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    Summary
    EudraCT Number:2014-005401-20
    Sponsor's Protocol Code Number:CSLCT-BIO-03-97
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-01-30
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2014-005401-20
    A.3Full title of the trial
    An Open-label, Multi-centre Study to Assess the Efficacy and Safety of Biostate® in Patients With von Willebrand's Disease (VWD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label, Multi-centre Study to Assess the Efficacy and Safety of Biostate® in Patients With von Willebrand's Disease (VWD)
    A.4.1Sponsor's protocol code numberCSLCT-BIO-03-97
    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 Limited
    B.1.3.4CountryAustralia
    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 Limited
    B.4.2CountryAustralia
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring
    B.5.2Functional name of contact pointTrial Registration Co-ordinator
    B.5.3 Address:
    B.5.3.1Street Address45 Poplar Road
    B.5.3.2Town/ cityParkville
    B.5.3.3Post code3052
    B.5.3.4CountryAustralia
    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 Voncento, Biostate®
    D.2.1.1.2Name of the Marketing Authorisation holderCSL Behring GmbH
    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.3.1Product nameHuman coagulation Factor VIII / von Willebrand Factor
    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 IMPIntravenous 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
    Von Willebrand's disease (VWD)
    E.1.1.1Medical condition in easily understood language
    Von Willebrand's disease (VWD)
    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 17.1
    E.1.2Level PT
    E.1.2Classification code 10047715
    E.1.2Term Von Willebrand's disease
    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
    The primary objectives of this study are to evaluate the:
    • Efficacy and
    • Safety
    of Biostate® in the treatment of non-surgery bleeds, in the management of surgery procedures and prophylactic therapy in patients with VWD where
    1-deamino-8-D-arginine vasopressin/Desmopressin (DDAVP) treatment is deemed by the Investigator to be ineffective, inadequate, or contraindicated.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients who fulfill the following criteria are eligible for inclusion into this study:

    a) Patients with a diagnosis VWD who require prophylactic therapy, have a non-surgery bleed, or are undergoing surgery.

    b) Patients who would routinely receive a plasma derived FVIII/VWF product as treatment for their VWD.

    c) Patients over 3 years of age and ≥14 kg.
    Inclusion of children < 13 years of age into the surgical and non-surgical bleed arms of the study will be at the discretion of the Investigator. The Investigator will take into consideration the blood profile of the patient and the required blood draw volume, bearing in mind that 17 ml of blood
    is to be drawn prior to each administration of Biostate® and that in accordance with the National Institute of Health Clinical Centre Guidelines, a paediatric blood draw should not exceed 3ml/kg, or 7ml/kg in a 6 week period.

    d) Patients and/or their legally acceptable representative must give written informed consent to participate in the study and must understand the nature of the study and must be willing to comply with all protocol requirements.

    In addition, patients receiving prophylactic therapy or being managed for elective surgery must also fulfill the following criterion:

    e) Patients must have been vaccinated against hepatitis A and B (unless protective antibodies are present resulting from a previously experienced infection). Documentation of immunisation (or protective antibodies resulting from a previous infection) must be present at baseline.

    E.4Principal exclusion criteria
    The following patients are not eligible for inclusion into this study:

    a) Patients with a known history of adverse drug reactions to factor VIII/VWF concentrates.

    b) Patients with a known or suspected VWF or FVIII inhibitor.

    c) Patients who are currently taking non-steroidal anti-inflammatory drugs (NSAIDs) which in the opinion of the Investigator would affect the efficacy and safety outcomes of the study.

    d) Patients with evidence or a history within the previous 12 months of abuse of any drug substance, licit or illicit, which in the opinion of the Investigator may affect the ability of the patient to comply with all protocol requirements.

    e) Patients who have within 90 days prior to the study screening visit, participated in a clinical study or used an investigational compound (eg. a new chemical entity not registered for clinical use), or who are planning to enter such a different study during the study period.

    f) Patients who are suffering from an acute or chronic medical condition, other than VWD, which may, in the opinion of the Investigator, affect the conduct of the study.

    g) Patients with a positive pregnancy test.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Endpoints:
    • Assessment of haemostatic efficacy using a grading scale of excellent, good, moderate and none.
    • For non-surgery bleed/surgery procedures:
    - The plasma levels of FVIII:C, VWF:RCo, VWF:Ag and VWF:CB.
    - Blood transfusion requirements.
    - FVIII:C and VWF:RCo in IU/kg (per treatment) required to resolve the event.
    - The number of treatments required until resolution of the event.
    - Assessment of blood loss during any surgery procedures.
    • For prophylaxis therapy:
    - FVIII:C IU/kg and VWF:RCo IU/kg per month.
    - Number of spontaneous bleeding events.

    Safety Endpoints:
    • The nature and incidence of AEs.
    • The development of inhibitors to FVIII.
    • The use of concomitant medications.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 13 months
    E.5.2Secondary end point(s)
    not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    not applicable
    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) Yes
    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.8.3 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
    Australia
    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 years1
    E.8.9.2In all countries concerned by the trial months1
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 1
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 9
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 13
    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 No
    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
    Patients and/or their legally acceptable representative must give written informed consent to participate in the study and must understand the nature of the study and must be willing to comply with all protocol requirements.
    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 23
    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
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Australia
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