Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
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)
Query!
Scientific title
A Phase IIa, Open-label Study of Multiple Doses of GLPG1837 in Subjects With Cystic Fibrosis and the G551D Mutation
Query!
Secondary ID [1]
0
0
2015-003291-77
Query!
Secondary ID [2]
0
0
GLPG1837-CL-201
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
SAPHIRA1
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Cystic fibrosis
Query!
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Oral and Gastrointestinal
0
0
0
0
Query!
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Connective tissue diseases
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
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
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Changes in adverse events
Query!
Assessment method [1]
0
0
To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit
Query!
Timepoint [1]
0
0
Up to 9 weeks
Query!
Primary outcome [2]
0
0
Changes in laboratory parameters
Query!
Assessment method [2]
0
0
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit
Query!
Timepoint [2]
0
0
Up to 7 weeks
Query!
Primary outcome [3]
0
0
Changes in vital signs - composite outcome measure
Query!
Assessment method [3]
0
0
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
Query!
Timepoint [3]
0
0
Up to 9 weeks
Query!
Primary outcome [4]
0
0
Changes in physical examination - composite outcome measure
Query!
Assessment method [4]
0
0
To evaluate the safety and tolerability of GLPG1837 in terms of abnormalities during physical examination at every visit
Query!
Timepoint [4]
0
0
Up to 9 weeks
Query!
Primary outcome [5]
0
0
Changes in electrocardiogram
Query!
Assessment method [5]
0
0
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit
Query!
Timepoint [5]
0
0
Up to 7 weeks
Query!
Secondary outcome [1]
0
0
Changes in sweat chloride concentration
Query!
Assessment method [1]
0
0
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
Query!
Timepoint [1]
0
0
Up to 9 weeks
Query!
Secondary outcome [2]
0
0
Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry
Query!
Assessment method [2]
0
0
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
Query!
Timepoint [2]
0
0
Up to 9 weeks
Query!
Secondary outcome [3]
0
0
Plasma levels of GLPG1837: Cmax, the maximum observed plasma concentration
Query!
Assessment method [3]
0
0
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
Query!
Timepoint [3]
0
0
Up to 3 weeks
Query!
Secondary outcome [4]
0
0
Plasma levels of GLPG1837: tmax, the time of occurrence of Cmax
Query!
Assessment method [4]
0
0
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
Query!
Timepoint [4]
0
0
Up to 3 weeks
Query!
Secondary outcome [5]
0
0
Plasma levels of GLPG1837: AUC, the area under the plasma concentration-time curve
Query!
Assessment method [5]
0
0
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
Query!
Timepoint [5]
0
0
Up to 3 weeks
Query!
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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
NA
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/02/2016
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/11/2016
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
26
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Royal Adelaide Hospital - Adelaide
Query!
Recruitment hospital [2]
0
0
The Prince Charles Hospital - Chermside
Query!
Recruitment hospital [3]
0
0
Monash Medical Centre - Clayton
Query!
Recruitment hospital [4]
0
0
Sir Charles Gairdner Hospital - Nedlands
Query!
Recruitment hospital [5]
0
0
Mater Adult Hospital - South Brisbane
Query!
Recruitment postcode(s) [1]
0
0
- Adelaide
Query!
Recruitment postcode(s) [2]
0
0
- Chermside
Query!
Recruitment postcode(s) [3]
0
0
- Clayton
Query!
Recruitment postcode(s) [4]
0
0
- Nedlands
Query!
Recruitment postcode(s) [5]
0
0
- South Brisbane
Query!
Recruitment outside Australia
Country [1]
0
0
Czech Republic
Query!
State/province [1]
0
0
Praha 5
Query!
Country [2]
0
0
Germany
Query!
State/province [2]
0
0
Berlin
Query!
Country [3]
0
0
Germany
Query!
State/province [3]
0
0
Cologne
Query!
Country [4]
0
0
Germany
Query!
State/province [4]
0
0
Dresden
Query!
Country [5]
0
0
Germany
Query!
State/province [5]
0
0
München
Query!
Country [6]
0
0
Ireland
Query!
State/province [6]
0
0
Dublin
Query!
Country [7]
0
0
United Kingdom
Query!
State/province [7]
0
0
Glasgow
Query!
Country [8]
0
0
United Kingdom
Query!
State/province [8]
0
0
Liverpool
Query!
Country [9]
0
0
United Kingdom
Query!
State/province [9]
0
0
London
Query!
Country [10]
0
0
United Kingdom
Query!
State/province [10]
0
0
Manchester
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Galapagos NV
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT02707562
Query!
Trial related presentations / publications
Davies JC, Van de Steen O, van Koningsbruggen-Rietschel S, Drevinek P, Derichs N, McKone EF, Kanters D, Allamassey L, Namour F, de Kock H, Conrath K. GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1). J Cyst Fibros. 2019 Sep;18(5):693-699. doi: 10.1016/j.jcf.2019.05.006. Epub 2019 May 27.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Olivier Van de Steen, MD, MBA
Query!
Address
0
0
Galapagos NV
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
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
Download to PDF