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    Summary
    EudraCT Number:2013-000916-10
    Sponsor's Protocol Code Number:CSL830_3001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-10-18
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2013-000916-10
    A.3Full title of the trial
    A double-blind, randomized, placebo-controlled, cross-over study to evaluate the clinical efficacy and safety of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema
    Estudio cruzado, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia clínica y la seguridad de la administración subcutánea del inhibidor de la esterasa C1 derivado de plasma humano en el tratamiento profiláctico del
    angioedema hereditario.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the clinical efficacy and safety of subcutaneously administered C1-esterase inhibitor in the prevention of hereditary angioedema.
    Estudio para evaluar la eficacia clínica y la seguridad de la administración subcutánea del inhibidor de la esterasa C1 en la prevención del angioedema hereditario.
    A.4.1Sponsor's protocol code numberCSL830_3001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01912456
    A.5.4Other Identifiers
    Name:IND NumberNumber:BB-IND 14992
    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 SponsorCSL Behring GmbH
    B.1.3.4CountryGermany
    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 supportCSL Behring GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring GmbH
    B.5.2Functional name of contact pointTrial Disclosure Manager
    B.5.3 Address:
    B.5.3.1Street AddressEmil-von-Behring-Str. 76
    B.5.3.2Town/ cityMarburg
    B.5.3.3Post code35041
    B.5.3.4CountryGermany
    B.5.6E-mailclinicaltrials@cslbehring.com
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameC1 esterase inhibitor
    D.3.2Product code CSL830
    D.3.4Pharmaceutical form Powder and solvent for 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 INNN/A
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeCSL830
    D.3.9.3Other descriptive nameC1 ESTERASE INHIBITOR
    D.3.9.4EV Substance CodeSUB22696
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1500
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    D.8.3Pharmaceutical form of the placeboPowder and solvent for solution for infusion
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary Angioedema (HAE) type I and II
    Angioedema hereditario (AEH) de tipo I y II
    E.1.1.1Medical condition in easily understood language
    HAE is a genetic disorder with 3 known types.Types I/II are associated with C1 esterase inhibitor deficiencies.Type III extremely rare is associated with normal C1 esterase inhibitor and disease state
    AEH es un trastorno genético con tres tipos. Tipos I/II con a una deficiencia del inhibidor de la esterasa C1. Tipo III raro y acociado a un inhibidor normal de esterasa C1 y estado de la enfermedad
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10019860
    E.1.2Term Hereditary angioedema
    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 demonstrate the clinical efficacy of SC CSL830 in the prophylactic treatment of HAE.To compare the clinical efficacy of 2 doses of SC
    CSL830.
    The comparison of the clinical efficacy of the 2 doses of CSL830 will only be done if the clinical efficacy of SC CSL830 in the prophylactic treatment of HAE is shown to be statistically significantly better than placebo.
    Demostrar la eficacia clínica de CSL830 s.c. en el tratamiento profiláctico del AEH.
    Comparar la eficacia clínica de dos dosis de CSL830 s.c. La comparación de la eficacia clínica de las 2 dosis de CSL830 se realizará únicamente si se ha visto que la eficacia clínica de CSL830 s.c. en el tratamiento profiláctico del AEH es significativamente mejor, desde el punto de vista estadístico, que la del placebo.
    E.2.2Secondary objectives of the trial
    To further characterize the clinical efficacy of the 2 doses of SC CSL830.
    To demonstrate the safety and tolerability of SC CSL830.
    Caracterizar adicionalmente la eficacia clínica de las 2 dosis de CSL830 s.c.
    Demostrar la seguridad y la tolerabilidad de CSL830 s.c.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Capable of providing written informed consent and willing and able to adhere to all protocol requirements, or the subject?s parent(s) or legally acceptable representative(s) capable of providing written informed consent.
    - Male or female
    - Aged 12 years or older at the time of providing written informed consent.
    - Clinical diagnosis of HAE (type I or II), which will be confirmed by central laboratory testing before inclusion into Treatment Period 1
    - HAE attacks over a consecutive 2-month period that required acute treatment, medical attention
    - Investigator believes that the subject is willing and able to adhere to all protocol requirements.
    - Assessed by the investigator as able to appropriately store study medication and is capable of being trained to administer study medication (by the subject or carer) outside of the study center setting.
    -Capacidad para otorgar su consentimiento informado por escrito y voluntad y capacidad para cumplir todos los requisitos del protocolo, o capacidad por parte de progenitores o representantes legales del paciente para otorgar su consentimiento informado por escrito. -Hombre o mujer. -12 años o más de edad en el momento de otorgar el consentimiento informado por escrito. -Diagnóstico clínico de AEH (de tipo I o II), que será confirmado mediante análisis del laboratorio central antes de su inclusión en el Período de tratamiento 1. -Ataques de AEH dentro de un período de 2 meses consecutivos que requirieran tratamiento urgente o atención médica. -El
    investigador cree que el paciente quiere y es capaz de cumplir todos los requisitos del protocolo. -El investigador considera al paciente capaz de conservar adecuadamente el medicamento del estudio y capaz de ser instruido para administrar el medicamento del estudio (bien el paciente o su cuidador) fuera del centro de estudio.
    E.4Principal exclusion criteria
    - History of clinically significant arterial or venous thrombosis, or a current history of a clinically significant prothrombotic risk.
    - Known incurable malignancies at the time of the Screening visit.
    - Any clinical condition that is likely to interfere with evaluation of CSL830 or satisfactory conduct of the study.
    - A clinically significant history of poor response to C1-INH therapy for the management of HAE.
    - Female subject of childbearing potential either not using or not willing to
    use a medically reliable method of contraception or not sexually abstinent during the study, or not surgically sterile.
    - Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (ie, estrogen / progesterone containing products) within 3 months before the Screening visit.
    - Intention to become pregnant during the course of the study.
    - Pregnancy or nursing mother
    - Participated in another interventional clinical study within 30 days before Screening or at any time during the study.
    - Alcohol, drug or medication abuse within 1 year before Screening.
    - Currently receiving a therapy not permitted during the study
    - Mental condition rendering the subject (or the subject?s legally acceptable representative[s]) unable to understand the nature, scope and possible consequences of the study.
    - Known or suspected hypersensitivity to the investigational product, or to any excipients of the investigational product.
    - Previously randomized or participated in the Run-in Period of the current study.
    - Employee at the study site, or spouse / partner or relative of the investigator or subinvestigators.
    -Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.
    -Antecedentes de trombosis arterial o venosa clínicamente significativa, o presencia actual de un riesgo protrombótico clínicamente significativo. -Neoplasias malignas incurables conocidas en el momento de la visita de Selección. -Cualquier cuadro clínico que pueda interferir con la evaluación de CSL830 o con la correcta realización del estudio. -Antecedentes clínicamente significativos de mala respuesta a la administración de C1-INH para el tratamiento del AEH. -Mujer en edad fértil y no esterilizada quirúrgicamente que no esté utilizando y no esté dispuesta a utilizar un método anticonceptivo médicamente fiable o abstenerse de mantener relaciones sexuales durante el estudio. -Mujer que haya empezado a tomar o haya cambiado la dosis de cualquier anticonceptivo hormonal o tratamiento de sustitución hormonal (es decir, productos que contentan estrógenos / progesterona) dentro de los 3 meses previos a la visita de Selección. -Mujer con intención de quedarse
    embarazada en el transcurso del estudio. -Mujer embarazada o en periodo de lactancia. -Participación en otro estudio clínico intervencionista dentro de los 30 días previos a la Selección o en cualquier momento durante el estudio. -Abuso de alcohol, drogas o medicamentos en el año previo a la Selección. -Estar recibiendo
    actualmente algún tratamiento no permitido durante el estudio. -Trastorno mental que impida que el paciente (o sus representantes legales) pueda entender la naturaleza, el alcance y las posibles consecuencias del estudio. -Hipersensibilidad
    conocida o presunta al producto en investigación o a cualquiera de los excipientes del producto en investigación. -Aleatorización o participación previa en el Período de preinclusión del presente estudio. -Ser empleado del centro de estudio, o cónyuge / pareja o familiar del investigador o los subinvestigadores. -Cualquier otro
    motivo que, en opinión del investigador, hiciera al paciente no apto para participar en el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    - The time-normalized number of HAE attacks when treated with
    CSL830 and when treated with placebo.
    - The time-normalized number of HAE attacks between placebo and
    the respective CSL830 dose for the 2 doses of CSL830.
    -El número normalizado con respecto al tiempo de ataques de AEH cuando se trata con CSL830 y cuando se trata con placebo. -El número normalizado con respecto al tiempo de ataques de AEH entre placebo y la respectiva dosis de CSL830 para las 2 dosis de CSL830.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The evaluation period will be from the start of Week 3 to the
    time point when the subject completes the treatment period (ie, the total assessment period will be up to 14 weeks duration). This is to account for a wash-in / wash-out period of 2 weeks at the start of each treatment period.
    El período de evaluación irá desde el comienzo de la Semana 3 hasta el momento en el que el paciente complete el período de tratamiento (es decir, el período total de evaluación tendrá una duración de hasta 14 semanas). Eso tiene en cuenta un período de lavado de 2 semanas al inicio de cada período de tratamiento.
    E.5.2Secondary end point(s)
    The proportion of ?responders? in the 2 CSL830 dose groups, when defined by the following criteria:
    - 50% relative reduction in the time-normalized number of HAE attacks during treatment with CSL830 compared with placebo.
    - The time-normalized number of uses of rescue medication
    when treated with CSL830 and when treated with placebo.
    The secondary safety endpoint is the proportion of subjects with any adverse events (AEs) that begin during or within 24 hours of administration of 40 IU/kg CSL830, 60 IU/kg CSL830 and placebo.
    La proporción de «pacientes que responden al tratamiento» en los 2 grupos de dosis de CSL830, cuando éstos vienen definidos por los criterios siguientes: -Un 50 % de reducción relativa en el número normalizado con respecto al tiempo de ataques de AEH durante el tratamiento con CSL830 en comparación con el placebo. -El número normalizado con respecto al tiempo de usos de medicación de rescate cuando se trata con CSL830 y cuando se trata con placebo. El criterio secundario de valoración de la seguridad es la proporción de pacientes con cualquier acontecimiento adverso (AA) surgido durante o dentro de las 24 horas siguientes a la administración de 40 UI/kg de CSL830, 60 UI/kg de CSL830 y placebo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The evaluation period for these assessments will start at the beginning of each treatment period and end at Week 14 (Day 92) of each treatment period, or at the time of early escape.
    El período contemplado para obtener tales valoraciones comenzará al inicio de cada período de tratamiento y finalizará en la Semana 14 (Día 92) de cada período de tratamiento, o en el momento de la retirada prematura.
    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 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) 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 Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA43
    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
    Argentina
    Australia
    Canada
    European Union
    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
    The scheduled end of study participation for an individual subject occurs:
    1) 1 week aftercompletion of Treatment Period 2 Week 16 (Day 112)
    2) 1 week after early exit from Treatment Period 2
    3) if a subject is withdrawn early from the study
    El fin de la participación de un paciente concreto en el estudio está previsto que se produzca: 1) 1 semana después de completar la Semana 16 (Día 112) del Período de tratamiento 2 2) 1 semana después de su salida prematura del Período de tratamiento 2 3) si el paciente es retirado prematuramente del estudio
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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: 20
    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) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 100
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 100
    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)
    Patients who end their participation in the trial can return to standard therapy according to their health care scheme.
    Los pacientes que terminen su participación en el ensayo pueden volver a la terapia estándar de acuerdo a su programa de salud.
    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 Decision2014-01-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-10-12
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