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    The EU Clinical Trials Register currently displays   37749   clinical trials with a EudraCT protocol, of which   6186   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2019-000679-18
    Sponsor's Protocol Code Number:EFC15467
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-04-30
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2019-000679-18
    A.3Full title of the trial
    ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B
    ATLAS-PEDS: Estudio abierto, multinacional de profilaxis con fitusiran en pacientes pediátricos varones de 1 a menos de 12 años de edad con hemofilia A o B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B
    Profilaxis con fitusiran en pacientes pediátricos varones de 1 a menos de 12 años de edad con hemofilia A o B. ATLAS-PEDS
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberEFC15467
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1223-4368
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/000/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenzyme Corporation
    B.1.3.4CountryUnited States
    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 supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi-Aventis, s.a.
    B.5.2Functional name of contact pointUnidad de Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street Addressc/ Josep Pla 2, 4ª planta
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.4Telephone number93 485 94 00
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1297; EU/3/14/1298
    D.3 Description of the IMP
    D.3.1Product nameFirusiran
    D.3.2Product code SAR439774
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFITUSIRAN
    D.3.9.1CAS number 1609016-97-8
    D.3.9.2Current sponsor codeSAR439774
    D.3.9.3Other descriptive nameALN-AT3SC
    D.3.9.4EV Substance CodeSUB190227
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Hemophilia A or B
    Hemofilia A o B
    E.1.1.1Medical condition in easily understood language
    Hemophilia A or B
    Hemofilia A o B
    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 LLT
    E.1.2Classification code 10066439
    E.1.2Term Hemophilia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B
    Confirmar los niveles de dosis adecuados de fitusiran cuando se administra a participantes pediátricos varones (de 1 a menos de 12 años de edad) que padecen hemofilia A o B grave.
    E.2.2Secondary objectives of the trial
    - To characterize the safety and tolerability
    - To characterize the pharmacokinetics (PK)
    - Caracterizar la seguridad y tolerabilidad
    - Caracterizar la farmacocinética (FC)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male, aged 1 to <12 years at the time of enrollment.
    - Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)
    - Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria:
    - Inhibitor titer of ≥0.6 BU/mL at screening, OR
    - Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
    - Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of anamnestic response.
    - Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
    - Weight requirements at the time of enrollment: 8 to <45 kg
    - Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the “parent”) and written or oral assent obtained from participant, per local and national requirements.
    - Hombres, de 1 a menos de 12 años en el momento de la selección
    - Hemofilia severa A o B (Factor VIII (FVIII) inferior a 1% o Factor IX (FIX) inferior o igual a 2%)
    - Los participantes deben tener anticuerpos inhibidores contra FVIII o FIX y deben cumplir uno de los siguientes criterios de resultados del ensayo Nijmegen-modified Bethesda:
    - Título de inhibidor de mayor o igual a 0.6 BU / mL en la selección, O
    - Título de inhibidor de menos de 0,6 BU / mL en la selección con evidencia de registro médico de 2 títulos consecutivos mayor o igual a 0,6 BU / mL, O
    - Título de inhibidor de menos de 0,6 BU / mL en el cribado con evidencia de registro clínico de respuesta anamnésica.
    - Acceso venoso periférico adecuado, según lo determine el investigador, para permitir las extracciones de sangre requeridas por el protocolo del estudio.
    - Requisitos de peso en el momento de la inscripción: 8 a menos de 45 kg.
    - Estar dispuesto y ser capaz de cumplir con los requisitos del estudio y proporcionar el consentimiento informado por escrito y firmado obtenido de los padres / tutor legal (en adelante, el "padre") y el consentimiento escrito u oral obtenido del participante, según los requisitos locales y nacionales.
