A study looking at the safety and tolerability of the drug GLPG2222 in patients with cystic fibrosis with the F508del CFTR mutation and a second gating (class III) mutation

• Register: EUCTR
• Last refreshed on: 28 February 2019
• Main ID: EUCTR2016-002837-31-GB
• Date of registration: 17/10/2016
• Prospective Registration: Yes
• Primary sponsor: Galapagos NV
• Public title: A study looking at the safety and tolerability of the drug GLPG2222 in patients with cystic fibrosis with the F508del CFTR mutation and a second gating (class III) mutation
• Scientific 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
• Date of first enrolment: 05/12/2016
• Target sample size: 35
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002837-31
• 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: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Belgium, Czech Republic, Germany, Ireland, United Kingdom

Contacts

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

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

Key inclusion & exclusion criteria

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.
3.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).
4.Weight = 40 kg.
5.Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline (including physician prescribed ivacaftor (Kalydeco®) 150 mg b.i.d.).
6.Forced expiratory volume in 1 second (FEV1) = 40% of predicted normal for age, gender and height at screening (pre- or post-bronchodilator).

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 34
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

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 (including rhinosinusitis) 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.History of hepatic cirrhosis with portal hypertension (e.g., signs/symptoms of splenomegaly, esophageal varices, etc).
5.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).
6.Estimated creatinine clearance < 60mL/min using the Cockroft-Gault formula at screening.



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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis
MedDRA version: 19.0 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: Oral suspension
INN or Proposed INN: Not Applicable
Current Sponsor code: G957389
Other descriptive name: GLPG2222
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use

Product Name: GLPG2222
Product Code: G957389
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Not Applicable
Current Sponsor code: G957389
Other descriptive name: GLPG2222
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral suspension
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

Main Objective: To evaluate the safety and tolerability of two doses of orally administered GLPG2222 in ivacaftor-treated adult subjects with CF harbouring one F508del CFTR mutation and a second gating (class III) mutation

Primary end point(s): Safety and tolerability will be assessed through:
Adverse events (AEs)
Oxygen saturation by pulse oximetry
Physical examinations
Vital signs
12-lead ECG
Spirometry
Safety laboratory assessments

Secondary Objective: •To assess changes in sweat chloride as biomarker of CFTR ion channel function
•To assess changes in pulmonary function (FEV1)
•To assess changes in Cystic Fibrosis Questionnaire – Revised (CFQ-R)

Secondary Outcome(s)

Secondary end point(s): Efficacy will be assessed through:
sweat Chloride Concentration Testing
Spirometry
Cystic Fibrosis Questionnaire – Revised (CFQ-R)

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

Secondary ID(s)

2016-002837-31-IE

GLPG2222-CL-201

Source(s) of Monetary Support

Galapagos NV

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 26/08/2018
• Date Completed: 24/08/2017
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-002837-31/results