Clinical trial NCT02981472: A Study of the Safety and Pharmacokinetics of Apixaban Versus Vitamin K Antagonist (VKA) or Low Molecular Weight Heparin (LMWH) in Pediatric Subjects With Congenital or Acquired Heart Disease Requiring Anticoagulation
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Thrombosis
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
Collaborators: name: Pediatric Heart Network
name: Pfizer
First posted: 2016-12-05
Enrollment: 192
Sex: ALL
Minimum age (years): 28
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To investigate the safety and pharmacokinetics of apixaban in children with congenital or acquired heart disease who have a need for anticoagulation.


Eligibility criteria: For more information regarding Bristol-Myers Squibb Clinical Trial participation, please visit www.BMSStudyConnect.com

Inclusion Criteria:

* Males and Females, 28 days to \< 18 years of age, inclusive
* Congenital or acquired heart diseases requiring chronic anticoagulation for thromboprophylaxis (eg, single ventricle physiology including all 3 stages of palliation, dilated cardiomyopathy, Kawasaki disease with coronary aneurysms, and pulmonary hypertension)
* Eligible participants include those who newly start anticoagulants and those who are currently on VKA or LMWH or other anticoagulants for thromboprophylaxis
* Able to tolerate enteral medication \[eg, by mouth, nasogastric tube, or gastric tube\]
* Participants 28 days to \< 3 months must be able to tolerate oral/nasogastric tube (NGT)/gastric tube (GT) feeds for at least 5 days prior to randomization

Exclusion Criteria:

* Recent thromboembolic events less than 6 months prior to enrollment
* Weight \< 3 kg
* 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
* Known antiphospholipid syndrome (APS).

Other protocol defined inclusion/exclusion criteria apply

Primary outcomes:
measure: Composite of Adjudicated Major or Clinically Relevant Non-Major (CRNM) Bleeding Events
timeFrame: From first dose to 2 days after last dose (Up to approximately 12 months)
Secondary outcomes:
measure: The Number of Participants With Thrombotic Events and Thromboembolic Event-Related Death
timeFrame: From randomization to 2 days after last dose (Up to approximately 12 months)
measure: The Number of Participants With Adjudicated Major Bleeding
timeFrame: From first dose to 2 days after last dose (Up to approximately 12 months)
measure: The Number of Participants With Adjudicated CRNM Bleeding
timeFrame: From first dose to 2 days after last dose (Up to approximately 12 months)
measure: The Number of Participants With All Adjudicated Bleeding
timeFrame: From first dose to 2 days after last dose (Up to approximately 12 months)
measure: The Number of Participants With Drug Discontinuation Due to Adverse Effects, Intolerability, or Bleeding
timeFrame: From first dose to 2 days after last dose (Up to approximately 12 months)
measure: The Number of Participant Deaths in the Study
timeFrame: From first dose to 2 days after last dose (Up to approximately 12 months)
measure: Maximum Observed Concentration (Cmax)
timeFrame: From first dose up to 6 months after first dose
measure: Trough Observed Concentration (Cmin)
timeFrame: From first dose up to 6 months after first dose
measure: Area Under the Concentration-Time Curve in One Dosing Interval (AUC (TAU))
timeFrame: From first dose up to 6 months after first dose
measure: Time of Maximum Observed Concentration (Tmax)
timeFrame: From first dose up to 6 months after first dose
measure: Anti-FXa Activity
timeFrame: From first dose up to 6 months after first dose
measure: Chromogenic FX Assay (Apparent FX Level)
timeFrame: From first dose up to 6 months after first dose
measure: The Child and Parent Reports of Pediatric Quality of Life Inventory (PedsQL)
timeFrame: from randomization up to 12 months after randomization
measure: Kids Informed Decrease Complications Learning on Thrombosis (KIDCLOT) IMPACT Score
timeFrame: from randomization up to 12 months after randomization
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children'S Hospital
state: Arizona
city: La Jolla
country: United States
facility: University Of California San Diego
state: California
city: Aurora
country: United States
facility: Children'S Hospital Colorado
state: Colorado
city: Atlanta
country: United States
facility: Childrens Healthcare Of Atlanta
state: Georgia
city: Indianapolis
country: United States
facility: Local Institution - 0008
state: Indiana
city: Boston
country: United States
facility: Local Institution - 0009
state: Massachusetts
city: Ann Arbor
country: United States
facility: Local Institution - 0013
state: Michigan
city: Kansas City
country: United States
facility: Childrens Mercy Hospital
state: Missouri
city: Cincinnati
country: United States
facility: Local Institution - 0006
state: Ohio
city: Philadelphia
country: United States
facility: Childrens Hospital Of Philadelphia
state: Pennsylvania
city: Charleston
country: United States
facility: Local Institution - 0012
state: South Carolina
city: Houston
country: United States
facility: Local Institution - 0011
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Seattle
country: United States
facility: Local Institution - 0055
state: Washington
city: Caba
country: Argentina
facility: Local Institution
state: Buenos Aires
city: Parkville
country: Australia
facility: Local Institution - 0030
state: Victoria
city: Vienna
country: Austria
facility: Local Institution - 0001
city: Curitiba
country: Brazil
facility: Local Institution - 0022
state: Parana
city: Porto Alegre
country: Brazil
facility: Instituto De Cardiologia Do Rio Grande Do Sul
state: Rio Grande Do Sul
city: Campinas
country: Brazil
facility: Local Institution - 0020
state: Sao Paulo
city: Sao Paulo
country: Brazil
facility: Instituto de Pesquisa PENSI
city: Sao Paulo
country: Brazil
facility: Universidade Federal De Sao Paulo
city: Toronto
country: Canada
facility: Local Institution - 0015
state: Ontario
city: HUS
country: Finland
facility: Local Institution - 0031
city: Freiburg
country: Germany
facility: Local Institution - 0002
city: Hamburg
country: Germany
facility: Local Institution - 0004
city: Muenchen
country: Germany
facility: Local Institution - 0003
city: Petach Tikva
country: Israel
facility: Local Institution - 0047
city: Tel Hashomer
country: Israel
facility: Local Institution - 0046
city: Rome
country: Italy
facility: Local Institution - 0026
state: Roma
city: Bologna
country: Italy
facility: Local Institution - 0028
city: Milano
country: Italy
facility: Local Institution - 0027
city: Mexico City
country: Mexico
facility: Local Institution - 0018
state: Distrito Federal
city: Mexico City
country: Mexico
facility: Local Institution - 0019
state: Distrito Federal
city: Mexico City
country: Mexico
facility: Local Institution - 0016
state: Distrito Federal
city: Leon
country: Mexico
facility: Local Institution - 0044
state: Guanajuato
city: Ekaterinburg
country: Russian Federation
facility: Local Institution - 0062
city: Kazan
country: Russian Federation
facility: Local Institution
city: Kemerovo
country: Russian Federation
facility: Local Institution
city: Novosibirsk
country: Russian Federation
facility: Local Institution - 0057
city: Barcelona
country: Spain
facility: Local Institution - 0049
city: Madrid
country: Spain
facility: Local Institution
city: Madrid
country: Spain
facility: Local Institution - 0048
city: Leicester
country: United Kingdom
facility: Local Institution - 0040
state: Leicestershire
city: Bristol
country: United Kingdom
facility: Local Institution - 0042
state: Somerset
city: Manchester
country: United Kingdom
facility: Local Institution
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: