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    Summary
    EudraCT Number:2019-002023-15
    Sponsor's Protocol Code Number:EFC16293
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-10-10
    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 ConcernedUK - MHRA
    A.2EudraCT number2019-002023-15
    A.3Full title of the trial
    Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients ≥12 Years of Age With Severe Hemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA Phase 3, Open-label Interventional Study of an Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, BIVV001, in Patients With Severe Hemophilia A (XTEND-1).
    A.3.2Name or abbreviated title of the trial where available
    XTEND-1
    A.4.1Sponsor's protocol code numberEFC16293
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1223-4867
    A.5.4Other Identifiers
    Name:INDNumber:17464
    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 SponsorBioverativ Therapeutics Inc.
    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 supportBioverativ Therapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPSI CRO AG
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street AddressBAARERSTRASSE 113A
    B.5.3.2Town/ cityZug
    B.5.3.3Post code6300
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number0037069439960
    B.5.6E-mailjurate.laisiene@psi-cro.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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2176
    D.3 Description of the IMP
    D.3.1Product nameRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.2Product code BIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.4Pharmaceutical form Powder 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 INNRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.9.2Current sponsor codeBIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN
    D.3.9.4EV Substance CodeSUB195807
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 No
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2176
    D.3 Description of the IMP
    D.3.1Product nameRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.2Product code BIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.4Pharmaceutical form Powder 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 INNRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.9.2Current sponsor codeBIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN
    D.3.9.4EV Substance CodeSUB195807
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    D.3.11 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 No
    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: 3
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2176
    D.3 Description of the IMP
    D.3.1Product nameRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.2Product code BIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.4Pharmaceutical form Powder 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 INNRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.9.2Current sponsor codeBIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN
    D.3.9.4EV Substance CodeSUB195807
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    D.3.11 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 No
    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: 4
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2176
    D.3 Description of the IMP
    D.3.1Product nameRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.2Product code BIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.4Pharmaceutical form Powder 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 INNRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.9.2Current sponsor codeBIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN
    D.3.9.4EV Substance CodeSUB195807
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000
    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 No
    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: 5
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2176
    D.3 Description of the IMP
    D.3.1Product nameRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.2Product code BIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.4Pharmaceutical form Powder 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 INNRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.9.2Current sponsor codeBIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN
    D.3.9.4EV Substance CodeSUB195807
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3000
    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 No
    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: 6
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2176
    D.3 Description of the IMP
    D.3.1Product nameRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.2Product code BIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.4Pharmaceutical form Powder 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 INNRecombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
    D.3.9.2Current sponsor codeBIVV001 (rFVIIIFc-VWF-XTEN)
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN
    D.3.9.4EV Substance CodeSUB195807
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4000
    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 No
    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
    Severe hemophilia A
    E.1.1.1Medical condition in easily understood language
    N/A
    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 10016080
    E.1.2Term Factor VIII deficiency
    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 evaluate the efficacy of BIVV001 as a prophylaxis treatment
    E.2.2Secondary objectives of the trial
    -To evaluate the efficacy of BIVV001 as a prophylaxis treatment
    -To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes
    - To evaluate BIVV001 consumption for the prevention and treatment of bleeding episodes
    - To evaluate the effect of BIVV001 prophylaxis on joint health outcomes
    - To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes
    - To evaluate the efficacy of BIVV001 for perioperative management
    Safety Objective
    - To evaluate the safety and tolerability of BIVV001 treatment
    Pharmacokinetic Objective
    - To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays
    Exploratory Objectives
    - To evaluate joint-health structural outcomes via ultrasound using the JADE protocol and/or HEAD-US
    - To assess the impact of BIVV001 treatment on patient reported outcome (PRO) measurements and physical activity (measures per study arm and treatment regimen)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participant, male or female, must be equal to or greater than 12 years of age inclusive, at the time of signing the informed consent.
    2. Severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity
    3. Previous treatment for hemophilia A (prophylaxis or on demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs.
    4. Current regimen includes one of the following:
    • Prophylactic treatment regimen with a marketed FVIII product or prophylactic emicizumab therapy for at least 6 months during the previous 12 months. Appropriate washout time needs to be taken into account.
    • On-demand regimen with a marketed FVIII product with a history of at least 12 bleeding episodes in the previous 12 months or at least 6 bleeding episodes in the previous 6 months prior to study enrollment.
    On-demand participant is accepting to move to a prophylaxis treatment regimen after 26-week on-demand period.
