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    Summary
    EudraCT Number:2019-003921-99
    Sponsor's Protocol Code Number:CSL830_3003
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-04-27
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2019-003921-99
    A.3Full title of the trial
    An open-label, single-arm, non-randomized phase 3 study to evaluate clinical efficacy, safety, and pharmacokinetics of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema in Japanese subjects
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Subcutaneous administration of human plasma-derived C1-esterase inhibitor for prevention of hereditary angioedema attacks in Japanese subjects
    A.4.1Sponsor's protocol code numberCSL830_3003
    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 K.K.
    B.1.3.4CountryJapan
    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 K.K.
    B.4.2CountryJapan
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring K.K.
    B.5.2Functional name of contact pointTrial Registration Coordinator
    B.5.3 Address:
    B.5.3.1Street Address1-2-3-Kita Aoyama, Minato-ku
    B.5.3.2Town/ cityTokyo
    B.5.3.3Post code107-0061
    B.5.3.4CountryJapan
    B.5.4Telephone number+1610878-4000
    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 Yes
    D.2.1.1.1Trade name Haegarda, Berinert
    D.2.1.1.2Name of the Marketing Authorisation holderCSL Behring
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 (C1-INH)
    D.3.2Product code CSL830
    D.3.4Pharmaceutical form Powder and solvent for solution for injection/infusion
    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 INNC1-INH
    D.3.9.3Other descriptive nameC1-INHIBITOR
    D.3.9.4EV Substance CodeSUB130159
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000
    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 angiodema (HAE) type I and II are genetic disorders that are associated with a deficiency in C1 esterase-inhibitor
    E.1.1.1Medical condition in easily understood language
    Hereditary angiodema type I and II
    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 20.0
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives of the study are to evaluate the clinical efficacy and PK of SC CSL830 in the prophylactic treatment of HAE in Japanese subjects
    E.2.2Secondary objectives of the trial
    • To further characterize the clinical efficacy of the SC CSL830
    • To demonstrate the safety and tolerability of SC CSL830
    • To evaluate C1-INH functional activity and C1-INH antigen (PK), and C4 antigen (pharmacodynamics [PD]) during CSL830 treatment
    • To evaluate subject-reported Angioedema Quality of Life (AEQoL) outcomes, and subject and investigator global assessments of response to therapy (SGART and IGART)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Males or females aged 12 years or older.
    • Japanese with a confirmed clinical diagnosis of HAE type I or II.
    • At least 4 HAE attacks over a consecutive 2-month period as documented in the subject’s medical records in the 3 months before the Screening Visit
    • C1-INH functional activity of less than 50% norm, AND C4 antigen concentration below the laboratory reference range
    • During their participation in the Run-In Period experienced ≥ 2 HAE attacks within any consecutive 4-week period, OR experienced ≥ 1 HAE attack during the first 2 weeks


    E.4Principal exclusion criteria
    • History of arterial or venous thrombosis requiring anticoagulant therapy, or current, clinically significant prothrombotic risk
    • Known malignancies at the time of the Screening Visit
    • Have a history of allergic reaction to C1-INH products, including Cinryze and Berinert P or other blood products
    • Unable to discontinue use of intravenous (IV) C1-INH for routine prophylaxis against HAE attacks by Day 1 of the Run-in Period or plans to use IV C1-INH for routine prophylaxis against HAE attacks by Day 1 of the Run-in Period
    • Assessed by the investigator as unable to have their HAE adequately managed with on-demand treatment, administered either independently or with assistance

    E.5 End points
    E.5.1Primary end point(s)
    -Time-normalized number of HAE attacks during treatment with CSL830.
    -C1-INH functional activity (PK: AUC0-tau, AUC0-last, Cmax, Tmax, T1/2) after the last dose of CSL830 Treatment.

    E.5.1.1Timepoint(s) of evaluation of this end point
    -Up to 14 weeks
    -Up to 11 days after the last dose
    E.5.2Secondary end point(s)
    -The percentage of subjects who achieved ≥ 90%, ≥ 70%, and ≥ 50% relative reduction of monthly HAE attack rate.
    -The relative reduction in the time-normalized number of rescue medication uses.
    -The number of reported adverse Events.
    -Percentage of subjects reporting adverse events (AEs) and injection site reactions that begin within 24 hours of CSL830 administration.
    -Mean trough C1-INH functional activity during treatment with CSL830.
    -Mean trough C1-INH and C4 antigens during treatment with CSL830.
    -Subject-reported Angioedema Quality of Life (AEQoL) outcome scores
    -Subject-reported Global Assessments of Reponse to Therapy outcomes (SGART)
    -Investigator-reported Global Assessments of Reponse to Therapy outcomes (IGART)


    E.5.2.1Timepoint(s) of evaluation of this end point
    -Up to 14 weeks
    -Up to 14 weeks
    -Up to 18 weeks
    -Up to 24 hours after dose
    -Up to 16 weeks
    -Up to 16 weeks
    -Up to 16 weeks
    -Up to 16 weeks
    -Up to 16 weeks





    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 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 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 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Japan
    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.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months12
    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: 1
    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: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 10
    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)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation HAEJ
    G.4.3.4Network Country Japan
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Japan
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