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    Summary
    EudraCT Number:2014-001054-42
    Sponsor's Protocol Code Number:CSL830_3002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-05-14
    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 ConcernedItaly - Italian Medicines Agency
    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
    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
    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
    Uno studio per valutare la sicurezza clinica a lungo termine e l'efficacia di somministrata per via sottocutanea inibitore della C1-esterasi nella prevenzione di angioedema ereditario
    A.3.2Name or abbreviated title of the trial where available
    not available
    not available
    A.4.1Sponsor's protocol code numberCSL830_3002
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U0000-0000-0000
    A.5.4Other Identifiers
    Name:n.d.Number:n.d.
    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 supportAzienda Farmaceutica: CSL 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 number496421394909
    B.5.5Fax number496421393172
    B.5.6E-mailSylvia.Herget@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.2.1.2Country 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.3.1Product nameC1-esterase inhibitor
    D.3.2Product code CSL830
    D.3.4Pharmaceutical form Powder and solution for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    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 Ereditario Tipo I e 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.
    AE (tipo I e II) sono disordini genetici associati ad una carenza di C1INH
    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 18.0
    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 assess the clinical safety of subcutaneously administered C1-INH in
    the long-term prophylactic treatment of HAE.
    valutare la sicurezza clinica di C1-INH somministrato per via sottocutanea nel trattamento profilattico a lungo termine di HAE.
    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."
    caratterizzare ulteriormente la sicurezza clinica di C1-INH somministrato per via sottocutanea nel trattamento profilattico a lungo termine di HAE.
    • caratterizzare l'efficacia clinica di C1-INH somministrato per via sottocutanea
    nel trattamento profilattico a lungo termine dell'HAE .
    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."
    maschi o femmine dai 6 anni o più grandi.
    • diagnosi confermata di HAE di tipo I o II
    • attacchi di angioedema ereditario nel corso di un periodo consecutivo di 2 mesi che ha richiesto
    trattamento intensivo, medico, o causato perdita significativa di valore funzionale.
    • soggetti che hanno utilizzato la terapia orale per la profilassi contro attacchi HAE entro 3 mesi dalla prima visita di studio: l'uso di un regime stabile
    entro 3 mesi dalla prima visita di studio. "
    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
    Tumori incurabili.
    - Qualsiasi condizione clinica che possa interferire con la valutazione della terapia C1-INH
    - storia clinicamentedi significativa di scarsa risposta alla terapia C1-esterasi per la gestione di HAE.
    • Sospetta o confermata la diagnosi di HAE acquisito o HAE con normale
    C1-INH.
    • Incapacità di avere HAE gestito farmacologicamente con on-demand
    trattamento
    E.5 End points
    E.5.1Primary end point(s)
    The person-time incidence rates of specified safety events.
    I tassi di incidenza persona-tempo di specificati eventi di sicurezza.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the treatment phase, up to 52 weeks
    Durante la fase di trattamento, fino a 52 settimane
    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
    - Percentuale di soggetti con effetti indesiderati gravi o altri eventi di sicurezza specificati. - Percentuale conseguente di iniezioni di C1-INH in reazioni avverse sollecitate (reazioni nel sito di iniezione). - Percentuale di soggetti con almeno 1 AE sollecitato (reazione al sito di iniezione). - Percentuale di soggetti che diventano sieropositivi al virus dell'immunodeficienza, dell'epatite B, o dell'epatite C. - Percentuale di soggetti che sperimentano <1 attacco di AE per un period di 4 settimane periodo. - Percentuale di soggetti con riduzione ≥ 50% nel tempo normalizzata del numero di attacchi di angioedema ereditario
    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 di trattamento, fino a 52 settimane. - Durante la fase di trattamento, fino a 52 settimane. - Durante la fase di trattamento, fino a 52 settimane. - Dalla linea di base attraverso la fase di trattamento, fino a 52 settimane. - Durante la fase di trattamento, fino a 52 settimane. - Dalla linea di base attraverso la fase di trattamento, fino a 52 settimane.
    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 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 concerned2
    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
    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
    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 days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    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: 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.
    Questo studio clinico sarà condotto in adulti e anche nei bambini di diversi gruppi di età compresi i bambini di età inferiore ai 12 anni.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    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."
    I pazienti che terminano la loro partecipazione allo studio possono tornare alla loro terapia standard in base al sistema sanitario.
    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 Decision2015-02-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-09-21
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