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    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2018-002562-40
    Sponsor's Protocol Code Number:16-021
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2019-10-31
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-002562-40
    A.3Full title of the trial
    A phase 1, open-label, single dose, non-randomized study to evaluate pharmacokinetics, pharmacodynamics, and safety of Betrixaban in pediatric patients.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An early stage study looking at how much betrixaban gets into the blood of young people after they take a single capsule, and to see if it is safe to do so.
    A.3.2Name or abbreviated title of the trial where available
    Protocol 16-021 – Pediatric PK
    A.4.1Sponsor's protocol code number16-021
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03346083
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/352/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPortola Pharma UK Ltd
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPortola Pharmaceuticals, Inc
    B.5.2Functional name of contact pointNancy Vinh Executive Director, Clin
    B.5.3 Address:
    B.5.3.1Street Address270 East Grand Avenue
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post code94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016502467000
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name BEVYXXA 40 mg
    D.2.1.1.2Name of the Marketing Authorisation holderPortola Pharmaceuticals, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBEVYXXA 40 mg
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBetrixaban
    D.3.9.1CAS number 330942-05-7
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name BEVYXXA 80 mg
    D.2.1.1.2Name of the Marketing Authorisation holderPortola Pharmaceuticals, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBEVYXXA 80 mg
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBetrixaban
    D.3.9.1CAS number 330942-05-7
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pediatric patients who are assessed to be at risk for Venous thromboembolism (VTE) but does not require immediate anticoagulant therapy, for example:
    a. Has previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
    b. Has any stable disease with a risk for arterial or venous thrombotic disorder, or
    c. Has any functional CVAD (Central Venous Access Device) in the upper or lower venous system.
    E.1.1.1Medical condition in easily understood language
    Young people who are at increased risk of having a blood clot.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10053468
    E.1.2Term Anticoagulant therapy
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The purpose of this study is to find out how much betrixaban gets into the blood stream when a single, oral dose is taken by children and teenagers up to 18 years of age.
    E.2.2Secondary objectives of the trial
    The secondary question of this study is what happens to betrixaban when it gets into the blood stream when a single, oral dose is taken by children and teenagers up to 18 years of age, and whether it is safe for them to take a single dose of betrixaban.

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Pediatric patients in the following age categories: 12 to < 18 years of age and 2 to < 12 years of age. Part 1 of the study will enroll only adolescent patients 12 to < 18 years of age.
    2. Patient is a pediatric subject who is assessed to be at risk for VTE but does not require immediate anticoagulant therapy, for example:
    a. Has previous thrombosis and completed a course of anti-coagulant therapy, and is considered to have a risk for recurrence of VTE, or
    b. Has any stable disease with a risk for arterial or venous thrombotic disorder, or
    c. Has any functional CVAD (Central Venous Access Device) in the upper or lower venous system.
    3. Patient must be able to have regular intake of a small amount of food (e.g., ≥ 100 mL) and eat before taking study medication, when instructed.
    4. Patient has normalized coagulation parameters (INR or PTT, as appropriate) within 7 days of study drug administration.
    5. Patient is sexually abstinent or, if not sexually abstinent, agrees to use an approved method of contraception (if applicable).
    6. Patient has adequate venous access to allow for blood sampling.
    7. Patient has provided Assent and responsible parent or guardian has signed Informed Consent.
    E.4Principal exclusion criteria
    1. Patient received any dose of anticoagulant therapy within 7 days of Day 1.
    2. Patient has active bleeding or has a comorbid disorder that places the patient at high risk for bleeding, including a positive fecal occult blood test or hematuria within 30 days prior to Day -1.
    3. Patient has a comorbid disorder that places the patient at risk of death within 90 days of enrollment.
    4. Patient has abnormal coagulation tests at baseline (within 3 days of Day -1).
    5. Patient has had recent or planned invasive procedures, including lumbar puncture and removal of non-peripherally placed central lines during study.
    6. Patient has 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 × 109/L or hemoglobin < 10.0 mg/dL.
     Hypertension.
    7. Patient requires concomitant therapy with a strong P-gp inhibitor.
    8. Patient has previous history of any non-traumatic bleeding event that was life threatening or required medical attention.
    9. Patient had been administered thrombolytic therapy, or had undergone thrombectomy, or insertion of a caval filter to treat prior VTE.
    10. Patient has known inherited or acquired bleeding diathesis or coagulopathy.
    11. Patient has abnormal QTc interval on baseline ECG.
    12. Patient will/has receive(d) a dose of any antiplatelet medication (including aspirin) within 14 days before study drug dosing. Planned or anticipated use of antiplatelet medication (including aspirin) in the first 24 hours following dosing shall exclude the patient.
    13. Patient has malabsorption disorders (e.g., cystic fibrosis or short bowel syndrome).
    14. Patient has an estimated Glomerular Filtration Rate (eGFR) < 30 mL/min.
    15. Patient is unable or reluctant to cooperate with the study procedures.
    16. Patient has hypersensitivity to other Factor Xa inhibitors, or the components of the dosage form.
    17. Patient has participated in a study with an investigational drug or medical device within 30 days prior to administration of betrixaban.
    18. Patient is female and of childbearing potential and is either pregnant or breastfeeding a child.
    19. Patient is sexually active and is not using medically accepted contraceptive method (if applicable).
    20. Patient has positive Drugs of Abuse urine test at Screening (Part 1 subjects only) at Screening excluding medications prescribed by a physician during the current hospital stay.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Pharmacokinetic Parameters
    The primary PK parameters to be determined are as follows:
    • Area under the concentration-time curve to the last measurable concentration above the quantitation limit (AUC(0-last)) per age group except for individuals with rich PK sampling
    • Maximum observed plasma concentration (Cmax)
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the first 24 hours, and 120 hours post dosing.
    E.5.2Secondary end point(s)
    The secondary objectives are as follows:
    • To assess the PD activity of betrixaban in pediatric patients in the following categories: 12 to < 18 years of age, and 2 to < 12 years of age.
    • To assess the safety of betrixaban in the pediatric patients in the following categories: 12 to < 18 years of age, and 2 to < 12 years of age.
    E.5.2.1Timepoint(s) of evaluation of this end point
    During the first 24 hours, and 120 hours post dosing.
    In addition safety will be evaluated also 7 days after dosing.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase 1 in pediatric population not involving healthy volunteers (previously studied in adults).
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA4
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Russian Federation
    Ukraine
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days18
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days19
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 33
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 11
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 21
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 33
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    This is single-dose PK/PD study. Single dose of betrixaban which is given to patients can not be considered as initiation of treatment. Furthermore, patients who require anti-coagulation therapy are not included in the study.
    One safety follow-up phone call/contact to the patient (if appropriate), parent, or legal guardian is planned according to protocol on Day 7 after the dosing.
    After end of the study patients will be treated according to local routine practice.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-11-26
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-08-05
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