This trial was a Phase 1, open-label, multicenter study of the pharmacokinetics (PK), pharmacodynamics (PD), and safety of a single dose of betrixaban in pediatric participants at risk of venous thromboembolism (VTE).
This study was to be conducted in 2 parts: Part 1 and Part 2. Part 1 (the initial opening of the study) was conducted in 21 adolescent participants (12 to \< 18 years of age) who were assessed to be at risk for VTE. Participants in Part 1 received either 40 or 80 milligrams (mg) of study drug. The PK and PD data from Part 1 was to be used for dose determination for the next youngest age group using population PK and physiological-based PK modeling and simulation. Following analysis of Part 1 data, Part 2 of the study was to commence and enroll 12 participants 2 to \< 12 years of age. However, after completion of Part 1 and prior to initiating Part 2, the Sponsor decided to cease developing betrixaban, prompting early study closure.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
21
Factor Xa inhibitor.
Children's Hospital Los Angeles
Los Angeles, California, United States
ACTCA, Axis Clinical Trials
Area Under The Plasma Concentration-Time Curve From 0 To Infinity (AUC(0-inf)) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
Time frame: Up to 6 days post dose
Maximum Observed Plasma Concentration (Cmax) Of Betrixaban
Data reported as "0.200" indicates that the data are below the lower limit of quantification. Note that the Measure of Central Tendency could not be determined for Cohort 1 or Cohort 2 due to the values that are below the lower limit of quantification.
Time frame: Up to 6 days post dose
AUC To The Last Measurable Concentration Above The Quantitation Limit (AUC(0-last)) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-last) were not collected.
Time frame: Up to 6 days post dose
Terminal Plasma Half-life (t½) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for t½ were not collected.
Time frame: Up to 6 days post dose
Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban
The Tmax that the highest (maximum) Cmax of betrixaban was observed per group up to Day 6 (120 hours) post dosing is reported.
Time frame: Up to 6 days post dose
Apparent Total Body Clearance Of Betrixaban From Plasma (CL)
Following the Sponsor's decision to cease developing betrixaban, data for CL were not collected.
Time frame: Up to 6 days post dose
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Los Angeles, California, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Tulane Medical Center
New Orleans, Louisiana, United States
Rainbow Babies & Children's Hospital
Cleveland, Ohio, United States
Children's Hospital of Tatarstan Republic
Kazan', Russia
Federal State Institution
Kemerovo, Russia
Children's City Clinical Hospital
Moscow, Russia
Pirogov Russian National Research Medical University
Moscow, Russia
State Budgetary Institution
Nizhny Novgorod, Russia
...and 10 more locations
Apparent Volume Of Distribution (Vd) Of Betrixaban
Following the Sponsor's decision to cease developing betrixaban, data for Vd were not collected.
Time frame: Up to 6 days post dose
Percent Change From Baseline In Thrombin Level At Day 6
Following the Sponsor's decision to cease developing betrixaban, data for thrombin levels were not collected.
Time frame: Baseline, Day 6
Count Of Participants With Treatment-related Adverse Events
A treatment-related adverse event was any undesirable event or any untoward medical occurrence that occurs to a participant during the course of a study, or the protocol-defined time after study termination. An Investigator qualified in medicine made the determination of relationship to the investigational product for each adverse event (Unrelated, Unlikely Related, Possibly Related, or Probably Related). If the relationship between the adverse event and the investigational product was determined to be "possible" or "probable", the event was considered to be related to the investigational product for the purposes of expedited regulatory reporting. One participant experienced a mild study-drug-related headache that resolved in less than 2 hours. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Time frame: Up to 7 days post dose