Study Evaluating Betrixaban in Pediatric Participants

NCT03346083 | Terminated Phase 1 Results FDA Drug

• 2 other identifiers: 16-0212018-002562-40
• Study Type: interventional
• Target: 21
• Locations: 4 countries
• Sites: 20

Brief Summary

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).

Conditions

VTE Prophylaxis

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Up to 6 days post dose

Secondary Outcomes (7)

• AUC To The Last Measurable Concentration Above The Quantitation Limit (AUC(0-last)) Of Betrixaban

  Up to 6 days post dose

• Terminal Plasma Half-life (t½) Of Betrixaban

  Up to 6 days post dose

• Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban

  Up to 6 days post dose

• Apparent Total Body Clearance Of Betrixaban From Plasma (CL)

  Up to 6 days post dose

• Apparent Volume Of Distribution (Vd) Of Betrixaban

  Up to 6 days post dose

Study Arms (2)

Cohort 1: Betrixaban 40 mg

EXPERIMENTAL

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

Cohort 2: Betrixaban 80 mg

EXPERIMENTAL

Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.

Drug: Betrixaban

Interventions

Betrixaban DRUG

Factor Xa inhibitor.

Also known as: PRT054021

Cohort 1: Betrixaban 40 mg; Cohort 2: Betrixaban 80 mg

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Pediatric participant who was assessed to be at risk for VTE but did not require immediate anticoagulant therapy, for example:
• Had previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
• Had any stable disease with a risk for arterial or venous thromboembolism, or
• Had any functional central venous access device in the upper or lower venous system.
• Participant had normalized coagulation parameters (international normalized ratio or partial thromboplastin time, as appropriate) within 7 days of study drug administration.

You may not qualify if:

• Participant received any dose of anti-coagulant therapy within 7 days of Day 1.
• Participant had active bleeding or had a comorbid disorder that placed the participant at high risk for bleeding.
• Participant had a comorbid disorder that placed the participant at risk of death within 90 days of enrollment.
• Participant had abnormal coagulation tests at baseline.
• Participant had recent or planned invasive procedures, including lumbar puncture and removal of non-peripherally placed central lines during study.
• 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.
• Participant had known congenital or acquired bleeding diathesis.
• Participant required concomitant therapy with a strong P-glycoprotein inhibitor.
• Participant had previous history of any non-traumatic bleeding event that was life threatening or required medical attention.
• Participant had been administered thrombolytic therapy, or had undergone thrombectomy, or insertion of a caval filter to treat prior VTE.
• Participant had known inherited or acquired bleeding diathesis or coagulopathy.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Portola Pharmaceuticals, LLC (a wholly owned subsidiary of Alexion Pharmaceuticals): collaborator

Study Sites (20)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

ACTCA, Axis Clinical Trials

Los Angeles, California, 90036, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Tulane Medical Center

New Orleans, Louisiana, 70001, United States

Location

Rainbow Babies & Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Children's Hospital of Tatarstan Republic

Kazan', 420138, Russia

Location

Federal State Institution

Kemerovo, 650002, Russia

Location

Children's City Clinical Hospital

Moscow, 119049, Russia

Location

Pirogov Russian National Research Medical University

Moscow, 125412, Russia

Location

State Budgetary Institution

Nizhny Novgorod, 603136, Russia

Location

Saint Petersburg State Pediatric Medical University

Saint Petersburg, 194100, Russia

Location

Ivano-Frankivsk Regional Children Clinical Hospital

Ivano-Frankivsk, 76000, Ukraine

Location

Odessa Regional Children Clinical Hospital

Odesa, Ukraine

Location

Sumy Regional Children's Hospital

Sumy, Ukraine

Location

Vinnytsia Regional Children's Clinical Hospital, Department of Anesthesiology and Intensive Care

Vinnitsa, Ukraine

Location

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, B4 6NH, United Kingdom

Location

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

Children's Hospital for Wales

Cardiff, CF14 4XW, United Kingdom

Location

Glenfield Hospital

Leicester, LE3 9QP, United Kingdom

Location

Evelina London Children's Hospital

London, SE1 7EH, United Kingdom

Location

MeSH Terms

Interventions

betrixaban

Limitations and Caveats

After completion of Part 1 and prior to initiating Part 2, the Sponsor decided to cease developing betrixaban, prompting early study closure.

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2017
• First Posted: November 17, 2017
• Study Start: July 13, 2018
• Primary Completion: October 8, 2019
• Study Completion: October 8, 2019
• Last Updated: January 12, 2024
• Results First Posted: January 12, 2024

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

RU (6); US (5); UA (4); GB (5)