Study Evaluating Betrixaban in Pediatric Participants

Study Identifier:
16-021
ClinicalTrials.gov Identifier:
NCT03346083
EudraCT Identifier:
2018-002562-40
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Available Documents

ProtocolStatistical Analysis Plan

Study Details

Medical Condition
  • VTE Prophylaxis
Study Drug
  • Drug: Betrixaban
Date
Jul 2018 - Oct 2019
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 2 - 17 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Pediatric participants in the following age categories: 12 to \< 18 years of age and 2 to \< 12 years of age. Part 1 of the study enrolled only adolescent participants 12 to \< 18 years of age.
  • Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:
  • Had previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
  • Had any stable disease with a risk for arterial or venous thromboembolism, or
  • Had any functional central venous access device in the upper or lower venous system.
  • Participant had normalized coagulation parameters (international normalized ratio or partial thromboplastin time, as appropriate) within 7 days of study drug administration.
Exclusion Criteria
  • Participants who meet any one of the following exclusion criteria were excluded from the study:
  • Participant received any dose of anti-coagulant therapy within 7 days of Day 1.
  • Participant had active bleeding or had a comorbid disorder that placed the participant at high risk for bleeding.
  • Participant had a comorbid disorder that placed the participant at risk of death within 90 days of enrollment.
  • Participant had abnormal coagulation tests at baseline.
  • Participant had recent or planned invasive procedures, including lumbar puncture and removal of non-peripherally placed central lines during study.
  • Participant had hepatic disease associated with one or more of the following:
  • Transaminase levels ≥ 2.5 × upper limit of normal (ULN) or bilirubin ≥ 1.5 × ULN at baseline.
  • Coagulopathy leading to a clinically relevant bleeding risk, or hepatic transaminase level of \> 2 × ULN or total bilirubin \> 2 × ULN with direct bilirubin \> 20% of the total.
  • Platelet count \< 75 × 10\^9/liter or hemoglobin \< 10.0 mg/deciliter.
  • Hypertension.
  • Participant had known congenital or acquired bleeding diathesis.
  • Participant required concomitant therapy with a strong P-glycoprotein inhibitor.
  • Participant had previous history of any non-traumatic bleeding event that was life threatening or required medical attention.
  • Participant had been administered thrombolytic therapy, or had undergone thrombectomy, or insertion of a caval filter to treat prior VTE.
  • Participant had known inherited or acquired bleeding diathesis or coagulopathy.
  • Participant had abnormal QTcF interval on baseline electrocardiogram.
  • Participant received a dose of any antiplatelet medication (including aspirin) within 14 days before study drug dosing.
  • Participant had malabsorption disorders (for example, cystic fibrosis or short bowel syndrome).
  • Participant had an estimated glomerular filtration rate \< 30 milliliters/minute.
  • Participant was unable or reluctant to cooperate with the study procedures.
  • Participant had hypersensitivity to other Factor Xa inhibitors, or the components of the dosage form.
  • Participant had participated in a study with an investigational drug or medical device within 30 days prior to administration of betrixaban.
  • Participant was female and of childbearing potential and was either pregnant or breastfeeding a child.
  • Participant was sexually active and was not using medically accepted contraceptive method (if applicable).
Sex
Female & Male
Age
2 - 17 Years

Study Details

Medical Condition
  • VTE Prophylaxis
Study Drug
  • Drug: Betrixaban
Date
Jul 2018 - Oct 2019
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 2 - 17 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Pediatric participants in the following age categories: 12 to \< 18 years of age and 2 to \< 12 years of age. Part 1 of the study enrolled only adolescent participants 12 to \< 18 years of age.
  • Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:
  • Had previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
  • Had any stable disease with a risk for arterial or venous thromboembolism, or
  • Had any functional central venous access device in the upper or lower venous system.
  • Participant had normalized coagulation parameters (international normalized ratio or partial thromboplastin time, as appropriate) within 7 days of study drug administration.
Exclusion Criteria
  • Participants who meet any one of the following exclusion criteria were excluded from the study:
  • Participant received any dose of anti-coagulant therapy within 7 days of Day 1.
  • Participant had active bleeding or had a comorbid disorder that placed the participant at high risk for bleeding.
  • Participant had a comorbid disorder that placed the participant at risk of death within 90 days of enrollment.
  • Participant had abnormal coagulation tests at baseline.
  • Participant had recent or planned invasive procedures, including lumbar puncture and removal of non-peripherally placed central lines during study.
  • Participant had hepatic disease associated with one or more of the following:
  • Transaminase levels ≥ 2.5 × upper limit of normal (ULN) or bilirubin ≥ 1.5 × ULN at baseline.
  • Coagulopathy leading to a clinically relevant bleeding risk, or hepatic transaminase level of \> 2 × ULN or total bilirubin \> 2 × ULN with direct bilirubin \> 20% of the total.
  • Platelet count \< 75 × 10\^9/liter or hemoglobin \< 10.0 mg/deciliter.
  • Hypertension.
  • Participant had known congenital or acquired bleeding diathesis.
  • Participant required concomitant therapy with a strong P-glycoprotein inhibitor.
  • Participant had previous history of any non-traumatic bleeding event that was life threatening or required medical attention.
  • Participant had been administered thrombolytic therapy, or had undergone thrombectomy, or insertion of a caval filter to treat prior VTE.
  • Participant had known inherited or acquired bleeding diathesis or coagulopathy.
  • Participant had abnormal QTcF interval on baseline electrocardiogram.
  • Participant received a dose of any antiplatelet medication (including aspirin) within 14 days before study drug dosing.
  • Participant had malabsorption disorders (for example, cystic fibrosis or short bowel syndrome).
  • Participant had an estimated glomerular filtration rate \< 30 milliliters/minute.
  • Participant was unable or reluctant to cooperate with the study procedures.
  • Participant had hypersensitivity to other Factor Xa inhibitors, or the components of the dosage form.
  • Participant had participated in a study with an investigational drug or medical device within 30 days prior to administration of betrixaban.
  • Participant was female and of childbearing potential and was either pregnant or breastfeeding a child.
  • Participant was sexually active and was not using medically accepted contraceptive method (if applicable).

Protocol Summary

This trial was a Phase 1, open-label, multicenter study of the pharmacokinetics (PK), pharmacodynamics (PD), and safety of a single dose of betrixaban in pediatric participants at risk of venous thromboembolism (VTE).

Trial Locations

Location
Status
Location
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Status
N/A
Location
ACTCA, Axis Clinical Trials
Los Angeles, California, United States, 90036
Status
N/A
Location
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Status
N/A
Location
Tulane Medical Center
New Orleans, Louisiana, United States, 70001
Status
N/A
Location
Rainbow Babies & Children's Hospital
Cleveland, Ohio, United States, 44106
Status
N/A
Location
Children's Hospital of Tatarstan Republic
Kazan', Russian Federation, 420138
Status
N/A
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