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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Timepoint [1] 0 0
Up to 9 weeks
Primary outcome [2] 0 0
Changes in laboratory parameters
Timepoint [2] 0 0
Up to 7 weeks
Primary outcome [3] 0 0
Changes in vital signs - composite outcome measure
Timepoint [3] 0 0
Up to 9 weeks
Primary outcome [4] 0 0
Changes in physical examination - composite outcome measure
Timepoint [4] 0 0
Up to 9 weeks
Primary outcome [5] 0 0
Changes in electrocardiogram
Timepoint [5] 0 0
Up to 7 weeks
Secondary outcome [1] 0 0
Changes in sweat chloride concentration
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
Timepoint [2] 0 0
Up to 9 weeks
Secondary outcome [3] 0 0
Plasma levels of GLPG1837: 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
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
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
Sex
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
NA
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
1/02/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/11/2016
Sample size
Target
Accrual to date
Final
26
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
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT02707562