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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03045523




Registration number
NCT03045523
Ethics application status
Date submitted
26/01/2017
Date registered
7/02/2017
Date last updated
21/11/2017

Titles & IDs
Public title
A Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis
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
Secondary ID [1] 0 0
GLPG2222-CL-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GLPG2222 150 mg q.d.
Treatment: Drugs - GLPG2222 300 mg q.d.
Treatment: Drugs - Placebo

Experimental: GLPG2222 Dose 1 -

Experimental: GLPG2222 Dose 2 -

Placebo comparator: Placebo -


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

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

Treatment: Drugs: Placebo
Placebo administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Changes in adverse events
Timepoint [1] 0 0
at screening and at each study visit up to day 43 which is the final FU visit
Primary outcome [2] 0 0
Changes in abnormal laboratory
Timepoint [2] 0 0
at screening and at each study visit up to day 43 which is the final FU visit
Primary outcome [3] 0 0
Changes in abnormal vital signs, ECG or physical examination
Timepoint [3] 0 0
at screening and at each study visit up to day 43 which is the final FU visit
Secondary outcome [1] 0 0
Change from baseline of Sweat chloride concentration
Timepoint [1] 0 0
at screening and at each study visit up to day 43 which is the final FU visit
Secondary outcome [2] 0 0
Change from baseline of FEV1 (L) and percent predicted FEV1 for age, gender and height as assessed by spirometry
Timepoint [2] 0 0
at screening and at each study visit up to day 43 which is the final FU visit
Secondary outcome [3] 0 0
Change from baseline on the respiratory domain of Revised Cystic Fibrosis Questionnaire (CFQ-R)
Timepoint [3] 0 0
at screening and at each study visit up to day 43 which is the final FU visit

Eligibility
Key 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 postbronchodilator).
Minimum age
18 Years
Maximum age
99 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
11/08/2017
Sample size
Target
Accrual to date
Final
37
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 0 0
The Alfred - Melbourne
Recruitment hospital [3] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [4] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Chermside
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Belgium
State/province [2] 0 0
Ghent
Country [3] 0 0
Belgium
State/province [3] 0 0
Leuven
Country [4] 0 0
Czechia
State/province [4] 0 0
Praha
Country [5] 0 0
Germany
State/province [5] 0 0
Dresden
Country [6] 0 0
Germany
State/province [6] 0 0
Erlangen
Country [7] 0 0
Germany
State/province [7] 0 0
Tübingen
Country [8] 0 0
Ireland
State/province [8] 0 0
Cork
Country [9] 0 0
Ireland
State/province [9] 0 0
Dublin
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Birmingham
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Exeter
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Leeds
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Liverpool
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Manchester
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Newcastle
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Galapagos NV
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT03045523
Trial related presentations / publications
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Olivier Van Steen, MD, MBA
Address 0 0
Galapagos NV
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03045523