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

Cystic Fibrosis Clinical Trial

— SAPHIRA2  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02690519
• Other study ID #: GLPG1837-CL-202
• Secondary ID: 2015-003292-30
• Status: Completed
• Phase: Phase 2
• First received: February 16, 2016
• Last updated: October 10, 2016
• Start date: January 2016
• Est. completion date: September 2016

Study information

• Verified date: March 2016
• Source: Galapagos NV
• Contact: n/a
• Is FDA regulated: No
• Health authority: Belgium: Federal Agency for Medicinal Products and Health ProductsNetherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: September 2016
• Est. primary completion date: August 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• GLPG1837 dose 1
one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks
• GLPG1837 dose 2
one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks

Locations

Country	Name	City	State
Belgium	University Hospital Antwerp	Antwerp
Belgium	University Hospital Ghent	Ghent
Belgium	University Hospitals Leuven	Leuven
Netherlands	AMC	Amsterdam
Netherlands	UMC Utrecht	Utrecht

Sponsors (1)

Lead Sponsor	Collaborator
Galapagos NV

Countries where clinical trial is conducted

Belgium,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes in adverse events	To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit	Up to 9 weeks	Yes
Primary	Changes in laboratory parameters	To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit	Up to 7 weeks	Yes
Primary	Changes in vital signs	To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit	Up to 9 weeks	Yes
Primary	Changes in physical examination	To evaluate the safety and tolerability of GLPG1837 in terms of abnormal physical examination at every visit	Up to 9 weeks	Yes
Primary	Changes in electrocardiogram	To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit	Up to 7 weeks	Yes
Secondary	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	Up to 9 weeks	No
Secondary	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	Up to 9 weeks	No
Secondary	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	Up to 4 weeks	No