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)

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

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

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)

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

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 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