).
  • Estimated creatinine clearance < 60mL/min using the Cockroft-Gault formula at screening.

Treatment and study plan

GLPG2222 150 mg q.d.

Drug

GLPG2222 150 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days

GLPG2222 300 mg q.d.

Drug

GLPG2222 300 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days

Placebo

Drug

Placebo administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days

Primary outcomes

  1. Changes in adverse events

    Time frame: at screening and at each study visit up to day 43 which is the final FU visit

    To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of adverse events

  2. Changes in abnormal laboratory

    Time frame: at screening and at each study visit up to day 43 which is the final FU visit

    To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of laboratory

  3. Changes in abnormal vital signs, ECG or physical examination

    Time frame: at screening and at each study visit up to day 43 which is the final FU visit

    To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of vital signs, ECG or physical examination

Secondary outcomes

  1. Change from baseline of Sweat chloride concentration

    Time frame: at screening and at each study visit up to day 43 which is the final FU visit

  2. Change from baseline of FEV1 (L) and percent predicted FEV1 for age, gender and height as assessed by spirometry

    Time frame: at screening and at each study visit up to day 43 which is the final FU visit

  3. Change from baseline on the respiratory domain of Revised Cystic Fibrosis Questionnaire (CFQ-R)

    Time frame: at screening and at each study visit up to day 43 which is the final FU visit

Sponsors and collaborators

Lead sponsor

Lakefront Biotherapeutics NV

Industry

Registry information

Official study title

A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis Harbouring One F508del CFTR Mutation and a Second Gating (Class III) Mutation

Important dates

Study start
2017
Primary completion
2017
Study completion
2017
First posted
Feb 7, 2017
Registry last updated
Nov 21, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.