Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study of GLPG1837 in Subjects With Cystic Fibrosis (S1251N Mutation) (SAPHIRA2)

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: NCT02690519
Recruitment Status : Completed
First Posted : February 24, 2016
Last Update Posted : October 11, 2016
Sponsor:
Information provided by (Responsible Party):
Galapagos NV

Brief Summary:

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.


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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase IIa, Open-label Study of Two Doses of GLPG1837 in Subjects With Cystic Fibrosis and the S1251N Mutation
Study Start Date : January 2016
Actual Primary Completion Date : August 2016
Actual Study Completion Date : September 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

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

Drug: GLPG1837 dose 2
one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 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 [ Time Frame: Up to 9 weeks ]
    To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit

  4. Changes in physical examination [ Time Frame: Up to 9 weeks ]
    To evaluate the safety and tolerability of GLPG1837 in terms of abnormal 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 [ Time Frame: Up to 4 weeks ]
    To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 1 and Day 29 at every visit



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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 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

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): NCT02690519


Locations
Layout table for location information
Belgium
University Hospital Antwerp
Antwerp, Belgium
University Hospital Ghent
Ghent, Belgium
University Hospitals Leuven
Leuven, Belgium
Netherlands
AMC
Amsterdam, Netherlands
UMC Utrecht
Utrecht, Netherlands
Sponsors and Collaborators
Galapagos NV
Investigators
Layout table for investigator information
Study Director: Olivier Van de Steen, MD, MBA Galapagos NV

Layout table for additonal information
Responsible Party: Galapagos NV
ClinicalTrials.gov Identifier: NCT02690519     History of Changes
Other Study ID Numbers: GLPG1837-CL-202
2015-003292-30 ( EudraCT Number )
First Posted: February 24, 2016    Key Record Dates
Last Update Posted: October 11, 2016
Last Verified: March 2016
Keywords provided by Galapagos NV:
Cystic fibrosis S1251N mutation
GLPG1837
Additional relevant MeSH terms:
Layout table for MeSH terms
Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases