Study Evaluating Betrixaban in Pediatric Participants

April 11, 2023 updated by: Alexion Pharmaceuticals, Inc.

A Phase 1, Open-Label, Single-Dose, Non-Randomized Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Betrixaban in Pediatric Patients

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Russian Federation
      • Kazan, Russian Federation, 420138
        • Children's Hospital of Tatarstan Republic
      • Kemerovo, Russian Federation, 650002
        • Federal State Institution
      • Moscow, Russian Federation, 125412
        • Pirogov Russian National Research Medical University
      • Moscow, Russian Federation, 119049
        • Children's City Clinical Hospital
      • Nizhny Novgorod, Russian Federation, 603136
        • State Budgetary Institution
      • Saint Petersburg, Russian Federation, 194100
        • Saint Petersburg State Pediatric Medical University
  • Ukraine
      • Ivano-Frankivs'k, Ukraine, 76000
        • Ivano-Frankivsk Regional Children Clinical Hospital
      • Odesa, Ukraine
        • Odessa Regional Children Clinical Hospital
      • Sumy, Ukraine
        • Sumy Regional Children's Hospital
      • Vinnitsa, Ukraine
        • Vinnytsia Regional Children's Clinical Hospital, Department of Anesthesiology and Intensive Care
  • United Kingdom
      • Birmingham, United Kingdom, B4 6NH
        • Birmingham Women's and Children's NHS Foundation Trust
      • Cambridge, United Kingdom, CB2 0QQ
        • Addenbrooke's Hospital
      • Cardiff, United Kingdom, CF14 4XW
        • Children's Hospital for Wales
      • Leicester, United Kingdom, LE3 9QP
        • Glenfield Hospital
      • London, United Kingdom, SE1 7EH
        • Evelina London Children's Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Los Angeles, California, United States, 90036
        • ACTCA, Axis Clinical Trials
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta
    • Louisiana
      • New Orleans, Louisiana, United States, 70001
        • Tulane Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Rainbow Babies & Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

4 weeks to 17 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Pediatric participants in the following age categories: 12 to < 18 years of age and 2 to < 12 years of age. Part 1 of the study enrolled only adolescent participants 12 to < 18 years of age.

  2. Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:

    1. Had previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
    2. Had any stable disease with a risk for arterial or venous thromboembolism, or
    3. Had any functional central venous access device in the upper or lower venous system.
  3. Participant had normalized coagulation parameters (international normalized ratio or partial thromboplastin time, as appropriate) within 7 days of study drug administration.

Exclusion Criteria:

Participants who meet any one of the following exclusion criteria were excluded from the study:

  1. Participant received any dose of anti-coagulant therapy within 7 days of Day 1.
  2. Participant had active bleeding or had a comorbid disorder that placed the participant at high risk for bleeding.
  3. Participant had a comorbid disorder that placed the participant at risk of death within 90 days of enrollment.
  4. Participant had abnormal coagulation tests at baseline.
  5. Participant had recent or planned invasive procedures, including lumbar puncture and removal of non-peripherally placed central lines during study.

  6. Participant had hepatic disease associated with one or more of the following:

    • Transaminase levels ≥ 2.5 × upper limit of normal (ULN) or bilirubin ≥ 1.5 × ULN at baseline.
    • Coagulopathy leading to a clinically relevant bleeding risk, or hepatic transaminase level of > 2 × ULN or total bilirubin > 2 × ULN with direct bilirubin > 20% of the total.
    • Platelet count < 75 × 10^9/liter or hemoglobin < 10.0 mg/deciliter.
    • Hypertension.
  7. Participant had known congenital or acquired bleeding diathesis.
  8. Participant required concomitant therapy with a strong P-glycoprotein inhibitor.
  9. Participant had previous history of any non-traumatic bleeding event that was life threatening or required medical attention.
  10. Participant had been administered thrombolytic therapy, or had undergone thrombectomy, or insertion of a caval filter to treat prior VTE.
  11. Participant had known inherited or acquired bleeding diathesis or coagulopathy.
  12. Participant had abnormal QTcF interval on baseline electrocardiogram.
  13. Participant received a dose of any antiplatelet medication (including aspirin) within 14 days before study drug dosing.
  14. Participant had malabsorption disorders (for example, cystic fibrosis or short bowel syndrome).
  15. Participant had an estimated glomerular filtration rate < 30 milliliters/minute.
  16. Participant was unable or reluctant to cooperate with the study procedures.
  17. Participant had hypersensitivity to other Factor Xa inhibitors, or the components of the dosage form.
  18. Participant had participated in a study with an investigational drug or medical device within 30 days prior to administration of betrixaban.
  19. Participant was female and of childbearing potential and was either pregnant or breastfeeding a child.
  20. Participant was sexually active and was not using medically accepted contraceptive method (if applicable).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort 1: Betrixaban 40 mg
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
Drug: Betrixaban
Factor Xa inhibitor.
Other Names:
  • PRT054021
Experimental: Cohort 2: Betrixaban 80 mg
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
Drug: Betrixaban
Factor Xa inhibitor.
Other Names:
  • PRT054021

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Area Under The Plasma Concentration-Time Curve From 0 To Infinity (AUC(0-inf)) Of Betrixaban
Time Frame: Up to 6 days post dose
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
Up to 6 days post dose
Maximum Observed Plasma Concentration (Cmax) Of Betrixaban
Time Frame: Up to 6 days post dose
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.
Up to 6 days post dose

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
AUC To The Last Measurable Concentration Above The Quantitation Limit (AUC(0-last)) Of Betrixaban
Time Frame: Up to 6 days post dose
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-last) were not collected.
Up to 6 days post dose
Terminal Plasma Half-life (t½) Of Betrixaban
Time Frame: Up to 6 days post dose
Following the Sponsor's decision to cease developing betrixaban, data for t½ were not collected.
Up to 6 days post dose
Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban
Time Frame: Up to 6 days post dose
The Tmax that the highest (maximum) Cmax of betrixaban was observed per group up to Day 6 (120 hours) post dosing is reported.
Up to 6 days post dose
Apparent Total Body Clearance Of Betrixaban From Plasma (CL)
Time Frame: Up to 6 days post dose
Following the Sponsor's decision to cease developing betrixaban, data for CL were not collected.
Up to 6 days post dose
Apparent Volume Of Distribution (Vd) Of Betrixaban
Time Frame: Up to 6 days post dose
Following the Sponsor's decision to cease developing betrixaban, data for Vd were not collected.
Up to 6 days post dose
Percent Change From Baseline In Thrombin Level At Day 6
Time Frame: Baseline, Day 6
Following the Sponsor's decision to cease developing betrixaban, data for thrombin levels were not collected.
Baseline, Day 6
Count Of Participants With Treatment-related Adverse Events
Time Frame: Up to 7 days post dose
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.
Up to 7 days post dose

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Pharmacodynamic (PD) - Thrombin Level
Time Frame: Day 1 - Day 6
The pharmacodynamic (PD) endpoint is the inhibition of thrombin generation during the first 24 hours post dosing, evaluated by the percent change in thrombin level from baseline.
Day 1 - Day 6
Number of patients with treatment related Adverse Events (AE)
Time Frame: Day 1 - Day 6
The safety endpoint is the clinical assessment of AEs and other safety measurements (vital signs, ECGs, laboratory tests, etc.)
Day 1 - Day 6

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Portola Pharmaceuticals, LLC (a wholly owned subsidiary of Alexion Pharmaceuticals)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 13, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 8, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 8, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 7, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 14, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 17, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 12, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 11, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 16-021
  • 2018-002562-40 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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