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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

Tracking Information
First Submitted Date  ICMJE March 15, 2018
First Posted Date  ICMJE March 22, 2018
Last Update Posted Date June 11, 2018
Actual Study Start Date  ICMJE November 29, 2017
Actual Primary Completion Date April 10, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 15, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03474042 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 15, 2018)
  • 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.
  • 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.
  • 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).
  • 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).
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
Official Title  ICMJE A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del Mutation
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: GLPG2737
    GLPG2737 oral capsules administered twice daily for 28 days on top of Orkambi.
  • Drug: Placebo
    Placebo oral capsules administered twice daily for 28 days on top of Orkambi.
Study Arms  ICMJE
  • Experimental: GLPG2737
    GLPG2737 will be provided as capsules for oral use.
    Intervention: Drug: GLPG2737
  • Placebo Comparator: Placebo
    Placebo will be provided as capsules for oral use.
    Intervention: Drug: Placebo
Publications * van Koningsbruggen-Rietschel S, Conrath K, Fischer R, Sutharsan S, Kempa A, Gleiber W, Schwarz C, Hector A, Van Osselaer N, Pano A, Corveleyn S, Bwirire D, Santermans E, Muller K, Bellaire S, Van de Steen O. GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN). J Cyst Fibros. 2019 Oct 5. pii: S1569-1993(19)30890-2. doi: 10.1016/j.jcf.2019.09.006. [Epub ahead of print]

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 15, 2018)
22
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE April 10, 2018
Actual Primary Completion Date April 10, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03474042
Other Study ID Numbers  ICMJE GLPG2737-CL-202
2017-002181-42 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Galapagos NV
Study Sponsor  ICMJE Galapagos NV
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Olivier Van de Steen, MD MBA Galapagos NV
PRS Account Galapagos NV
Verification Date June 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP