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GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis (PELICAN)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03474042
Recruitment Status : Completed
First Posted : March 22, 2018
Last Update Posted : June 11, 2018
Sponsor:
Information provided by (Responsible Party):
Galapagos NV

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: GLPG2737 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del Mutation
Actual Study Start Date : November 29, 2017
Actual Primary Completion Date : April 10, 2018
Actual Study Completion Date : April 10, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: GLPG2737
GLPG2737 will be provided as capsules for oral use.
Drug: GLPG2737
GLPG2737 oral capsules administered twice daily for 28 days on top of Orkambi.

Placebo Comparator: Placebo
Placebo will be provided as capsules for oral use.
Drug: Placebo
Placebo oral capsules administered twice daily for 28 days on top of Orkambi.




Primary Outcome Measures :
  1. Change from baseline in sweat chloride concentration compared to placebo [ Time Frame: Between day 1 pre-morning dose and Day 28. ]
    To assess Change from baseline in sweat chloride concentration compared to placebo.


Secondary Outcome Measures :
  1. Change versus placebo in the proportion of subjects with adverse events. [ Time Frame: 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.

  2. Change from baseline in sweat chloride concentration. [ Time Frame: From baseline (pre-morning dose on Day 1) through 28 days. ]
    To assess the change from baseline in sweat chloride concentration.

  3. Change in percent predicted forced expiratory volume in 1 second (FEV1). [ Time Frame: 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).

  4. Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R). [ Time Frame: 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).

  5. Maximum observed plasma concentration of GLPG2737 (Cmax) [ Time Frame: Between day 1 pre-dose and day 14. ]
    To characterize the PK of GLPG2737 and its active metabolite, ivacaftor, and lumacaftor.

  6. 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) [ Time Frame: Between day 1 pre-dose and day 14. ]
    To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.

  7. Trough plasma concentration observed at the end of the dosing interval (Ctrough). [ Time Frame: Between day 1 pre-dose and day 28. ]
    To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03474042


Locations
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Germany
Study Site II
Berlin, Germany
Study Site X
Dresden, Germany
Study Site III
Essen, Germany
Study Site IV
Frankfurt, Germany
Study Site I
Heidelberg, Germany
Study Site V
Köln, Germany
Study Site VI
München, Germany
Study Site IX
Stuttgart, Germany
Study Site VIII
Tübingen, Germany
Sponsors and Collaborators
Galapagos NV
Investigators
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Study Director: Olivier Van de Steen, MD MBA Galapagos NV

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Galapagos NV
ClinicalTrials.gov Identifier: NCT03474042    
Other Study ID Numbers: GLPG2737-CL-202
2017-002181-42 ( EudraCT Number )
First Posted: March 22, 2018    Key Record Dates
Last Update Posted: June 11, 2018
Last Verified: June 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases