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    Summary
    EudraCT Number:2020-003918-12
    Sponsor's Protocol Code Number:CSL312_3002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-06-08
    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 number2020-003918-12
    A.3Full title of the trial
    An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
    Studio in aperto volto a valutare la sicurezza e l’efficacia a lungo termine di CSL312 (garadacimab) nel trattamento profilattico dell’angioedema ereditario
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term safety and efficacy of CSL312 (garadacimab) in the prophylactic
    treatment of hereditary angioedema attacks
    Studio in aperto volto a valutare la sicurezza e l’efficacia a lungo termine di CSL312 (garadacimab) nel trattamento profilattico dell’angioedema ereditario
    A.3.2Name or abbreviated title of the trial where available
    CSL312_3002
    CSL312_3002
    A.4.1Sponsor's protocol code numberCSL312_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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring LLC
    B.5.2Functional name of contact pointTrial Registration Coordinator
    B.5.3 Address:
    B.5.3.1Street Address1020 First Avenue
    B.5.3.2Town/ cityKing of Prussia
    B.5.3.3Post codePA19406
    B.5.3.4CountryUnited States
    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 nameFactor XIIa antagonist monoclonal antibody
    D.3.2Product code [CSL312 garadacimab]
    D.3.4Pharmaceutical form Concentrate 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 INNFXIIa inhibitor monoclonal antibody
    D.3.9.2Current sponsor codeCSL312
    D.3.9.3Other descriptive namegaradacimab
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number170
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Yes
    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 Yes
    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
    Angioedema ereditario
    E.1.1.1Medical condition in easily understood language
    Hereditary angioedema (HAE) is a condition characterized by painful, recurring attacks of swelling in parts of the body including the face,throat, hands, feet, abdomen.
    L'angioedema ereditario (HAE) è una condizione caratterizzata da dolore,attacchi ricorrenti di gonfiore in parti del corpo compreso il viso, gola, mani, piedi, addome.
    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 23.1
    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
    The primary objective of the study is to evaluate the long-term safety of SC administration of CSL312 in the prophylactic treatment of subjects with C1-INH HAE.
    L'obiettivo principale dello studio è valutare la sicurezza a lungo termine della somministrazione sottocutanea di CSL312 nel trattamento profilattico dei soggetti con AE C1-INH.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the long-term efficacy, safety and patient reported assessment of response to therapy.
    Gli obiettivi secondari di questo studio sono valutare l'efficacia a lungo termine, la sicurezza e la valutazione della risposta alla terapia riferita dal paziente.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    "1. Males and females aged = 12 years
    2. Diagnosed with clinically confirmed C1-INH HAE
    3. Experienced = 3 HAE attacks during the 3 months before Screening
    4. Participated in the Run-in Period for at least 1 month (CSL312-naïve
    subjects only)
    5. Experienced at least an average of 1 HAE attack per month during the
    Run-in Period"
    1. Maschi e femmine di età = 12 anni
    2. Diagnosi di AE C1-INH clinicamente confermata
    3. Esperto = 3 attacchi di HAE durante i 3 mesi prima dello screening
    4. Ha partecipato al periodo di run-in per almeno 1 mese (CSL312-naïve
    solo soggetti)
    5. Ha subito almeno una media di 1 attacco HAE al mese durante il
    Run-in Period "
    E.4Principal exclusion criteria
    "1. Concomitant diagnosis of another form of angioedema, such as
    idiopathic or acquired angioedema or recurrent angioedema associated
    with urticaria
    2. Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period
    3. Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3
    months before the first day of the Run-in Period.
    4. Female subjects use estrogen-containing oral contraceptives or
    hormone replacement therapy within 4 weeks prior to screening
    5. Female or male subjects who are fertile and sexually active not using
    or not willing to use an acceptable method of contraception to avoid
    pregnancy during the study and for 30 days after receipt of the last dose
    of CSL312
    6. Pregnant, breastfeeding, or not willing to cease breastfeeding"
    1. Diagnosi concomitante di un'altra forma di angioedema, come angioedema idiopatico o acquisito o angioedema ricorrente associato
    con orticaria
    2. Uso di prodotti C1-INH, androgeni, antifibrinolitici o altri farmaci a piccole molecole per la profilassi di routine contro gli attacchi di HAE almeno 2 settimane prima del primo giorno del periodo di run-in Period
    3. Uso di anticorpi monoclonali come lanadelumab (Takhzyro®) 3mesi prima del primo giorno del periodo di rodaggio.
    4. I soggetti di sesso femminile usano contraccettivi orali contenenti estrogeni o terapia ormonale sostitutiva entro 4 settimane prima dello screening
    5. Soggetti di sesso femminile o maschile che sono fertili e sessualmente attivi non utilizzano o non disposto a utilizzare un metodo contraccettivo accettabile per evitare
    gravidanza durante lo studio e per 30 giorni dopo aver ricevuto l'ultima dose
    di CSL312
    6. Incinta, allattamento o non disposta a cessare l'allattamento al seno
    E.5 End points
    E.5.1Primary end point(s)
    1. Number of subjects with treatment emergent adverse events (TEAEs)
    2. Percentage of subjects with TEAEs
    3. TEAEs rates per injection
    4. TEAEs rates per subject year
    1. Numero di soggetti con eventi avversi emergenti dal trattamento (TEAE)
    2. Percentuale di soggetti con TEAE
    3. Tariffe TEAE per iniezione
    4. Tariffe TEAE per anno di disciplina
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 32 months
    2. Up to 32 months
    3. Up to 32 months
    4. Up to 32 months
    1. Fino a 32 mesi
    2. Fino a 32 mesi
    3. Fino a 32 mesi
    4. Fino a 32 mesi
    E.5.2Secondary end point(s)
    1. The time-normalized number (per month and year) of Hereditary
    Angioedema (HAE attacks) for the entire study
    2. The percentage reduction and the number of subjects experiencing at
    least = 50% = 70%, = 90 or equal to 100% (attack free) reduction in
    the time-normalized number of HAE attacks on Treatment compared to
    Run-in Period
    3. The time-normalized number (per month and year) of HAE attacks
    requiring on-demand treatment in subjects on treatment
    4. The time-normalized number (per month and year) of moderate
    and/or severe HAE attacks in subjects on treatment
    5. Number and percentage of subjects rating their response to therapy
    as good or excellent
    6. The number and percentage of subjects experiencingTEAEs with
    CSL312 or while on placebo
    7. The number and percentage of subjects experiencing adverse events
    of special interest (AESIs) with CSL312 or while on placebo
    8. The number and percentage of subjects experiencing TEAEs for naïve
    C1-INH subjects only with CSL312 or while on placebo
    9. The number and percentage of subjects experiencing serious adverse
    events (SAEs), including deaths, with CSL312 or while on placebo
    10. The number and percentage of subjects experiencing CSL312
    induced anti-CSL312 antibodies with CSL312 or while on placebo
    1. Il numero normalizzato nel tempo (per mese e anno) di Ereditari Angioedema (attacchi di HAE) per l'intero studio
    2. La riduzione percentuale e il numero di soggetti che sperimentano a minimo = 50% = 70%, = 90 o uguale al 100% (senza attacco) riduzione di
    il numero normalizzato nel tempo di attacchi di HAE al trattamento rispetto al Rui-in Period
    3. Il numero normalizzato nel tempo (per mese e anno) di attacchi HAE che richiedono un trattamento su richiesta nei soggetti in trattamento
    4. Il numero normalizzato nel tempo (per mese e anno) di moderato e / o gravi attacchi di HAE nei soggetti in trattamento
    5. Numero e percentuale di soggetti che valutano la loro risposta alla terapia buono o eccellente
    6. Il numero e la percentuale di soggetti che hanno sperimentato TEAE CSL312 o durante il trattamento con placebo
    7. Il numero e la percentuale di soggetti che hanno manifestato eventi avversi
    di particolare interesse (AESI) con CSL312 o durante il trattamento con placebo
    8. Il numero e la percentuale di soggetti che hanno sperimentato TEAE per naïve Soggetti C1-INH solo con CSL312 o durante il trattamento con placebo
    9. Il numero e la percentuale di soggetti che hanno subito gravi eventi avversi (SAE), compresi i decessi, con CSL312 o durante il trattamento con placebo
    10. Il numero e la percentuale di soggetti che sperimentano CSL312 anticorpi anti-CSL312 indotti con CSL312 o durante il trattamento con placebo
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Up to 34 months
    2. Up to 34 months
    3. Up to 32 months
    4. Up to 32 months
    5. Up to 32 months
    6. Up to 32 months
    7. Up to 32 months
    8. Up to 32 months
    9. Up to 32 months
    10. Up to 32 months
    1. Fino a 34 mesi
    2. Fino a 34 mesi
    3. Fino a 32 mesi
    4. Fino a 32 mesi
    5. Fino a 32 mesi
    6. Fino a 32 mesi
    7. Fino a 32 mesi
    8. Fino a 32 mesi
    9. Fino a 32 mesi
    10. Fino a 32 mesi
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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 No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    In Aperto
    open
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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 Information not present in EudraCT
    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
    Hong Kong
    Israel
    Japan
    New Zealand
    Russian Federation
    Taiwan
    United States
    Germany
    Hungary
    Italy
    Netherlands
    Spain
    United Kingdom
    Czechia
    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
    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 months10
    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 months10
    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.1.1.1In 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: 110
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 150
    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)
    Standard of care
    Standard of care
    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 Decision2021-06-01
    N.Ethics Committee Opinion of the trial applicationWithdrawn
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-12
    P. End of Trial
    P.End of Trial Status
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