    5. Willingness and ability of the participant or surrogate (a caregiver or a family member ≥18 years of age) to complete training in the use of the study electronic Patient Diary (ePD) and to use the ePD throughout the study.
    6. Ability of the participant or his or her legally authorized representative (eg., parent or legal guardian) to understand the purpose and risks of the study, willing and able to comply with study requirements and provide signed and dated informed consent or assent (as applicable) and authorization to use protected health information in accordance with national and local participant privacy regulations.
    E.4Principal exclusion criteria
    - Clinically significant liver disease.
    - Serious active bacterial or viral infection (other than chronic hepatitis or HIV) present within 30 days of Screening.
    - Other known coagulation disorder(s) in addition to hemophilia A.
    - History of hypersensitivity or anaphylaxis associated with any FVIII
    product
    - Positive inhibitor results, defined as ≥0.6 BU/mL at Screening. History of a positive inhibitor test defined as ≥0.6 BU/mL. Family history of inhibitors will not exclude the participant.
    - Use of Emicizumab within the 20 weeks prior to Screening
    - Major surgery within 8 weeks prior to Screening.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    Annualized bleeding rate (ABR) in prophylaxis treatment arm
    E.5.1.1Timepoint(s) of evaluation of this end point
    as per schedule of activities from study protocol
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints:
    -Intra-patient comparison of ABR of participants of this study to the ABR
    of same participants previously treated in an observational study
    -Annualized bleeding rate (ABR) by type of bleed
    -Annualized bleeding rate (ABR) by location of bleed
    -Annualized bleeding rate (ABR) for all bleeding episodes including untreated bleeding episodes
    -Intra-patient comparison of ABR during the weekly once (QW) prophylaxis treatment period versus ABR during the on-demand treatment period
    - Percentage of participants who maintain FVIII activity levels
    -Number of injection and dose of BIVV001 to treat a bleeding episode
    -Percentage of bleeding episodes treated with a single injection of BIVV001
    -Assessment of response to BIVV001 treatment of individual bleeding episodes
    -Physician's global assessment of the participant's response based on BIVV001 treatment
    -Total annualized BIVV001 consumption
    -Annualized Joint Bleeding Rate (AJBR)
    -Target joint resolution
    -Change in Hemophilia Joint Health Score (HJHS) total score and domain scores
    -Change in PROMIS-SF Physical Function
    -Changes in Haem-A-QoL total score and physical health score
    -Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment
    -Number of injections and dose to maintain hemostasis during perioperative period for major surgery
    -Total BIVV001 consumption during perioperative period for major surgery
    -Number of blood component transfusions used during perioperative period for major surgery
    -Type of blood component transfusions used during perioperative period for major surgery
    -Estimated blood loss during perioperative period for major surgery
    -Number of participants with occurance of adverse events (AEs) and serious adverse events (SAEs)
    -Number of participants with inhibitor development
    -Number of participants with occurrence of embolic and thrombotic events
    -PK parameter: Maximum activity (Cmax)
    -PK parameter: Elimination half-life (t1/2)
    -PK parameter: Total clearance (CL)
    -PK parameter: Total clearance at steady state (CLss)
    -PK parameter: Accumulation index (AI)
    -PK parameter: Area under the activity time curve (AUC)
    -PK parameter: Volume of distribution at steady state (Vss)
    -PK parameter: Mean residence time (MRT)
    -PK parameter: Incremental recovery (IR)
    -PK parameter: Trough activity (Ctrough)
    -PK parameter: Time above FVIII activity levels
    E.5.2.1Timepoint(s) of evaluation of this end point
    For efficacy, safety and PK assessments, planned time points are according to the Schedule of Activities in the study 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 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) 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 Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    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 trial2
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    Australia
    Belgium
    Brazil
    Bulgaria
    Canada
    Colombia
    France
    Germany
    Greece
    Hungary
    Italy
    Japan
    Mexico
    Netherlands
    Spain
    Taiwan
    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
    EoS will occur when all of the following have been met:
    - 104 pts. reached 50 Exposure Days (ED) and completed a valid inhibitor test after the 50th ED.
    - At least 63 pts. in Arm A have at least 6 months of participation in this study and have at least 6 months of participation in Study 242HA201/OBS16221 prior to baseline.
    - 13 pts. in the PK subgroup completed the BIVV001 Day 1 PK profile,
    and a repeat 14-day BIVV001 PK profile 26 weeks later with adequate estimate of
    terminal half-life.
    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 months3
    E.8.9.1In the Member State concerned days22
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 149
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 75
    F.4.2.2In the whole clinical trial 164
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-11-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-06
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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