Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43846   clinical trials with a EudraCT protocol, of which   7282   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2014-001054-42
    Sponsor's Protocol Code Number:CSL830_3002
    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:2015-01-05
    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 number2014-001054-42
    A.3Full title of the trial
    An open-label, randomized study to evaluate the long-term clinical safety and efficacy of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema
    Estudio abierto, randomizado para evaluar la seguridad y eficacia clínica a largo plazo 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 long-term clinical safety and efficacy of subcutaneously administered C1-esterase inhibitor in the prevention of hereditary angioedema
    Un estudio para evaluar la seguridad y eficacia clínica a largo plazo de la administración subcutánea del inhibidor de la esterasa C1 en el tratamiento profiláctico del angioedema hereditario
    A.4.1Sponsor's protocol code numberCSL830_3002
    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 Registration Coordinator
    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.5.4Telephone number34917088600
    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.3.9.2Current sponsor codeCSL830
    D.3.9.3Other descriptive nameCOMPLEMENT C1 ESTERASE INHIBITOR
    D.3.9.4EV Substance CodeSUB22696
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary Angioedema Types I and II
    Angioedema hereditario de tipo I y II
    E.1.1.1Medical condition in easily understood language
    HAE (type I and II) are genetic disorders that are associated with a deficiency in C1-INH.
    AEH (tipo I y II) es un trastorno genético asociado a una deficiencia de C1-INH
    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 17.1
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.
    Evaluar la seguridad clínica de la administración subcutánea de C1-INH en el tratamiento profiláctico a largo plazo del AEH
    E.2.2Secondary objectives of the trial
    -To further characterize the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.

    -To characterize the clinical efficacy of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.
    - Caracterizar adicionalmente la seguridad clínica de la administración subcutánea de C1-INH en el tratamiento profiláctico a largo plazo del AEH.

    - Caracterizar la eficacia clínica de la administración subcutánea de C1-INH en el tratamiento profiláctico a largo plazo del AEH.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    "-Males or females aged 6 years or older.
    -A confirmed diagnosis of HAE type I or II.
    -HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
    -For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit."
    - Hombres o mujeres de edad 6 años o más.
    - Un diagnóstico confirmado de AEH de tipo I o II.
    - Ataques de AEH durante cualquier período consecutivo de 2 meses que requieran tratamiento urgente o atención médica o que provocaron un deterioro funcional importante.
    - Pacientes que han que han recibido medicación por vía oral para la prevención de ataques de AEH durante los 3 meses anteriores a la primera visita del estudio.
    E.4Principal exclusion criteria
    "-Incurable malignancies.
    -Any clinical condition that will interfere with the evaluation of C1-INH therapy.
    -Clinically significant history of poor response to C1-esterase therapy for the management of HAE.
    -Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.
    -Inability to have HAE managed pharmacologically with on-demand treatment."
    - Neoplasias malignas incurables.
    - Cualquier cuadro clínico que pueda interferir con la evaluación del tratamiento con C1-INH.
    - Antecedentes clínicamente significativos de mala respuesta a la administración de C1-INH para el tratamiento del AEH.
    - Sospecha o diagnóstico confirmado de AEH adquirido o AEH con C1-INH normal.
    - AEH que no puede tratarse de forma adecuada con tratamiento farmacológico a demanda.
    E.5 End points
    E.5.1Primary end point(s)
    The person-time incidence rates of specified safety events.
    Densidades de incidencia de acontecimientos de seguridad específicos.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the treatment phase, up to 52 weeks.
    Durante la fase de tratamiento, hasta 52 semanas.
    E.5.2Secondary end point(s)
    - Percentage of subjects with SAEs or other specified safety events.
    - Percentage of C1-INH injections resulting in solicited AEs (injection site reactions).
    - Percentage of subjects with at least 1 solicited AE (injection site reaction).
    - Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus.
    - Percentage of subjects who experience < 1 HAE attack per 4-week period.
    - Percentage of subjects with a ? 50% reduction in the time-normalized number of HAE attacks.
    - El porcentaje de pacientes que experimentan AAG u otros acontecimientos específicos de seguridad.
    - El porcentaje de inyecciones de C1-INH que dieron lugar a AA locales solicitados (reacciones en el lugar de la inyección).
    - El porcentaje de pacientes que experimentan como mínimo 1 AA local solicitado (reacción en el lugar de la inyección).
    - El porcentaje de pacientes que dieron resultado positivo en la prueba del virus de inmunodeficiencia humana, el virus de la hepatitis B o el virus de la hepatitis C.
    - El porcentaje de pacientes que experimentan < 1 ataque de AEH en un período de 4 semanas.
    - El porcentaje de pacientes con una reducción de >= 50% del número de ataques de AEH normalizado en el tiempo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - During the treatment phase, up to 52 weeks.
    - During the treatment phase, up to 52 weeks.
    - During the treatment phase, up to 52 weeks.
    - From baseline through the treatment phase, up to 52 weeks.
    - During the treatment phase, up to 52 weeks.
    - From baseline through the treatment phase, up to 52 weeks.
    - Durante la fase de tratamiento, hasta 52 semanas.
    - Durante la fase de tratamiento, hasta 52 semanas.
    - Durante la fase de tratamiento, hasta 52 semanas.
    - Desde el inicio durante toda la fase de tratamiento, hasta 52 semanas.
    - Durante la fase de tratamiento, hasta 52 semanas.
    - Desde el inicio durante toda la fase de tratamiento, hasta 52 semanas.
    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 No
    E.8.1.1Randomised Yes
    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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Australia
    Canada
    European Union
    Israel
    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 years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    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: 11
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 9
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 88
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 11
    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 Yes
    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
    This clinical trial will be conducted in adults and also in children in different age groups including children younger than 12 years of age.
    El ensayo clínico se realizará en adultos y también en niños de diferentes grupos de edad, icluyendo a niños menores de 12 años de edad.
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 37
    F.4.2.2In the whole clinical trial 110
    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."
    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 Decision2015-02-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-09-21
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri Apr 19 16:55:46 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA