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Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation) (SAPHIRA1)

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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02707562
Recruitment Status : Completed
First Posted : March 14, 2016
Last Update Posted : December 7, 2016
Sponsor:
Information provided by (Responsible Party):
Galapagos NV

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.


Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: GLPG1837 dose 1 Drug: GLPG1837 dose 2 Drug: GLPG1837 dose 3 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase IIa, Open-label Study of Multiple Doses of GLPG1837 in Subjects With Cystic Fibrosis and the G551D Mutation
Study Start Date : February 2016
Actual Primary Completion Date : November 2016
Actual Study Completion Date : November 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks
Drug: GLPG1837 dose 1
two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

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

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




Primary Outcome Measures :
  1. Changes in adverse events [ Time Frame: Up to 9 weeks ]
    To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit

  2. Changes in laboratory parameters [ Time Frame: Up to 7 weeks ]
    To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit

  3. Changes in vital signs - composite outcome measure [ Time Frame: Up to 9 weeks ]
    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

  4. Changes in physical examination - composite outcome measure [ Time Frame: Up to 9 weeks ]
    To evaluate the safety and tolerability of GLPG1837 in terms of abnormalities during physical examination at every visit

  5. Changes in electrocardiogram [ Time Frame: Up to 7 weeks ]
    To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit


Secondary Outcome Measures :
  1. Changes in sweat chloride concentration [ Time Frame: Up to 9 weeks ]
    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

  2. Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry [ Time Frame: Up to 9 weeks ]
    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

  3. Plasma levels of GLPG1837: Cmax, the maximum observed plasma concentration [ Time Frame: Up to 3 weeks ]
    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

  4. Plasma levels of GLPG1837: tmax, the time of occurrence of Cmax [ Time Frame: Up to 3 weeks ]
    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

  5. Plasma levels of GLPG1837: AUC, the area under the plasma concentration-time curve [ Time Frame: Up to 3 weeks ]
    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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02707562


Locations
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Australia
Royal Adelaide Hospital
Adelaide, Australia
The Prince Charles Hospital
Chermside, Australia
Monash Medical Centre
Clayton, Australia
Sir Charles Gairdner Hospital
Nedlands, Australia
Mater Adult Hospital
South Brisbane, Australia
Czech Republic
Fakultni nemocnice v Motole
Praha 5, Czech Republic
Germany
Charité Universitätsmedizin Berlin
Berlin, Germany
Universitätsklinkikum Koeln
Cologne, Germany
Uniklinik Carl-Gustav-Carus
Dresden, Germany
Lungenheilkunde München-Pasing
München, Germany
Ireland
Beamont Hospital
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
United Kingdom
Queen Elizabeth University Hospital
Glasgow, United Kingdom
Liverpool Heart and Chest Hospital
Liverpool, United Kingdom
Royal Brompton Hospital
London, United Kingdom
The Medicines Evaluation Unit Ltd
Manchester, United Kingdom
Sponsors and Collaborators
Galapagos NV
Investigators
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Study Director: Olivier Van de Steen, MD, MBA Galapagos NV
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Galapagos NV
ClinicalTrials.gov Identifier: NCT02707562    
Other Study ID Numbers: GLPG1837-CL-201
2015-003291-77 ( EudraCT Number )
First Posted: March 14, 2016    Key Record Dates
Last Update Posted: December 7, 2016
Last Verified: March 2016
Keywords provided by Galapagos NV:
Cystic fibrosis G551D mutation
GLPG1837
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases