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

NCT02690519 | Completed Phase 2 DMC

• 2 other identifiers: GLPG1837-CL-2022015-003292-30
• Study Type: interventional
• Target: 7
• Locations: 2 countries
• Sites: 5

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.

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosis S1251N mutation; GLPG1837

Outcome Measures

Primary Outcomes (5)

• Changes in adverse events

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

  Up to 9 weeks

• 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

• 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

• 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

• Changes in electrocardiogram

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

  Up to 7 weeks

Secondary Outcomes (3)

• Changes in sweat chloride concentration

  Up to 9 weeks

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

  Up to 9 weeks

• Plasma levels of GLPG1837

  Up to 4 weeks

Study Arms (1)

GLPG1837 dose 1 and GLPG1837 dose 2

EXPERIMENTAL

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

Drug: GLPG1837 dose 1; Drug: GLPG1837 dose 2

Interventions

GLPG1837 dose 1 DRUG

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

GLPG1837 dose 1 and GLPG1837 dose 2

GLPG1837 dose 2 DRUG

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

GLPG1837 dose 1 and GLPG1837 dose 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Galapagos NV: lead

Study Sites (5)

University Hospital Antwerp

Antwerp, Belgium

Location

University Hospital Ghent

Ghent, Belgium

Location

University Hospitals Leuven

Leuven, Belgium

Location

AMC

Amsterdam, Netherlands

Location

UMC Utrecht

Utrecht, Netherlands

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Olivier Van de Steen, MD, MBA

  Galapagos NV

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 16, 2016
• First Posted: February 24, 2016
• Study Start: January 1, 2016
• Primary Completion: August 1, 2016
• Study Completion: September 1, 2016
• Last Updated: October 11, 2016

Record last verified: 2016-03

Locations

BE (3); NL (2)