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

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

Tracking Information

• First Submitted Date: February 16, 2016
• First Posted Date: February 24, 2016
• Last Update Posted Date: October 11, 2016
• Study Start Date: January 2016
• Actual Primary Completion Date: August 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 19, 2016)

• 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
• 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
• 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
• 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
• 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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: February 19, 2016)

• 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
• 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
• 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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Study of GLPG1837 in Subjects With Cystic Fibrosis (S1251N Mutation)
• Official Title: A Phase IIa, Open-label Study of Two Doses of GLPG1837 in Subjects With Cystic Fibrosis and the S1251N Mutation

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Cystic Fibrosis

Intervention

• 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

Study Arms

Experimental: GLPG1837 dose 1 and GLPG1837 dose 2

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

Interventions:

• Drug: GLPG1837 dose 1
• Drug: GLPG1837 dose 2

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 10, 2016): 7
• Original Estimated Enrollment (submitted: February 19, 2016): 6
• Actual Study Completion Date: September 2016
• Actual Primary Completion Date: August 2016 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Netherlands

Administrative Information

• NCT Number: NCT02690519
• Other Study ID Numbers: GLPG1837-CL-202; 2015-003292-30 ( EudraCT Number )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Galapagos NV
• Original Responsible Party: Same as current
• Current Study Sponsor: Galapagos NV
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Olivier Van de Steen, MD, MBA; Galapagos NV

• PRS Account: Galapagos NV
• Verification Date: March 2016