GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis

A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del Mutation

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03474042

Acronym

PELICAN

Enrollment

Registered

2018-03-22

Start date

2017-11-29

Completion date

2018-04-10

Last updated

2018-06-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

Brief summary

This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.

Interventions

DRUGGLPG2737

GLPG2737 oral capsules administered twice daily for 28 days on top of Orkambi.

DRUGPlacebo

Placebo oral capsules administered twice daily for 28 days on top of Orkambi.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Male or female subject ≥18 years of age on the day of signing the ICF. * A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation. * Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study. * FEV1 ≥40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator). * Sweat chloride concentration ≥60 mmol/L at screening.

Exclusion criteria

* History of serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug. * 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. * Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks prior to the first study drug administration. * History of hepatic cirrhosis with portal hypertension (e.g.,signs/symptoms of splenomegaly, esophageal varices, etc.). * Abnormal liver function test at screening, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gammaglutamyl transferase (GGT) ≥3 x the upper limit of normal (ULN), and/or total bilirubin ≥1.5 x the ULN at screening.

Design outcomes

Primary

Measure	Time frame	Description
Change from baseline in sweat chloride concentration compared to placebo	Between day 1 pre-morning dose and Day 28.	To assess Change from baseline in sweat chloride concentration compared to placebo.

Secondary

Measure	Time frame	Description
Change from baseline in sweat chloride concentration.	From baseline (pre-morning dose on Day 1) through 28 days.	To assess the change from baseline in sweat chloride concentration.
Change in percent predicted forced expiratory volume in 1 second (FEV1).	From baseline (pre-morning dose on Day 1) through 28 days.	To assess the change from baseline in percent predicted forced expiratory volume in 1 second (FEV1).
Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).	From baseline (pre-morning dose on Day 1) through 28 days.	To assess the change from baseline in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).
Change versus placebo in the proportion of subjects with adverse events.	Between Day 1 and 3 weeks after the last dose.	To assess safety and tolerability by the number and percentage of subjects with adverse events.
Area under the plasma concentration-time curve from time zero until 8 hours (AUC0-8h) post-dose calculated by the linear up - logarithmic down trapezoidal rule (on Day 14)	Between day 1 pre-dose and day 14.	To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.
Trough plasma concentration observed at the end of the dosing interval (Ctrough).	Between day 1 pre-dose and day 28.	To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.
Maximum observed plasma concentration of GLPG2737 (Cmax)	Between day 1 pre-dose and day 14.	To characterize the PK of GLPG2737 and its active metabolite, ivacaftor, and lumacaftor.

Countries

Germany

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 1, 2026