A study looking at the safety, tolerability and efficacy of the study drug GLPG2222 in patients with cystic fibrosis who have the F508del CFTR mutation on both alleles

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2016-004477-40-GB
• Date of registration: 06/02/2017
• Prospective Registration: Yes
• Primary sponsor: Galapagos NV
• Public title: A study looking at the safety, tolerability and efficacy of the study drug GLPG2222 in patients with cystic fibrosis who have the F508del CFTR mutation on both alleles
• Scientific title: A Phase IIa, randomized, double-blind, placebo-controlled study to evaluate multiple doses of GLPG2222 in subjects with Cystic Fibrosis who are homozygous for the F508del mutation
• Date of first enrolment: 26/05/2017
• Target sample size: 50
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-004477-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 5
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Netherlands, Serbia, Spain, United Kingdom, United States

Contacts

• Name: Clinical trial information desk
• Affiliation: Galapagos NV

• Name: Clinical trial information desk
• Affiliation: Galapagos NV

Key inclusion & exclusion criteria

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 (documented in the subject’s medical record or CF registry).
• Weight = 40 kg during the screening period.
• Stable concomitant medication regimen for at least 4 weeks prior to the first study drug administration and continuing the same regimen for the duration of the study.
• FEV1 = 40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 49
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

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 prior to the first study drug administration.
• Need for supplemental oxygen during the day, and > 2 L/minute while sleeping.
• History of hepatic cirrhosis with portal hypertension (e.g. signs/symptoms of splenomegaly, esophageal varices, etc.).
• Concomitant use of any strong inhibitor(s) or inducer(s) of CYP3A4 within 4 weeks prior to the first study drug administration.
• Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to the first study drug administration.
• Concomitant use of CYP2C8 substrates within 4 weeks prior the first study drug administration.
• Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or ALT and/or alkaline phosphatase and/or gamma-glutamyl transferase (GGT) = 3 x the upper limit of normal (ULN); and/or total bilirubin = 1.5 x the ULN.
• Estimated creatinine clearance < 60 mL/minute using Cockcroft-Gault equation at screening.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic Fibrosis
MedDRA version: 19.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Intervention(s)

Product Name: GLPG2222
Product Code: G957389
Pharmaceutical Form: Tablet
INN or Proposed INN: Not applicable
Current Sponsor code: G957389
Other descriptive name: GLPG2222
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Product Name: GLPG2222
Product Code: G957389
Pharmaceutical Form: Tablet
INN or Proposed INN: Not applicable
Current Sponsor code: G957389
Other descriptive name: GLPG2222
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Product Name: GLPG2222
Product Code: G957389
Pharmaceutical Form: Tablet
INN or Proposed INN: Not applicable
Current Sponsor code: G957389
Other descriptive name: GLPG2222
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Various time points throughout the trial as specified in the protocol

Secondary Objective: To assess changes in biomarkers of CFTR activity.
To assess changes in respiratory symptoms.
To assess the pharmacokinetics (PK) of GLPG2222.

Primary end point(s): Safety and tolerability, assessed by the incidence of adverse events (AEs), as well as changes over time in weight, vital signs, oxygen saturation by pulse oximetry, 12-lead ECG, spirometry, and clinical safety laboratory data

Main Objective: To evaluate the safety and tolerability of 4 different doses of GLPG2222 administered orally and q.d. for 29 days in adult subjects with CF who are homozygous for the F508del CFTR mutation.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: • Sweat chloride concentration, percent predicted FEV1 and the respiratory domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at day 29
• PK parameters of GLPG2222 at various time points throughout the trial as specified in the protocol

Secondary end point(s): • Change from baseline in sweat chloride concentration
• Change from baseline in percent predicted FEV1
• Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
• PK parameters of GLPG2222

Secondary ID(s)

GLPG2222-CL-202

Source(s) of Monetary Support

Galapagos NV

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 04/11/2018
• Date Completed: 19/10/2017
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-004477-40/results