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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02707562




Registration number
NCT02707562
Ethics application status
Date submitted
22/02/2016
Date registered
14/03/2016
Date last updated
7/12/2016

Titles & IDs
Public title
Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation)
Scientific title
A Phase IIa, Open-label Study of Multiple Doses of GLPG1837 in Subjects With Cystic Fibrosis and the G551D Mutation
Secondary ID [1] 0 0
2015-003291-77
Secondary ID [2] 0 0
GLPG1837-CL-201
Universal Trial Number (UTN)
Trial acronym
SAPHIRA1
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 - GLPG1837 dose 1
Treatment: Drugs - GLPG1837 dose 2
Treatment: Drugs - GLPG1837 dose 3

Experimental: GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3 - GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks


Treatment: Drugs: GLPG1837 dose 1
two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

Treatment: Drugs: GLPG1837 dose 2
two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

Treatment: Drugs: GLPG1837 dose 3
two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for two weeks

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Changes in adverse events - To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit
Timepoint [1] 0 0
Up to 9 weeks
Primary outcome [2] 0 0
Changes in laboratory parameters - To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit
Timepoint [2] 0 0
Up to 7 weeks
Primary outcome [3] 0 0
Changes in vital signs - composite outcome measure - To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs as measured by temperature, blood pressure, heart rate and respiratory rate, at every visit
Timepoint [3] 0 0
Up to 9 weeks
Primary outcome [4] 0 0
Changes in physical examination - composite outcome measure - To evaluate the safety and tolerability of GLPG1837 in terms of abnormalities during physical examination at every visit
Timepoint [4] 0 0
Up to 9 weeks
Primary outcome [5] 0 0
Changes in electrocardiogram - To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit
Timepoint [5] 0 0
Up to 7 weeks
Secondary outcome [1] 0 0
Changes in sweat chloride concentration - To evaluate the effect of GLPG1837 in terms of change in sweat chloride concentration, a biomarker to measure cystic fibrosis transmembrane conductance regulator (CFTR) ion channel function at every visit
Timepoint [1] 0 0
Up to 9 weeks
Secondary outcome [2] 0 0
Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry - To explore the effect of GLPG1837 in terms of change in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry at every visit
Timepoint [2] 0 0
Up to 9 weeks
Secondary outcome [3] 0 0
Plasma levels of GLPG1837: Cmax, the maximum observed plasma concentration - To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 8 and Day 29 at every visit; On Day 29, an 8-hour profile will determine the Cmax, the maximum observed plasma concentration
Timepoint [3] 0 0
Up to 3 weeks
Secondary outcome [4] 0 0
Plasma levels of GLPG1837: tmax, the time of occurrence of Cmax - To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 8 and Day 29 at every visit; On Day 29, an 8-hour profile will determine the tmax, the time of occurrence of Cmax
Timepoint [4] 0 0
Up to 3 weeks
Secondary outcome [5] 0 0
Plasma levels of GLPG1837: AUC, the area under the plasma concentration-time curve - To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 8 and Day 29 at every visit; On Day 29, an 8-hour profile will determine the AUC, the area under the plasma concentration-time curve
Timepoint [5] 0 0
Up to 3 weeks

Eligibility
Key inclusion criteria
- Male or female subjects = 18 years of age, with a confirmed diagnosis of cystic
fibrosis

- Subjects with gating G551D CFTR mutation on at least one allele in the CFTR gene

- Subjects currently receiving treatment with ivacaftor on a stable regimen or not on a
treatment regimen with ivacaftor, for at least 2 weeks prior to screening

- Weight = 40.0 kg

- Subjects on stable concomitant treatment regimen for at least 4 weeks prior to
baseline (excluding ivacaftor)

- Pre- or post-bronchodilator FEV1 = 40% of predicted normal

- Subject will have to use highly effective contraceptive methods
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- On an ivacaftor-containing treatment regimen and unable or unwilling to discontinue
ivacaftor for the washout and treatment periods of the study

- Concomitant use of antifungal drugs within 4 weeks of baseline

- A history of a clinically meaningful unstable or uncontrolled chronic disease

- Liver cirrhosis and portal hypertension

- Any significant change in the medical regimen for pulmonary health within 4 weeks of
baseline

- Unstable pulmonary status or respiratory tract infection or changes in therapy for
pulmonary disease within 4 weeks of baseline

- Abnormal liver function

- Clinically significant abnormalities on ECG

- History of malignancy, solid organ/haematological transplantation

- Abnormal renal function

- Participation in another experimental therapy study within 30 days or 5 times halflife

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [4] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [5] 0 0
Mater Adult Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Chermside
Recruitment postcode(s) [3] 0 0
- Clayton
Recruitment postcode(s) [4] 0 0
- Nedlands
Recruitment postcode(s) [5] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
Czech Republic
State/province [1] 0 0
Praha 5
Country [2] 0 0
Germany
State/province [2] 0 0
Berlin
Country [3] 0 0
Germany
State/province [3] 0 0
Cologne
Country [4] 0 0
Germany
State/province [4] 0 0
Dresden
Country [5] 0 0
Germany
State/province [5] 0 0
München
Country [6] 0 0
Ireland
State/province [6] 0 0
Dublin
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Glasgow
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Liverpool
Country [9] 0 0
United Kingdom
State/province [9] 0 0
London
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Manchester

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

Ethics approval
Ethics application status

Summary
Brief summary
32 cystic fibrosis patients with the G551D mutation will be treated for 4 weeks, consisting
of three consecutive treatment periods: two 1-week periods followed by one 2-week period,
evaluating one dose of GLPG1837 each. After the treatment period, there is a 7-10 days
follow-up period.

During the course of the study, subjects will be examined for any side effects that may occur
(safety and tolerability).

Changes in sweat chloride will be assessed as biomarker from baseline onwards, and changes in
pulmonary function (efficacy) will be explored throughout the study. The amount of GLPG1837
present in the blood (pharmacokinetics) will also be determined.
Trial website
https://clinicaltrials.gov/show/NCT02707562
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Olivier Van de 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
Other publications