Phase 2
N=192
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
Thrombosis
Bottom Line
View on ClinicalTrials.gov: NCT02981472 ↗Enrolled (actual)
192
Serious AEs
20.7%
Results posted
May 2022
Primary outcome: Primary: Composite of Adjudicated Major or Clinically Relevant Non-Major (CRNM) Bleeding Events — 1; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Apixaban (Drug); Vitamin K Antagonist (VKA) (Drug); Low Molecular Weight Heparin (LMWH) (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Bristol-Myers Squibb
- Primary completion
- Oct 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Composite of Adjudicated Major or Clinically Relevant Non-Major (CRNM) Bleeding Events |
1; 3 | — |
| SECONDARY The Number of Participants With Thrombotic Events and Thromboembolic Event-Related Death |
0; 0; 0; 0 | — |
| SECONDARY The Number of Participants With Adjudicated Major Bleeding |
1; 1 | — |
| SECONDARY The Number of Participants With Adjudicated CRNM Bleeding |
1; 2 | — |
| SECONDARY The Number of Participants With All Adjudicated Bleeding |
47; 23 | — |
| SECONDARY The Number of Participants With Drug Discontinuation Due to Adverse Effects, Intolerability, or Bleeding |
7; 1 | — |
| SECONDARY The Number of Participant Deaths in the Study |
0; 0 | — |
| SECONDARY Maximum Observed Concentration (Cmax) |
185; 218; 222; 244; 249; 203 | — |
| SECONDARY Trough Observed Concentration (Cmin) |
57.9; 82.7; 64.3; 67.4; 73.1; 72.7 | — |
| SECONDARY Area Under the Concentration-Time Curve in One Dosing Interval (AUC (TAU)) |
1460; 1840; 1610; 1760; 1840; 1630 | — |
| SECONDARY Time of Maximum Observed Concentration (Tmax) |
2.24; 2.47; 1.72; 1.74; 1.65; 1.85 | — |
| SECONDARY Anti-FXa Activity |
147.69; 86.24; 242.34; 228.88; 66.93 | — |
| SECONDARY Chromogenic FX Assay (Apparent FX Level) |
58.87; 18.90; 35.88; 21.26; 18.25; 36.57 | — |
| SECONDARY The Child and Parent Reports of Pediatric Quality of Life Inventory (PedsQL) |
69.64; 60.71; 73.37; 64.81; 65.61; 65.42 | — |
| SECONDARY Kids Informed Decrease Complications Learning on Thrombosis (KIDCLOT) IMPACT Score |
24.35; 26.45; 22.81; 22.57; 22.50; 25.32 | — |
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
Data sourced from ClinicalTrials.gov (NCT02981472). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.