Study of the Safety and Pharmacokinetics of apixaban versus warfarin or low molecular weight heparin in children with congenital or acquired heart disease that need anticoagulation

A Prospective, Randomized, Open Label, Multi-center Study of the Safety and Pharmacokinetics of Apixaban versus Vitamin K Antagonist or LMWH in Pediatric Subjects with Congenital or Acquired Heart Disease Requiring Chronic Anticoagulation for Thromboembolism Prevention - Safety and Pharmacokinetics of apixaban versus warfarin or low molecular weight heparin in children

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-001247-39-FI

Enrollment

150

Registered

2017-02-23

Start date

2017-05-09

Completion date

Unknown

Last updated

2021-11-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital or Acquired Heart Disease Requiring Chronic Anticoagulation for Thromboembolism Prevention MedDRA version: 20.0 Level: PT Classification code 10019273 Term: Heart disease congenital System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10007636 Term: Cardiomyopathy System Organ Class: 10007541 - Cardiac disorders MedDRA version: 20.0 Level: LLT Classification code 10019276 Term: Heart disease, unspecified System

Interventions

Product Name: APIXABAN Product Code: BMS-562247-01 Pharmaceutical Form: Oral solution INN or Proposed INN: APIXABAN Current Sponsor code: PF-0465257/BMS-562247-01 Concentration unit: mg/ml milligram(s

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Males and females, 34 weeks adjusted gestational age to =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: •Recent thromboembolic events less than 6 months prior to enrollment •Use of aggressive life-saving therapies such as ventricular assist devices (VAD) or extracorporeal membrane oxygenation (ECMO) at the time of enrollment •Artificial heart valves and mechanical heart valves •Known inherited bleeding disorder or coagulopathy (e.g. hemophilia, von Willebrand disease, etc.) •Active bleeding at the time of enrollment •Any major bleeding other than perioperative in the preceding 3 months •Known intracranial congenital vascular malformation or tumor •Confirmed diagnosis of a GI ulcer

Design outcomes

Primary

Measure	Time frame
Main Objective: Primary Objectives: To assess the safety of apixaban, compared to VKA or subcutaneous LMWH and to evaluate apixaban PK in pediatric subjects with congenital or acquired heart disease requiring chronic anticoagulation for thromboprophylax;Secondary Objective: Secondary Objectives: In pediatric subjects with congenital or acquired heart disease requiring chronic anticoagulation for thromboprophylaxis: • To assess apixaban PD by measuring FX using chromogenic assay and anti FXa activity • To compare the effects of apixaban on QOL measures versus VKAs antagonists or subcutaneous LMWH • To gather exploratory data on the efficacy of apixaban;Primary end point(s): •A composite of adjudicated major or clinically relevant non-major (CRNM) bleeding events per the Perinatal and Paediatric Haemostasis Subcommittee of the International Society on Thrombosis and Haemostasis (ISTH) criteria Major bleeding is defined as bleeding that satisfies one or more of the following criteria: ofatal bleeding oclinically overt bleeding associated with a decrease in hemoglobin of at least 20 g/L (ie, 2 g/dL) in a 24 hour period obleeding that is retroperitoneal, pulmonary, intracranial, or otherwise involves the central nervous system obleeding that requires surgical intervention in an operating suite, including interventional radiology CRNM bleeding is defined as bleeding which satisfies one or both of the following criteria: oovert bleeding for which blood product is administered and that is not directly attributable to the subject's underlying medical condition obleeding that requires medical or surgical intervention to restore hemostasis, other than in an operating room ;Timepoint(s) of evaluation of this end point: 14 months	—

