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    Summary
    EudraCT Number:2013-002453-29
    Sponsor's Protocol Code Number:0624-301
    National Competent Authority:Romania - National Agency for Medicines and Medical Devices
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-05-13
    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 ConcernedRomania - National Agency for Medicines and Medical Devices
    A.2EudraCT number2013-002453-29
    A.3Full title of the trial
    A PHASE 3, MULTICENTER, RANDOMIZED, SINGLE-BLIND, DOSE-RANGING, CROSSOVER STUDY TO EVALUATE THE SAFETY AND EFFICACY OF INTRAVENOUS ADMINISTRATION OF CINRYZE® (C1 ESTERASE INHIBITOR [HUMAN]) FOR THE PREVENTION OF ANGIOEDEMA ATTACKS IN CHILDREN 6 TO 11 YEARS OF AGE WITH HEREDITARY ANGIOEDEMA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A PHASE 3, MULTICENTER, RANDOMIZED, SINGLE-BLIND, DOSE-RANGING, CROSSOVER STUDY TO EVALUATE THE SAFETY AND EFFICACY OF INTRAVENOUS ADMINISTRATION OF CINRYZE® (C1 ESTERASE INHIBITOR [HUMAN]) FOR THE PREVENTION OF ANGIOEDEMA ATTACKS IN CHILDREN 6 TO 11 YEARS OF AGE WITH HEREDITARY ANGIOEDEMA
    A.4.1Sponsor's protocol code number0624-301
    A.5.4Other Identifiers
    Name:US IND Number:11838
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/164/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorViroPharma Incorporated
    B.1.3.4CountryUnited States
    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 supportViroPharma Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationViroPharma SPRL
    B.5.2Functional name of contact pointDanielle Tierens
    B.5.3 Address:
    B.5.3.1Street AddressRue Montoyer 47
    B.5.3.2Town/ cityBrussels
    B.5.3.3Post code1000
    B.5.3.4CountryBelgium
    B.5.4Telephone number+32 02 7470971
    B.5.5Fax number+32 02 7062421
    B.5.6E-maildanielle.tierens@viropharma.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 CINRYZE®
    D.2.1.1.2Name of the Marketing Authorisation holderViroPharma SPRL
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeVP 20624
    D.3.9.3Other descriptive nameCOMPLEMENT C1 ESTERASE INHIBITOR
    D.3.9.4EV Substance CodeSUB22696
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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
    Prevention of angioedema attacks in children 6 to 11 years of age with hereditary angioedema.
    E.1.1.1Medical condition in easily understood language
    Prevention of angioedema attacks in children 6 to 11 years of age with hereditary angioedema.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    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
    To assess the relative efficacy of two dose levels of CINRYZE (500 U and 1000 U) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age.
    E.2.2Secondary objectives of the trial
    SECONDARY OBJECTIVES
    - To assess the safety and tolerability of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age hereditary angioedema (HAE).
    - To characterize the pharmacokinetics (PK) and pharmacodinamics (PD) of CINRYZE administered by IV injection in children 6 to 11 years of age.
    - To assess the immunogenecity of CINRYZE following IV administration.

    OTHER OBJECTIVE
    - To assess the impact of treatment on health status (quality of life) in children 6 to 11 years of age with HAE.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be a child (male of female), ≥6 to < 12years of age at the time of screening.
    2. Have a confirmed diagnosis of Type I or Type II HAE and have a functional C1 inhibitor (C1 INH) level less than 50% of normal.
    3. Have a history of ≥ 1.0 moderate or severe angioedema attacks per month (average) during the 3 consecutive months prior to screening.
    4. Agree to adhere to the protocol-defined schedule of assessments and procedures.
    5. Have a parent(s)/legal guardian who is informed of the nature of the study provide written informed consent for the child to participate in the study before any study-specific procedures are performed (with assent from the child when appropriate).
    Additional Inclusion Criteria (Qualifying for Randomization):
    6. Have experienced ≥ 1.0 moderate or severe angioedema attacks per month (average) during the 12-week baseline observation period.
    E.4Principal exclusion criteria
    1. Have a history of hypercoagulability (abnormal blood clotting).
    2. Have a diagnosis of acquired angioedema or known to have C1 INH antibodies.
    3. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
    4. Be pregnant or breastfeeding.
    5. Have received an investigational drug other than dose required for prevention or treatment of angioedema attacks within 30 days prior to screening.
    6. Have, as determined by the Investigator and the Sponsor's medical monitor, any surgical or medical condition that could interfere with the administration of study drug or interpretation of study results.
    Additional Exclusion Criteria (Disqualifying from Randomization)
    7. Have any active infectious illness or fever defined as an oral temperature >38 oC (100.4 oF), tympanic >38.5 oC (101.3 oF), axillary >38 oC (100.4 oF), or rectal/core >38.5 oC(101.3 oF) within 24 hours prior to the first dose of study drug in Treatment Period 1.
    8. Have had signs or symptoms of an angioedema attack within 2 days prior to the first dose of study drug in Treatment Period 1.
    E.5 End points
    E.5.1Primary end point(s)
    The number of angioedema attacks normalized to a 12-week treatment period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    It will be measured continuously throughout the treatment period.
    E.5.2Secondary end point(s)
    SECONDARY EFFICACY ENDPOINTS
    - Cumulative Attack Severity. This score is the sum of the maximum symptom severity recorded for each angioedema attack in a treatment period.
    - Cumulative Daily Severity. This score is the sum of the severity scores recorded for every day of reported symptoms in a treatment period.
    - Time (measured in days from the first dose of study drug in a treatment period) to the first angioedema attack reported in that treatment period.
    - Change from pre- to post-dose in C1 INH functional activity, C1 INH antigen, and C4 levels.
    - Number of angioedema attacks requiring acute treatment during each treatment period.
    SAFETY ENDPOINTS
    Safety and tolerability, including:
    - adverse events by dose group
    - adverse events by exposure (dose normalized to body weight [U/kg]
    - adverse events by time of onset (e.g. during administration of study drug or within 24 hours after the end of injection of study drug).
    PK/PD ENDPOINTS
    Concentrations of C1 INH antigen, functional C1 INH activity, and complement C4 for individual subjects will be determined using validated analytical methods. Results will be summarized using descriptive statistics for values at each time point and for change from baseline at each post-injection time.

    OTHER ENDPOINTS
    Results of the EQ-5D-Y health status questionnaire will be presented in accordance with the EQ-5D-3L User Guide (version 4.0) and adaptd as appropriate for the youth version.
    E.5.2.1Timepoint(s) of evaluation of this end point
    •Efficacy endpoints: continuously throughout the treatment period
    •Safety endpoints: continuously throughout the treatment period
    The timepoints of blood sampling for determination of the PK/PD endpoints are specified in Schedule 5 on page 22 of the protocol.

    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 Yes
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    500U Cinryze then 1000U Cinryze will be compared with 1000U Cinryze then 500U Cinryze
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Germany
    Italy
    Mexico
    Romania
    Spain
    United Kingdom
    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 months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 12
    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: 12
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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
    CHILDREN 6 TO 11 YEARS
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 12
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-07-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-05-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-05-04
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