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    Summary
    EudraCT Number:2013-003305-25
    Sponsor's Protocol Code Number:CSLCT-BIO-12-83
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-11-10
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2013-003305-25
    A.3Full title of the trial
    An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects with Von Willebrand Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Voncento® in Subjects with Von Willebrand Disease
    A.4.1Sponsor's protocol code numberCSLCT-BIO-12-83
    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 number4906421 39 2930
    B.5.5Fax number4906421 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 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 nameVoncento
    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.3.8 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 typeequal
    D.3.10.3Concentration number500
    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 typeequal
    D.3.10.3Concentration number1200
    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.IMP: 2
    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 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 nameVoncento
    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.3.8 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 typeequal
    D.3.10.3Concentration number1000
    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 typeequal
    D.3.10.3Concentration number2400
    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 Disease
    E.1.1.1Medical condition in easily understood language
    VWD is an inherited bleeding disorder caused by deficiency of a protein normally found in the blood called von Willebrand factor (VWF).
    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 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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to collect long-term data on the haemostatic efficacy of Voncento in subjects with VWD who require a VWF product to control an NSB event or as prophylaxis therapy.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    • To assess the efficacy of Voncento in the prevention and treatment of surgical bleeding events.
    • To collect long-term data on the safety of Voncento used as on-demand therapy to treat an NSB event or as prophylaxis therapy.
    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, or the subject’s parent(s) or legally acceptable representative(s) is / are capable of providing written informed consent.
    2. Male or female.
    3.Subjects of any age.
    4. Diagnosis of severe type 1, 2A, or 3 VWD where VWF:RCo is <20% at screening.
    5. Availability of detailed patient documentation (eg, medical record, diary, logbook) covering prior bleeding and VWD treatment history over the past 12 months prior to screening.
    6. Subjects where DDAVP treatment is ineffective or contraindicated. In countries where DDAVP is not available only subjects with type 3 VWD are allowed to be enrolled.
    7. A documented vaccination against hepatitis A and B in the subject’s medical record (or presence of antibodies against hepatitis A and B due to either a previous infection or vaccination) prior to the first dose of Voncento.
    8. Investigator believes that the subject or the subject’s parent(s) or legally acceptable representative(s) is / are willing and able to adhere to all protocol requirements.
    9.Subjects who require a VWF product to control a NSB event or for
    ongoing prophylactic therapy.
    E.4Principal exclusion criteria
    Subjects meeting any of the following exclusion criteria must not be enrolled into the study:
    1. A known history of VWF or FVIII inhibitors, or are suspected to have VWF or FVIII inhibitors.
    2. Suffering any acute or chronic medical condition, other than VWD, which may, in the opinion of the investigator, affect the conduct of the study.
    3. Known or suspected hypersensitivity or previous evidence of severe side effects to Voncento, VWF / FVIII concentrates, or human albumin.
    4. Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
    5. Female subjects who are pregnant, breast-feeding, or who have a positive pregnancy test at screening or have the intention to become pregnant during the course of the study.
    6. Female subjects of childbearing potential or male subjects who have a female partner of childbearing potential, who either do not use or are not willing to use a medically reliable method of contraception or who are not sexually abstinent during the study, or not surgically sterile.
    7. Alcohol, drug, or medication abuse within 1 year before the study.
    8. Currently receiving a therapy not permitted during the study
    9. Previous participation in a Voncento / Biostate study (patients can also not be enrolled a second time into the current study).
    10. 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.
    11. 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 endpoints of the study are:
    • Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento in its usage for an NSB event.
    • Number of NSB events per month and annual bleeding rate (ABR).
    • Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat an NSB event.
    E.5.1.1Timepoint(s) of evaluation of this end point
    • Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento in its usage for an NSB event: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Assessed by the Investigator at approximately Months 3, 6, 9 and 12.
    • Number of infusions: For the duration of the subject's participation in the study (approximately 12 months)
    •Total dose of Voncento (in IU VWF:RCo) required to treat an NSB event: For the duration of the subject's participation in the study (approximately 12 months).
    •Number of NSB events per month: For the duration of the subject's
    participation in the study (approximately 12 months).
    E.5.2Secondary end point(s)
    The secondary endpoints of the study are:
    • Investigator’s or surgeon’s assessment of blood loss during a surgical event and an overall haemostatic efficacy assessment by the investigator after the surgical procedure.
    • Number of NSB events per month.
    • Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat a surgical bleeding event.
    • Nature and incidence of AEs, including serious adverse events (SAEs), AEs related to Voncento, and adverse events of special interest (AESIs), and number of VWF and FVIII inhibitors which occur during the administration of Voncento as on-demand therapy to treat an NSB or surgical bleeding event.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The secondary endpoints of the study are:
    • Investigator's or surgeon's assessment of blood loss during a surgical event and an overall haemostatic efficacy assessment by the investigator after the surgical procedure.
    • Number of infusions and total dose of Voncento (in IU VWF:RCo)
    required to treat a surgical bleeding event.
    • Nature and incidence of AEs, including serious adverse events (SAEs), AEs related to Voncento, and adverse events of special interest (AESIs), and number of VWF and FVIII inhibitors which occur during the
    administration of Voncento as on-demand therapy to treat an NSB or surgical bleeding event.
    •Haemostatic efficacy - prophylaxis: App. every month (subject
    assessment) and every 3 months (Investigator assessment)during
    subject's participation in study
    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 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 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 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 concerned3
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years2
    E.8.9.1In the Member State concerned months3
    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 months5
    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: 14
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 4
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 14
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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)
    Patinets who end their paticipation in the trial will be treated according to the current state of the art therapy recommendations. Patients who have received Voncento and discontinue participation in the trial prematurely will also be treated according to the current state of the art therapy recommendations.
    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 Decision2015-12-07
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-02-15
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