Measure

Time frame

Main Objective: Primary Objectives: To assess the safety of apixaban, compared to VKA or subcutaneous LMWH and to evaluate apixaban PK in pediatric subjects with congenital or acquired heart disease requiring chronic anticoagulation for thromboprophylax;Secondary Objective: Secondary Objectives: In pediatric subjects with congenital or acquired heart disease requiring chronic anticoagulation for thromboprophylaxis: • To assess apixaban PD by measuring FX using chromogenic assay and anti FXa activity • To compare the effects of apixaban on QOL measures versus VKAs antagonists or subcutaneous LMWH • To gather exploratory data on the efficacy of apixaban;Primary end point(s): •A composite of adjudicated major or clinically relevant non-major (CRNM) bleeding events per the Perinatal and Paediatric Haemostasis Subcommittee of the International Society on Thrombosis and Haemostasis (ISTH) criteria Major bleeding is defined as bleeding that satisfies one or more of the following criteria: ofatal bleeding oclinically overt bleeding associated with a decrease in hemoglobin of at least 20 g/L (ie, 2 g/dL) in a 24 hour period obleeding that is retroperitoneal, pulmonary, intracranial, or otherwise involves the central nervous system obleeding that requires surgical intervention in an operating suite, including interventional radiology CRNM bleeding is defined as bleeding which satisfies one or both of the following criteria: oovert bleeding for which blood product is administered and that is not directly attributable to the subject's underlying medical condition obleeding that requires medical or surgical intervention to restore hemostasis, other than in an operating room ;Timepoint(s) of evaluation of this end point: 14 months

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Secondary

Measure	Time frame
Secondary end point(s): •Apparent total body clearance •Central volume of distribution •Absorption rate constant •Cmax Maximum observed concentration •Cmin Trough observed concentration •AUC (TAU) Area under the concentration-time curve in one dosing interval •Factor X activity measured using chromogenic assay •Anti-FXa (Anti-factor 10a) activity measured using chromogenic assay •Exposure-response (E-R) relationships •Any thromboembolic events detected by imaging or clinical diagnosis Thromboembolic events include but are not limited to: intra-cardiac, shunt, inside Fontan pathway, pulmonary embolism (PE), stroke, other venous or arterial thromboembolic events •Thromboembolic event-related death •Patient/proxy reported outcome or quality of life Determined by The Pediatric Quality of Life Inventory (PedsQL) generic core and cardiac modules, and Kids Informed Decrease Complications Learning on Thrombosis (KIDCLOT©) pediatric quality of life inventory •Adjudicated major bleeding •Adjudicated CRNM bleeding •All bleeding This includes minor bleeding, which is defined as any overt or macroscopic evidence of bleeding that does not fulfill the criteria for either major bleeding or CRNM bleeding •Drug discontinuation Can be due to adverse effects, intolerability, or bleeding •All causes of death;Timepoint(s) of evaluation of this end point: 14 months	—

Measure

Time frame

Secondary end point(s): •Apparent total body clearance •Central volume of distribution •Absorption rate constant •Cmax Maximum observed concentration •Cmin Trough observed concentration •AUC (TAU) Area under the concentration-time curve in one dosing interval •Factor X activity measured using chromogenic assay •Anti-FXa (Anti-factor 10a) activity measured using chromogenic assay •Exposure-response (E-R) relationships •Any thromboembolic events detected by imaging or clinical diagnosis Thromboembolic events include but are not limited to: intra-cardiac, shunt, inside Fontan pathway, pulmonary embolism (PE), stroke, other venous or arterial thromboembolic events •Thromboembolic event-related death •Patient/proxy reported outcome or quality of life Determined by The Pediatric Quality of Life Inventory (PedsQL) generic core and cardiac modules, and Kids Informed Decrease Complications Learning on Thrombosis (KIDCLOT©) pediatric quality of life inventory •Adjudicated major bleeding •Adjudicated CRNM bleeding •All bleeding This includes minor bleeding, which is defined as any overt or macroscopic evidence of bleeding that does not fulfill the criteria for either major bleeding or CRNM bleeding •Drug discontinuation Can be due to adverse effects, intolerability, or bleeding •All causes of death;Timepoint(s) of evaluation of this end point: 14 months

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Countries

Argentina, Australia, Austria, Belgium, Brazil, Canada, Finland, Germany, Ireland, Italy, Mexico, Spain, Sweden, United Kingdom

Contacts

Public ContactGCT-SU

Bristol-Myers Squibb International Corporation

clinical.trials@bms.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026