A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03119649
• Date of registration: 11/04/2017
• Prospective Registration: No
• Primary sponsor: Galapagos NV
• Public title: A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis
• Scientific title: A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate Multiple Doses of GLPG2222 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation
• Date of first enrolment: March 18, 2017
• Target sample size: 59
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03119649
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Belgium, Netherlands, Serbia, Spain, United Kingdom, United States

Contacts

• Name: Olivier Van Steen, MD, MBA
• Affiliation: Galapagos NV

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female subject = 18 years of age, on the day of signing the Informed Consent
Form (ICF).

2. A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation

3. Weight = 40 kg.

4. Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline

5. Forced expiratory volume in 1 second (FEV1) = 40% of predicted normal for age, gender
and height at screening

Exclusion Criteria:

1. History of clinically meaningful unstable or uncontrolled chronic disease that makes
the subject unsuitable for inclusion in the study in the opinion of the investigator.

2. Unstable pulmonary status or respiratory tract infection requiring a change in therapy
within 4 weeks of baseline.

3. Need for supplemental oxygen during the day, and >2 liters per minute (LPM) while
sleeping.

4. Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to
the first study drug administration.

5. History of hepatic cirrhosis with portal hypertension.

6. Abnormal liver function test at screening; defined as aspartate aminotransferase (AST)
and/or alanine aminotransferase (ALT) and/ or alkaline phosphatase and/or
gamma-glutamyl transferase (GGT) = 3x the upper limit of normal (ULN); and/or total
bilirubin (>1.5 times ULN)

7. Estimated creatinine clearance < 60 mL/min using the Cockcroft-Gault formula at
screening.



Age minimum: 18 Years
Age maximum: 99 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: GLPG2222 50 mg

Drug: GLPG2222 100 mg

Drug: GLPG2222 200 mg

Drug: GLPG2222 400 mg

Primary Outcome(s)

Number of Participants With Treatment-Emergent Adverse Events [Time Frame: First administration (Day 1) through Follow-up (Day 43)]

Secondary Outcome(s)

Median Time to Occurrence of GLPG2222 Cmax (Tmax; Hours [h]) [Time Frame: Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29]

Mean Area Under the Concentration-Time Curve From Time 0 up to 24 Hours Following Multiple Dosing (AUC[0-t]; ng.h/mL) of GLPG2222 [Time Frame: Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29]

Mean Change From Baseline in Sweat Chloride Concentration at Day 29 [Time Frame: Prior to dosing on Days 1 and 29, or at early discontinuation]

Mean Maximum Observed Plasma Concentration (Cmax; Nanograms Per Milliliter [mg/mL]) of GLPG2222 [Time Frame: Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29]

Mean Change From Baseline in Percent (%) Predicted FEV1 (%FEV1) at Day 29 [Time Frame: Predose and between 1 and 2 hours postdose on Days 1 and 29, or at early discontinuation]

Mean Change From Baseline in the Respiratory Domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at Day 29 [Time Frame: Prior to dosing on Days 1 and 29, or at early discontinuation]

Mean GLPG2222 Plasma Concentration Observed at Predose (Ctrough; ng/mL) [Time Frame: Days 15 and 29 (predose)]

Secondary ID(s)

GLPG2222-CL-202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 16/11/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03119649