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    Summary
    EudraCT Number:2013-001056-35
    Sponsor's Protocol Code Number:TXA
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-09-29
    Trial 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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2013-001056-35
    A.3Full title of the trial
    The Effectiveness and Population Pharmacokinetics and Pharmacogenomics of a Reduced Dose of Tranexamic Acid for Craniosynostosis Surgery: A multicenter study. The TXA Study.
    Studio multicentrico sull’efficacia, farmacocinetica/farmacogenomica dello schema a dosaggio ridotto di acido tranexamico nella chirurgia riparativa della craniostenosi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Low dose tranexamic acid for craniosynostosis surgery
    Dose bassa dell'acido tranexamico per la chirurgia della craniostenosi
    A.3.2Name or abbreviated title of the trial where available
    Low dose tranexamic acid for craniosynostosis surgery
    Dose bassa dell'acido tranexamico per la chirurgia della craniostenosi
    A.4.1Sponsor's protocol code numberTXA
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorIRCCS Istituto Giannina Gaslini
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportIRCCS Istituto Giannina Gaslini
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS Istituto Giannina Gaslini
    B.5.2Functional name of contact pointEpidemiologia statistica e comitati
    B.5.3 Address:
    B.5.3.1Street AddressVia G.Gaslini
    B.5.3.2Town/ cityGenova
    B.5.3.3Post code16133
    B.5.3.4CountryItaly
    B.5.4Telephone number003901056363462
    B.5.5Fax number00390108981116
    B.5.6E-mailcomitatoetico@ospedale-gaslini.ge.it
    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 UGUROL
    D.2.1.1.2Name of the Marketing Authorisation holderRottapharm S.p.A.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTranexamic Acid
    D.3.9.3Other descriptive nameTRANEXAMIC ACID
    D.3.9.4EV Substance CodeSUB11214MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    craniosynostosis
    craniostenosi
    E.1.1.1Medical condition in easily understood language
    early cranial suture fusion
    prematura fusione delle suture craniche
    E.1.1.2Therapeutic area Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Anesthesia and Analgesia [E03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10049889
    E.1.2Term Craniosynostosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the pharmacodynamics (PD), i.e. the clinical effectiveness (efficacy, tolerability and safety) of two dosing regimens of TXA in the target pediatric population.
    Determinare l’efficacia (misurata attraverso il calcolo delle perdite ematiche e delle richieste di emoderivati) del regime infusionale di TXA a basso dosaggio.
    E.2.2Secondary objectives of the trial
    1. To determine the population PK (popPK) of two dosing regimens.
    2. To determine the PK/PD profiles of two TXA dosing regimens.
    3. To evaluate the two TXA dosing regimens’ tolerability and safety profiles.
    4. To explore the impact of PAI-1 gene polymorphism and levels on TXA clinical effects.
    • Determinare la farmacocinetica (PK) dell’ TXA nei lattanti e nei bambini con tale schema di dosaggio che è influenzata da fattori demografici (età, peso, funzione renale).
    • Determinare gli effetti del polimorfismo del gene dell’inibitore del fattore attivatore del plasminogeno (PAI-1)
    e dei livelli di PAI-1 sulla riduzione del sanguinamento e sulla richiesta di trasfusioni TXA mediata.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Infants and children, M or F, aged 3 months to 3 years, undergoing craniosynostosis repair, fronto-orbital advancement surgery and cranial remodeling surgery (i.e., total cavernal remodeling surgery);
    - Parents’/guardian written informed consent given before any study-related procedure not part of the subject’s normal medical care, with the understanding that consent may be withdrawn at any time without prejudice to child’s future medical care.
    Pazienti di età compresa tra i 3 mesi e i 3 anni sottoposti a interventi di craniostenosi, avanzamento fronto-orbitario e chirurgia di rimodellamento cranio
    E.4Principal exclusion criteria
    - Preexisting hematological abnormality (defined as a positive history of bleeding disorder or a known diagnosis of a genetic or acquired bleeding disorder);
    - Preexisting coagulation defect (defined as PT, PTT or INR >1.5 times normal or a n pre-existing genetic or acquired coagulation defect);
    - Preexisting hepatic, renal, vascular, ocular and/or metabolic disorder;
    - History of acetylsalicylate administration within the last 14 days;
    - History of NSAIDs administration with 2 days of the scheduled surgery date.
    • alterazioni ematologiche preesistenti
    • difetti della coagulazione genetici o acquisiti (PT, PTT, INR > 1.5 volte il valore normale)
    • disfunzione epatica, renale, vascolare, oculare o metabolica preesistente
    • Ingestione di acido acetil salicilico nelle 2 settimane precedenti l’intervento
    • Assunzione di NSAIDs nei 2 giorni precedenti l’intervento___
    E.5 End points
    E.5.1Primary end point(s)
    The primary EP is the estimated volume of blood lost as calculated using the formula: ERCVlost = ERCVpreop + ERCVtransfused - ERCVpostop
    (where ERCV = Estimated Red Cell Volume).
    • Perdite ematiche stimate calcolate usando la formula: ERCVlost = ERCVpreop + ERCVtransfused - ERCVpostop (ERCV = Estimated Red Cell Volume)
    E.5.1.1Timepoint(s) of evaluation of this end point
    end of surgery
    fine della chirurgia
    E.5.2Secondary end point(s)
    1. Pop-PK concentration-time curves of two dosing regimens of TXA in the target population as influenced by demographic covariates such as age, body weight and renal function.
    2. Concentration-effect relationships (PK/PD profiles) of two TXA dosing regimens to be evaluated and described and the appropriate statistical model to be fitted.
    3. Proportion of patients with treatment-emergent adverse events (AEs) – i.e., thromboembolic events (vascular occlusive, stroke), neurologic events (seizures), serious AEs (SAEs), and laboratory abnormalities up to 90 days after surgery.
    4. Effect of polymorphism of the PAI-1 gene and PAI-1 levels on TXA-mediated reduction in blood loss and transfusion requirement.
    Tempo chirurgico
    Totale volume di cristalloidi e colloidi somministrati
    Totale somministrazione di sangue ed emoderivati in sala operatoria e nel post operatorio,
    Diuresi totale
    E.5.2.1Timepoint(s) of evaluation of this end point
    end of surgery
    fine della chirurgia
    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 Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3.1Comparator description
    dose alta verso dose bassa
    high dose vs low dose
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA1
    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
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    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: 60
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 60
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    young children
    neonati e lattanti
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    None
    Nessuno
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-11-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-16
    P. End of Trial
    P.End of Trial StatusOngoing
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