- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02690519
Study of GLPG1837 in Subjects With Cystic Fibrosis (S1251N Mutation) (SAPHIRA2)
A Phase IIa, Open-label Study of Two Doses of GLPG1837 in Subjects With Cystic Fibrosis and the S1251N Mutation
At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks 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.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Antwerp, Belgium
- University Hospital Antwerp
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Ghent, Belgium
- University Hospital Ghent
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Leuven, Belgium
- University Hospitals Leuven
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Amsterdam, Netherlands
- AMC
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Utrecht, Netherlands
- UMC Utrecht
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Male or female subjects ≥ 18 years of age, with a confirmed diagnosis of cystic fibrosis
- Subjects with gating S1251N 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
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 half-life
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: GLPG1837 dose 1 and GLPG1837 dose 2
GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks
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one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks
one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in adverse events
Time Frame: Up to 9 weeks
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To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit
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Up to 9 weeks
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Changes in laboratory parameters
Time Frame: Up to 7 weeks
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To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit
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Up to 7 weeks
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Changes in electrocardiogram
Time Frame: Up to 7 weeks
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To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit
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Up to 7 weeks
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Changes in vital signs
Time Frame: Up to 9 weeks
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To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit
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Up to 9 weeks
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Changes in physical examination
Time Frame: Up to 9 weeks
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To evaluate the safety and tolerability of GLPG1837 in terms of abnormal physical examination at every visit
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Up to 9 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in sweat chloride concentration
Time Frame: Up to 9 weeks
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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
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Up to 9 weeks
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Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry
Time Frame: Up to 9 weeks
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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
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Up to 9 weeks
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Plasma levels of GLPG1837
Time Frame: Up to 4 weeks
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To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 1 and Day 29 at every visit
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Up to 4 weeks
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Olivier Van de Steen, MD, MBA, Galapagos NV
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- GLPG1837-CL-202
- 2015-003292-30 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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