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Res Pract Thromb Haemost. 2018 Apr 24;2(3):580-590. doi: 10.1002/rth2.12093. eCollection 2018 Jul.

Phase 3, single-arm, multicenter study of dabigatran etexilate for secondary prevention of venous thromboembolism in children: Rationale and design.

Author information

1
OncoHematology Department Bambino Gesù Children's Hospital Rome Italy.
2
Hematology Department University Children's Hospital Zürich Switzerland.
3
Department of Clinical Development Boehringer Ingelheim RCV GmbH & Co. KG Vienna Austria.
4
Texas Children's Cancer Center Baylor College of Medicine Houston TX USA.
5
Clinical Development and Medical Affairs Boehringer Ingelheim Pharma GmbH & Co. KG Ingelheim Germany.
6
Faculty of Medicine Mannheim University of Heidelberg Mannheim Germany.
7
Royal Hospital for Children Glasgow UK.
8
Children's Hospital of Eastern Ontario University of Ottawa Ottawa ON Canada.
9
Boehringer Ingelheim Ltd Bracknell Berkshire UK.
10
Translational Medicine and Clinical Pharmacology Boehringer Ingelheim Pharmaceuticals, Inc. Ridgefield CT USA.
11
University of Alberta Edmonton AB Canada.
12
Biostatistics and Data Sciences Boehringer Ingelheim Pharmaceuticals, Inc. Ridgefield CT USA.
13
Department of Pediatrics Division of Hematology/Oncology The Hospital for Sick Children University of Toronto Toronto ON Canada.

Abstract

Background:

Anticoagulant therapy for venous thromboembolism (VTE) in children is largely based on treatment recommendations for adults. However, differences in both physiology (ie, renal maturation and drug excretion) and developmental hemostasis must be considered when treating children, as such differences could affect dose appropriateness, safety and efficacy.

Objectives:

To address these concerns, a study was designed to evaluate the safety of dabigatran etexilate in children requiring secondary thrombus prevention in whom an initial VTE was associated with an identified risk factor that persisted after the acute VTE treatment period. We report herein the rationale and design of the study.

Patients/Methods:

This phase 3, open-label, single-arm, multicenter, multinational, prospective cohort study will be conducted in ≥100 children aged 0 to <18 years at ~100 specialist sites worldwide. Children will be treated with dabigatran etexilate for 12 months, or for a shorter duration if their identified VTE risk factor resolves, as per current American College of Chest Physicians recommendations. A nomogram will be used to determine starting doses for each patient.

Results:

The primary outcomes of the study will be VTE recurrence, bleeding events, overall mortality, and VTE-related mortality. Secondary outcomes will include occurrence of post-thrombotic syndrome, the pharmacokinetics of dabigatran, and the need for dose adjustments during treatment. Data on adverse events during the study will also be collected.

Conclusion:

This study will evaluate the safety of dabigatran etexilate for the secondary prevention of VTE in children, in addition to providing further data to guide pediatric dosing with dabigatran.

KEYWORDS:

anticoagulant; dabigatran; pediatrics; secondary prevention; venous thromboembolism

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