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

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

GLPG1837 dose 1

GLPG1837 dose 2

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic fibrosis S1251N mutation, GLPG1837

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

University Hospital Antwerp
University Hospital Ghent
University Hospitals Leuven
AMC
UMC Utrecht

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

GLPG1837 dose 1 and GLPG1837 dose 2

Arm Description

GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks

Outcomes

Primary Outcome Measures

Changes in adverse events

To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit

Changes in laboratory parameters

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit

Changes in vital signs

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit

Changes in physical examination

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal physical examination at every visit

Changes in electrocardiogram

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit

Secondary Outcome Measures

Changes in sweat chloride concentration

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

Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry

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

Plasma levels of GLPG1837

To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 1 and Day 29 at every visit

Full Information

NCT ID

NCT02690519

First Posted

February 16, 2016

Last Updated

October 10, 2016

Sponsor

Galapagos NV

1. Study Identification

Unique Protocol Identification Number

NCT02690519

Brief Title

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

Acronym

SAPHIRA2

Official Title

A Phase IIa, Open-label Study of Two Doses of GLPG1837 in Subjects With Cystic Fibrosis and the S1251N Mutation

Study Type

Interventional

2. Study Status

Record Verification Date

March 2016

Overall Recruitment Status

Completed

Study Start Date

January 2016 (undefined)

Primary Completion Date

August 2016 (Actual)

Study Completion Date

September 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Galapagos NV

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

Keywords

Cystic fibrosis S1251N mutation, GLPG1837

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

7 (Actual)

8. Arms, Groups, and Interventions

Arm Title

GLPG1837 dose 1 and GLPG1837 dose 2

Arm Type

Experimental

Arm Description

GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks

Intervention Type

Drug

Intervention Name(s)

GLPG1837 dose 1

Intervention Description

one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks

Intervention Type

Drug

Intervention Name(s)

GLPG1837 dose 2

Intervention Description

one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks

Primary Outcome Measure Information:

Title

Changes in adverse events

Description

To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit

Time Frame

Up to 9 weeks

Title

Changes in laboratory parameters

Description

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit

Time Frame

Up to 7 weeks

Title

Changes in vital signs

Description

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit

Time Frame

Up to 9 weeks

Title

Changes in physical examination

Description

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal physical examination at every visit

Time Frame

Up to 9 weeks

Title

Changes in electrocardiogram

Description

To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit

Time Frame

Up to 7 weeks

Secondary Outcome Measure Information:

Title

Changes in sweat chloride concentration

Description

Time Frame

Up to 9 weeks

Title

Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry

Description

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

Time Frame

Up to 9 weeks

Title

Plasma levels of GLPG1837

Description

To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 1 and Day 29 at every visit

Time Frame

Up to 4 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Olivier Van de Steen, MD, MBA

Organizational Affiliation

Galapagos NV

Official's Role

Study Director

Facility Information:

Facility Name

University Hospital Antwerp

City

Antwerp

Country

Belgium

Facility Name

University Hospital Ghent

City

Ghent

Country

Belgium

Facility Name

University Hospitals Leuven

City

Leuven

Country

Belgium

Facility Name

AMC

City

Amsterdam

Country

Netherlands

Facility Name

UMC Utrecht

City

Utrecht

Country

Netherlands

12. IPD Sharing Statement

Learn more about this trial

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

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