Apixaban for the Acute Treatment of Venous Thromboembolism in Children

Bristol-Myers Squibb229 enrolled

Overview

To assess the safety and descriptive efficacy of apixaban in pediatric subjects requiring anticoagulation for the treatment of a VTE.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

229

Conditions

Venous Thromboembolism

Interventions

ApixabanDRUG

Tablet or Solution

Standard of CareDRUG

Unfractionated heparin, low molecular weight heparin, and/or a vitamin K antagonist. For subjects under 2 years of age, standard of care will be limited to unfractionated heparin or low molecular weight heparin.

Eligibility

Sex: ALLMin age: 0 DaysMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Birth to \<18 years of age with a minimum weight of 2.6 kg at the time of randomization. 2. Presence of an index VTE which is confirmed by imaging. 3. Intention to manage the index VTE with anticoagulation treatment for at least 6 to 12 weeks. 4. Subjects able to tolerate oral feeding, nasogastric (NG), gastric (G) feeding and are currently tolerating enteric medications, as per investigator's judgement. Exclusion Criteria: 1. Anticoagulant treatment for the index VTE for greater than 14 days prior to randomization. Neonates that are enrolled into the PK cohort must be on a minimum of 5 days and a maximum of 14 days SOC anticoagulation prior to randomization. Neonates that are enrolled into the post PK cohort may receive SOC anticoagulation for up to 14 days prior to randomization. 2. Thrombectomy, thrombolytic therapy, or insertion of a caval filter to treat the index VTE. 3. A mechanical heart valve. 4. Active bleeding or high risk of bleeding at the time of randomization. 5. Intracranial bleed, including intraventricular hemorrhage, within 3 months prior to randomization. 6. Abnormal baseline liver function at randomization. 7. Inadequate renal function at the time of randomization. 8. Platelet count \<50×109 per L at randomization. 9. Uncontrolled severe hypertension at the time of randomization. 10. Use of prohibited concomitant medication at the time of randomization. 11. Female subjects who are either pregnant or breastfeeding a child. 12. Use of aggressive life-saving therapies such as ventricular assist devices (VAD) or extracorporeal membrane oxygenation (ECMO) at the time of enrollment. 13. Unable to take oral or enteric medication via the NG or G tube. 14. Known inherited or acquired antiphospholipid syndrome (APS). 15. Known inherited bleeding disorder or coagulopathy with increased bleeding risk (eg, hemophilia, von Willebrand disease, etc.)

Locations (128)

Outcomes

Primary Outcomes

Percentage of Participants With Composite of Major and Clinically Relevant Non-Major (CRNM) Bleeding

Bleeding definitions are based on the Perinatal and Paediatric Haemostasis Subcommittee of the International Society on Thrombosis and Haemostasis (ISTH) criteria. Major bleeding includes: (i) fatal bleeding; (ii) clinically overt bleeding with a decrease in Hgb of at least 20 g/L (2 g/dL) in 24 hours; (iii) retroperitoneal, pulmonary, intracranial, or central nervous system bleeding; and (iv) bleeding requiring surgical intervention in an operating suite (including interventional radiology). Clinically relevant non-major bleeding includes: (i) overt bleeding requiring a blood product not attributable to the participant's underlying condition; and (ii) bleeding requiring medical or surgical intervention to restore hemostasis, other than in an operating suite.

Time frame: From first dose (Day 1) up to 114 days

Percentage of Participants With Symptomatic and Asymptomatic Recurrent Venous Thromboembolism (VTE) and VTE-Related Mortality

Recurrent VTE, defined as either contiguous progression or non-contiguous new thrombus and including, but not limited to deep vein thrombosis (DVT), pulmonary embolism (PE) and paradoxical embolism. 95% CI was from the Agresti-Coull method.

Time frame: From first dose (Day 1) up to 114 days

Secondary Outcomes

Percentage of Participants Who Died

Death due to any cause was assessed. 95% CI was calculated using the Agresti-Coull method.

Time frame: From first dose (Day 1) up to 114 days

Percentage of Participants With Venous Thromboembolism (VTE)-Related Mortality

Participants were assessed for death due to Venous Thromboembolism (VTE).

Time frame: From first dose (Day 1) up to 114 days

Number of Participants With Index Venous Thromboembolism (VTE) Status

Data from ClinicalTrials.gov

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Pediatric Cardiology Clinic

Birmingham, Alabama, United States

University of Alabama

Birmingham, Alabama, United States

Phoenix Children's Hospital

Phoenix, Arizona, United States

Loma Linda University Cancer Center

Loma Linda, California, United States

Loma Linda University Children's Hospital

Loma Linda, California, United States

Loma Linda University Medical Center

Loma Linda, California, United States

Valley Children's Hospital

Madera, California, United States

Children's Hospital and Research Center Oakland

Oakland, California, United States

Bass Speicalty Pharmacy

Palo Alto, California, United States

Inpatient Pharmacy

Palo Alto, California, United States

...and 118 more locations

Number of Participants With Clinically Relevant Non-Major (CRNM) Bleeding, Major Bleeding and Minor Bleeding

Time frame: From first dose (Day 1) up to 114 days