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

Galapagos

Status and phase

Completed

Phase 2

Conditions

Cystic Fibrosis

Treatments

Drug: GLPG1837 dose 1

Drug: GLPG1837 dose 3

Drug: GLPG1837 dose 2

Study type

Interventional

Funder types

Industry

Identifiers

NCT02707562

GLPG1837-CL-201

2015-003291-77 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

26 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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