EDO312: Edoxaban, a new Oral Anticoagulant, for Blood Clots in Children

Brief Summary

Currently available anticoagulants have significant limitations in children. The most commonly used agents for long-term anticoagulation in pediatric patients are low molecular weight heparin (LMWH) and vitamin K antagonists (VKAs). These standard therapies carry associated risks and are difficult to administer in young children. LMWH requires subcutaneous injection twice a day and VKAs require frequent blood level monitoring.

Edoxaban is an oral direct inhibitor of activated Factor X with predictable pharmacokinetics and pharmacodynamics. It has been approved for use in the United States to reduce the risk of stroke and systemic embolism and treatment of deep vein thrombosis and pulmonary embolism in certain adult populations. It has not been approved for use in children.

This study will evaluate the pharmacokinetics and pharmacodynamics of edoxaban and compare its efficacy and safety with Standard of Care in pediatric patients with confirmed venous thromboembolism.

Primary Objective

• To demonstrate the non-inferiority of edoxaban to standard of care in the treatment and secondary prevention of venous thromboembolism (VTE) in children

Eligibility Criteria

Inclusion criteria include:

• Newborn to 17 years old (must be younger than 18)
• Diagnosis of venous thromboembolism
• Requires anticoagulant therapy for at least 90 days
• Received at least 5 days of heparin therapy prior to study

Exclusion Criteria include:

• Pregnant
• Active bleeding or high risk of bleeding
• Prior or current treatment with thrombolytic agents, thrombectomy or caval filter insertion
• Antiplatelet therapy (except for low-dose aspirin)
• Stage 2 hypertension

Study Design

Phase III, prospective, randomized, open-label, blinded endpoint evaluation parallel group study

Study Sites

St. Jude Children’s Research Hospital, Memphis, Tennessee
Collaborating sites in and outside the U.S.

About this study

Edoxaban is an investigational medicine that may reduce blood clots. It is considered an experimental drug because it is still being studied. It has been approved by the United States Food and Drug Administration (FDA) for use in adults but not for use in children.

A blood clot is blood that becomes a solid instead of a liquid. When a blood clot occurs in a vein, it is known as venous thromboembolism.

Several drugs are currently used to treat blood clots in children.  These are called “Standard of Care.” This study will compare edoxaban to currently available drugs to see which one works better. Study participants will be randomly placed in one of two groups.  You will have a 50/50 chance (like flipping a coin) to get in the edoxaban group or the Standard of Care group.

Children in one group will be given the study medicine, while children in the other group will be given the drug that is currently being used to treat blood clots. You will know which drug you are going to receive.  At the end of the study, researchers will compare which of the two treatments has the best results.

Purpose of this clinical trial

The main goal of this study is to see if edoxaban is safe to use in children and how well it treats blood clots in children, compared to currently available medicines.

Eligibility overview

• Newborn to 17 years old (must be younger than 18)
• Diagnosis of blood clot (venous thromboembolism, also called VTE)
• Requires anticoagulant therapy for at least 90 days
• Received at least 5 days of heparin therapy prior to study