    E.4Principal exclusion criteria
    - Known co-existing bleeding disorders other than hemophilia A or B
    - Antithrombin (AT) activity <60% at Screening
    - Co-existing thrombophilic disorder
    - Clinically significant liver disease
    - Active Hepatitis C virus infection
    - Acute or chronic Hepatitis B virus infection
    - Acute Hepatitis A or hepatitis E infection
    - HIV positive with a CD4 count of <400 cells/μL
    - History of arterial or venous thromboembolism, unrelated to an indwelling venous access
    - Inadequate renal function
    - History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
    - Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
    - History of intolerance to subcutaneous (SC) injection(s)
    - Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment
    - Trastornos hemorrágicos coexistentes conocidos distintos de la hemofilia A o B
    - Actividad de antitrombina (AT) inferior a 60% en la selección
    - Trastorno trombofílico coexistente
    - Enfermedad hepática clínicamente significativa.
    - Infección activa por el virus de la hepatitis C
    - Infección aguda o crónica por el virus de la hepatitis B
    - Infección aguda por hepatitis A o hepatitis E
    - VIH positivo con un recuento de CD4 de menos de 400 células / μL
    - Antecedentes de tromboembolismo arterial o venoso, no relacionado con un acceso venoso permanente.
    - Función renal inadecuada.
    - Historial de alergias a múltiples medicamentos o historial de reacción alérgica a un oligonucleótido o N-acetilgalactosamina (GalNAc)
    - Sujetos con catéteres permanentes centrales o periféricos, con antecedentes de complicaciones de acceso venoso que llevan a hospitalización y / o terapia de anticoagulación sistémica.
    - Historial de intolerancia a la inyección (s) subcutánea (SC)
    - Cualquier otra condición o comorbilidad que haga que el paciente no sea adecuado para la selección o que pueda interferir con la participación o la finalización del estudio, según el criterio del investigador.
    E.5 End points
    E.5.1Primary end point(s)
    Lowering of plasma antithrombin (AT) activity level; Lowering of plasma antithrombin (AT) activity level from Day 1 pre-fitusiran dose to Day 85
    Reducción del nivel de actividad de antitrombina (AT) plasmática desde el día 1 antes de la dosis de fitusiran hasta el día 85.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1 to Day 85
    Desde el día 1 hasta el día 85.
    E.5.2Secondary end point(s)
    1 - Number of participants reported with adverse events; Number of participants reported with treatment-emergent adverse events (TEAEs)
    2 - Pharmacokinetics (PK): Maximum plasma concentration (Cmax); Plasma samples will be collected for measurement of plasma concentrations of fitusiran such as Cmax.
    3 - Pharmacokinetics (PK): Time to reach maximum plasma concentration (Tmax); To evaluate time to reach Cmax
    4 - Pharmacokinetics (PK): Ctrough; To evaluate concentration observed just before investigational medicinal product (IMP) administration during repeated dosing (Ctrough)
    1 - Número de participantes que informan acontecimientos adversos (AA); Número de participantes que informan de acontecimientos adversos emergentes del tratamiento (AAET)
    2 - Farmacocinética (FC): concentración plasmática máxima (Cmax); Se recogerán muestras de plasma para medir las concentraciones plasmáticas de fitusiran, como la Cmax.
    3 - Farmacocinética (FC): tiempo para alcanzar la concentración plasmática máxima (Tmax); Evaluar el tiempo para alcanzar la Cmax.
    4 - Farmacocinética (FC): Ctrough; Para evaluar la concentración observada justo antes de la administración del medicamento en investigación (IMP) durante la dosificación repetida (Ctrough)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1 : 160 weeks
    2, 3, 4 : Day 1, Day 29, Day 57
    1: 160 semanas
    2, 3, 4: Día 1, Día 29, Día 57
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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 Yes
    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
    Última visita del último paciente
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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: 12
    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. of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 11
    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 No
    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. of subjects incapable of giving consent
    Legally minor patients, as defined by local regulation
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 12
    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)
    Participants completing the treatment period will be proposed to enroll in an extension study
    Los pacientes que completen el periodo de tratamiento serán propuestos para participar en un estudio de extensión.
    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 Decision2019-07-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-29
    P. End of Trial
    P.End of Trial StatusOngoing
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