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    Summary
    EudraCT Number:2004-001186-17
    Sponsor's Protocol Code Number:CE1145_3001
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2006-02-02
    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 ConcernedSweden - MPA
    A.2EudraCT number2004-001186-17
    A.3Full title of the trial
    Human pasteurized C1 esterase inhibitor concentrate (CE1145) in subjects with congenital C1-INH deficiency and acute abdominal or facial HAE attacks
    A.3.2Name or abbreviated title of the trial where available
    C1-INH HAE study
    A.4.1Sponsor's protocol code numberCE1145_3001
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 organisation
    B.5.2Functional name of contact point
    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 Berinert P
    D.2.1.1.2Name of the Marketing Authorisation holderZLB Behring GmbH
    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 concentrate
    D.3.2Product code CE1145
    D.3.4Pharmaceutical form Powder and solvent for solution for infusion
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 codeCE1145
    D.3.9.3Other descriptive nameC1 esterase inhibitor concentrate, human, pasteurized
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number40 - to 62.5
    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) Information not present in EudraCT
    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 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 Information not present in EudraCT
    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 Information not present in EudraCT
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboIntravenous infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary angioedema (HAE) is characterized by congenital C1-INH deficiency. This is reflected by insufficient plasma concentrations of C1 – inhibitor or by synthesis of dysfunctional C1 – inhibitor molecules, which can lead to attacks of abdominal pain and/or severe swelling in the tissues e.g. of the face. Replacement with C1 esterase inhibitor concentrate is the treatment of choice of an acute attack of HAE.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10019860
    E.1.2Term Hereditary angioedema
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level PT
    E.1.2Classification code 10019860
    E.1.2Term Hereditary angioedema
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To show that pasteurized C1-INH concentrate (Berinert® P) shortens the time to onset of relief of symptoms of abdominal or facial HAE attack compared to placebo
    E.2.2Secondary objectives of the trial
    - To compare the efficacy of two different dosing schemes of Berinert® P in abdominal or facial attacks of edema due to HAE
    - To compare the safety of Berinert® P in subjects with HAE between the initial three treatment groups (Berinert® P 10 Units per kg b.w., Berinert® P 20 Units per kg b.w, placebo)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male and female subjects >= 6 years
    - Documented congenital C1-INH deficiency with immunogenic and/or functional C1-INH level below the lower limit of the laboratory reference range (HAE type I or II); to be confirmed by central laboratory.
    - Documented history of abdominal or facial attacks
    - Acute abdominal or facial attack with moderate /severe intensity for no longer than
    5.0 hours at the time of study medication administration
    - Informed written consent has been obtained (signed by subject, or, in case of a minor, by his/her legally acceptable representative(s))
    E.4Principal exclusion criteria
    - Life expectancy < 6 months
    - Incurable malignancies with metastases
    - History of hypersensitivity to the study medication
    - Acquired angioedema due to C1-INH deficiency (e. g., onset at age > 40 years, no family history of congenital C1-INH deficiency, low C1q level, and no known HAE mutation)
    - All other types of angioedema not associated with C1-INH deficiency
    (including e.g. anaphylactic angioedema)
    - Abdominal pain caused by other pathology (e. g., appendicitis, myocardial infarction)
    - End-stage liver disease (i.e., Child-Pugh-score B or C)
    - Fever (> 38.3°C or 101.0 F)
    - High white blood cell count (> 20.0 x 10 exp9/L )
    - Pregnant women (pregnancy test required), women currently breast-feeding, or with the intention to breast feed
    - Treatment with any other investigational drug (except IMP and any other drugs appropriate for the treatment of acute angioedema) 30 days before start of study treatment on Day 1.
    - Treatment with any C1-INH concentrate or any other drugs appropriate for the treatment of acute angioedema within 7 days before start of study treatment on Day 1.
    - Treatment with fresh frozen plasma or native plasma within 7 days before start of study treatment on Day 1.
    - Treatment with ACE inhibitors within 4 weeks before start of study treatment on Day 1.
    - Narcotic pain medication and/or anti-emetics between start of attack and administration of study medication.
    - Evidence of narcotic seeking behavior and/or drug addiction (including alcohol abuse)
    - Mental condition rendering the subject (or the subject’s legally acceptable representative(s)) unable to understand the nature, scope and possible consequences of the study
    - Prior inclusion into this study
    E.5 End points
    E.5.1Primary end point(s)
    Time to onset of relief from the evaluated (abdominal or facial) attack will be assessed by the investigator every 15 min for the first 2 hours, every 30 min for the next 2 hours, and at 5, 6, 7, 8, 12, 16, 20 and 24 hours after start of administration of the study medication.
    The investigator or a delegate will ask each subject at the above defined time points:

    1. Taking into account all of the symptoms you experienced with this HAE attack,
    are you confident that it is starting to improve?

    If the answer to Question 1 has been “yes” for two consecutive time points, asking Question 1 will stop. The time of onset of relief from attack is defined by the time determined at the first of the two consecutive “yes” responses.

    If rescue medication or non-permitted medication was administered before this time has been reached, the subject will be considered as a non-responder and the time to onset of relief will be set to a poor/failure outcome for the primary analysis.

    Subjects will not be discharged from hospital, before the relief from symptoms of the evaluated (abdominal or facial) attack has started, including both the time to reaching the primary endpoint and the time to relief after rescue medication, if applicable. In addition, the 4-hour sample for C1-INH and C4 determination is
    to be taken before discharge.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    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 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
    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
    Last visit of the last patient
    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 months9
    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 months9
    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) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 Yes
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.6.1Details of subjects incapable of giving consent
    Children >= 6 years
    F.3.3.7Others No
    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 32
    F.4.2.2In the whole clinical trial 84
    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 Decision2006-03-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2006-06-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2007-12-28
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