Back to resultsA Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
GLPG2222 150 mg q.d.
GLPG2222 300 mg q.d.
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
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.
Sites / Locations
- The Prince Charles Hospital
- The Alfred
- Sir Charles Gairdner Hospital
- Westmead Hospital
- UZ Brussel
- UZ Gent
- UZ Leuven
- Fakultni nemocnice v Motole
- Universitaetsklinikum Carl Gustav Carus TU Dresden
- Universitätsklinikum Erlangen
- University Children´s Hospital
- Cork University Hospital
- Beaumont Hospital
- St Vincents University Hospital
- Birmingham Heartlands
- Royal Devon and Exeter
- St James's University
- Liverpool Heart and Chest Hospital
- Royal Brompton Hospital
- University Hospital of South Manchester
- Royal Victoria Infirmary
- Southampton General Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
GLPG2222 Dose 1
GLPG2222 Dose 2
Placebo
Arm Description
Outcomes
Primary Outcome Measures
Changes in adverse events
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of adverse events
Changes in abnormal laboratory
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of laboratory
Changes in abnormal vital signs, ECG or physical examination
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of vital signs, ECG or physical examination
Secondary Outcome Measures
Change from baseline of Sweat chloride concentration
Change from baseline of FEV1 (L) and percent predicted FEV1 for age, gender and height as assessed by spirometry
Change from baseline on the respiratory domain of Revised Cystic Fibrosis Questionnaire (CFQ-R)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03045523
Brief Title
A Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2017
Overall Recruitment Status
Completed
Study Start Date
January 2017 (undefined)
Primary Completion Date
August 11, 2017 (Actual)
Study Completion Date
August 11, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Galapagos NV
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
37 (Actual)
8. Arms, Groups, and Interventions
Arm Title
GLPG2222 Dose 1
Arm Type
Experimental
Arm Title
GLPG2222 Dose 2
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
GLPG2222 150 mg q.d.
Intervention Description
GLPG2222 150 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
Intervention Type
Drug
Intervention Name(s)
GLPG2222 300 mg q.d.
Intervention Description
GLPG2222 300 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
Primary Outcome Measure Information:
Title
Changes in adverse events
Description
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of adverse events
Time Frame
at screening and at each study visit up to day 43 which is the final FU visit
Title
Changes in abnormal laboratory
Description
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of laboratory
Time Frame
at screening and at each study visit up to day 43 which is the final FU visit
Title
Changes in abnormal vital signs, ECG or physical examination
Description
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of 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
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Olivier Van Steen, MD, MBA
Organizational Affiliation
Galapagos NV
Official's Role
Study Director
Facility Information:
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
The Alfred
City
Melbourne
Country
Australia
Facility Name
Sir Charles Gairdner Hospital
City
Nedlands
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
Country
Australia
Facility Name
UZ Brussel
City
Brussels
Country
Belgium
Facility Name
UZ Gent
City
Ghent
Country
Belgium
Facility Name
UZ Leuven
City
Leuven
Country
Belgium
Facility Name
Fakultni nemocnice v Motole
City
Praha
Country
Czechia
Facility Name
Universitaetsklinikum Carl Gustav Carus TU Dresden
City
Dresden
Country
Germany
Facility Name
Universitätsklinikum Erlangen
City
Erlangen
Country
Germany
Facility Name
University Children´s Hospital
City
Tübingen
Country
Germany
Facility Name
Cork University Hospital
City
Cork
Country
Ireland
Facility Name
Beaumont Hospital
City
Dublin
Country
Ireland
Facility Name
St Vincents University Hospital
City
Dublin
Country
Ireland
Facility Name
Birmingham Heartlands
City
Birmingham
Country
United Kingdom
Facility Name
Royal Devon and Exeter
City
Exeter
Country
United Kingdom
Facility Name
St James's University
City
Leeds
Country
United Kingdom
Facility Name
Liverpool Heart and Chest Hospital
City
Liverpool
Country
United Kingdom
Facility Name
Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
University Hospital of South Manchester
City
Manchester
Country
United Kingdom
Facility Name
Royal Victoria Infirmary
City
Newcastle
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
33331662
Citation
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived
PubMed Identifier
31056441
Citation
Bell SC, Barry PJ, De Boeck K, Drevinek P, Elborn JS, Plant BJ, Minic P, Van Braeckel E, Verhulst S, Muller K, Kanters D, Bellaire S, de Kock H, Geller DE, Conrath K, Van de Steen O, van der Ent K. CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials. J Cyst Fibros. 2019 Sep;18(5):700-707. doi: 10.1016/j.jcf.2019.04.014. Epub 2019 May 3.
Results Reference
derived
Learn more about this trial
A Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis
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