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    Summary
    EudraCT Number:2016-002510-50
    Sponsor's Protocol Code Number:Biostate_4001
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-09-21
    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 ConcernedGermany - PEI
    A.2EudraCT number2016-002510-50
    A.3Full title of the trial
    A low-interventional Multicentre Post-Authorisation Safety Study for Voncento/Biostate/Aleviate for Routine Prophylaxis, Treatment of Bleeding Events and/or Surgery in Male Subjects with Haemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A low-interventional Multicentre Post-Authorisation Safety Study for Voncento/Biostate/Aleviate for Routine Prophylaxis, Treatment of Bleeding Events and/or Surgery in Male Subjects with Haemophilia A
    A.4.1Sponsor's protocol code numberBiostate_4001
    A.5.4Other Identifiers
    Name:EU PAS Register ReferenceNumber:EUPAS19806
    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 supportCSL Behring GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring GmbH
    B.5.2Functional name of contact pointKerstin Jung,Clinical Study Manager
    B.5.3 Address:
    B.5.3.1Street AddressEmil-von-Behring-Strasse 76
    B.5.3.2Town/ cityMarburg
    B.5.3.3Post code35041
    B.5.3.4CountryGermany
    B.5.4Telephone number49 0 6421 39 2930
    B.5.5Fax number49 0 6421 39 3172
    B.5.6E-mailKerstin.Jung@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
    D.2.1.1.2Name of the Marketing Authorisation holderCSL 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 nameVoncento
    D.3.4Pharmaceutical form Powder and solution 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.3.8INN - Proposed INNHuman Coagulation Factor VIII
    D.3.9.3Other descriptive nameHUMAN COAGULATION FACTOR VIII
    D.3.9.4EV Substance CodeSUB78203
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number250 to 1000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman Von Willebrand Factor
    D.3.9.3Other descriptive nameHUMAN VON WILLEBRAND FACTOR
    D.3.9.4EV Substance CodeSUB22288
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number600 to 2400
    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
    Haemophilia A
    E.1.1.1Medical condition in easily understood language
    Haemophilia A is a hereditary bleeding disorder due to a deficiency of functional plasma coagulation FVIII
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10060612
    E.1.2Term Hemophilia A
    E.1.2System Organ Class 100000011915
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the immunogenicity of Voncento in Previously Treated Patients (PTPs) with haemophilia A.
    E.2.2Secondary objectives of the trial
    To investigate the long-term safety of Voncento in previously treated patients with haemophilia A.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects meeting all of the following inclusion criteria may be enrolled into the study:
    1. Capable of providing written informed consent and willing and able to adhere to all protocol requirements and / or the subject’s parent(s) or legally acceptable representative(s) capable of providing written informed consent.
    2. Male subjects of any age.
    3. Diagnosis of haemophilia A with plasma FVIII levels <2% documented in medical records
    4. No history of FVIII inhibitors; or the successful completion of ITI treatment and documented FVIII inhibitor level <0.6 BU for at least 3 years (following ITI).
    5. Subjects who have previously been treated with plasma-derived and / or recombinant FVIII product(s) for at least 150 EDs for the treatment of acute bleeding episodes, routine prophylaxis, and / or perioperative bleeding.
    E.4Principal exclusion criteria
    Subjects meeting any of the following exclusion criteria must not be enrolled into the study:
    1. Participated in another clinical study with a plasma-derived or recombinant FVIII product other than Voncento® / Biostate® / Aleviate® / TBSF High Purity FVIII Concentrate within 30 days before the first administration of Voncento or at any time during the study.
    2. Known history of FVIII inhibitors or suspicion of having FVIII inhibitors (however subjects who have completed successful ITI treatment and have had documented FVIII inhibitor level <0.6 BU for at least 3 years following ITI may be included) .
    3. Currently receiving a therapy not permitted during the study (as defined in Section 9.3.1.4), which cannot be discontinued prior to enrolment into the study.
    4. Previous participation in the current study.
    5. 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.
    6. Known or suspected hypersensitivity to Voncento, or to any excipients of Voncento.
    7. Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.
    E.5 End points
    E.5.1Primary end point(s)
    The primary study endpoint is the incidence of FVIII inhibitor development.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The presence of FVIII inhibitors will be assessed by a central laboratory at every scheduled site visit (Visit 1-4), and additionally as required to assess any case of suspected inhibitors.
    E.5.2Secondary end point(s)
    The secondary endpoints of the study are based on the AEs and AESIs reported:
    • Nature and incidence of reported AEs, including SAEs.
    • (S)AEs related to Voncento treatment.
    • AESIs: thromboembolic events, hypersensitivity reactions, and suspected transmission of infectious agents.
    • Lack of effect (ie, a less than expected response to Voncento treatment when given for a bleeding event) as assessed by the investigator.
    E.5.2.1Timepoint(s) of evaluation of this end point
    AEs after signing the informed consent form with a start date on or after the first dose of Voncento will be collected.
    AEs will be assessed at every scheduled site visit (Visit 2-4).
    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 No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Low-interventional
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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
    Hong Kong
    Malaysia
    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 years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    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: 10
    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: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 90
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 Yes
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 100
    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)
    Patients who end their participation in the trial will continue the standard therapy according to their health care scheme. Patients who discontinue participation in the study prematurely will also continue the standard therapy according to their health care scheme.
    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 Decision2018-03-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-19
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-03-31
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