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    Summary
    EudraCT Number:2026-000042-29
    Sponsor's Protocol Code Number:YO39309
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2026-02-12
    Trial 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 number2026-000042-29
    A.3Full title of the trial
    A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
    A.3.2Name or abbreviated title of the trial where available
    HAVEN 5
    A.4.1Sponsor's protocol code numberYO39309
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03315455
    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 SponsorF. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    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 supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4058
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number41616881111
    B.5.6E-mailglobal.eudract@roche.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 Hemlibra
    D.2.1.1.2Name of the Marketing Authorisation holderRoche Registration 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 nameEmicizumab
    D.3.2Product code RO5534262
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous 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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeEmicizumab (RO5534262) is a recombinant humanized anti-activated blood coagulation Factor IX (anti-FIXa) and anti-blood coagulation Factor X (anti-FX) bispecific monoclonal antibody.
    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
    Hemophilia A with or without Factor VIII inhibitors
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is a genetic deficiency in blood clotting factor VIII, which causes increased bleeding. Inhibitors, however, prevent replacement factor VIII concentrates from controlling bleeds.
    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 28.0
    E.1.2Level LLT
    E.1.2Classification code 10053753
    E.1.2Term Hemophilia A without inhibitors
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 28.0
    E.1.2Level LLT
    E.1.2Classification code 10053751
    E.1.2Term Hemophilia A with anti factor VIII
    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 prophylactic emicizumab (i.e., administered on a scheduled basis with the intent to prevent bleeds) compared with no prophylaxis in patients with hemophilia A
    - To evaluate the clinical effect of prophylactic emicizumab on the number of bleeds in pediatric patients
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of prophylactic emicizumab compared with no prophylaxis
    - To evaluate the overall safety of prophylactic emicizumab compared with no prophylaxis in patients with hemophilia A
    - To evaluate the overall safety of prophylactic emicizumab treatment in pediatric patients with hemophilia A and inhibitors
    - To characterize the exposure (through plasma concentration) of emicizumab in patients treated on QW or Q4W dosing
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria for Arms A, B, and C:

    - Diagnosis of severe congenital hemophilia A or hemophilia A with FVIII inhibitors
    - Aged 12 years or older at the time of informed consent
    - Body weight ≥40 kilograms (kg) at the time of screening
    - Participants without FVIII inhibitors (<0.6 Bethesda unit per milliliter [BU/mL]) who completed successful immune tolerance induction (ITI) must have done so at least 5 years before screening and have no evidence of inhibitor recurrence (permanent or temporary)
    - Documentation of the details of episodic therapy (FVIII or bypassing agents) and of number of bleeding episodes for at least the last 24 weeks and ≥5 bleeds in the last 24 weeks prior to study entry
    - Adequate hematologic, hepatic, and renal function
    - For women of child bearing potential: agreement to remain abstinent or use a protocol defined contraceptive measure during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug

    Inclusion Criteria for Arm D:

    - Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e., ≥5 BU/mL)
    - Children <12 years old at time of informed consent
    - Body weight >3 kg at time of informed consent
    - Requires treatment with bypassing agents
    - Adequate hematologic, hepatic, and renal function
    - For female participants who are of childbearing potential, follow the same contraception criteria as listed above for Arms A, B, and C
    E.4Principal exclusion criteria
    Exclusion Criteria for Arms A, B, and C:

    - Inherited or acquired bleeding disorder other than hemophilia A
    - At high risk for thrombotic microangiopathy, in the investigator’s judgment
    - History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator’s judgment
    - Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
    - Other conditions that may increase risk of bleeding or thrombosis
    - History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
    - Known human immuno-deficiency virus (HIV) infection with cluster of differentiation 4 (CD4) count <200 cells/microliter (cells/mcL) within 24 weeks prior to screening. Participants with HIV infection who have CD4 >200 cells/mcL and meet all other criteria are eligible
    - Use of systemic immunomodulators at enrollment or planned use during the study, with the exception of anti-retroviral therapy
    - Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant’s safe participation in and completion of the study
    - Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study
    - Receipt of: Emicizumab in a prior investigational study; An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration; A non-hemophilia-related investigational drug concurrently, within last 30 days or 5 half-lives, whichever is shorter
    - Pregnant or lactating, or intending to become pregnant during the study

    Exclusion Criteria for Arm D:

    - Inherited or acquired bleeding disorder other than hemophilia A
    - Ongoing (or plan to receive during the study) ITI therapy or prophylaxis treatment with FVIII
    - Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
    - Other diseases that may increase risk of bleeding or thrombosis
    - History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
    - Known infection with HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV)
    - At high risk for thrombotic microangiopathy, in the investigator’s judgment
    - Use of systemic immunomodulators at enrollment or planned use during the study
    - Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study
    - Inability (or unwillingness by caregiver) to receive (allow receipt of) blood or blood products (or any standard-of-care treatment for a life-threatening condition)
    - Receipt of: Emicizumab in a prior investigational study; An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration; A non-hemophilia-related investigational drug concurrently, within last 30 days or 5 half-lives, whichever is shorter
    - Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant’s safe participation in and completion of the study
    - Pregnant or lactating, or intending to become pregnant during the study
    E.5 End points
    E.5.1Primary end point(s)
    Annualized bleeding rate (ABR) for Treated Bleeds
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline to at least 24 weeks
    E.5.2Secondary end point(s)
    1) Annualized bleeding rate (ABR) for All Bleeds
    2) ABR for Treated Spontaneous Bleeds
    3) ABR for Treated Joint Bleeds
    4) ABR for Treated Target Joint Bleeds
    5) Intra-Participant Comparison of the ABR for Treated Bleeds
    6) Intra-Participant Comparison of the ABR for All Bleeds
    7) Hemophilia A Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Week 25 in Participants ≥18 Years of Age
    8) Haemo-QoL-SF Questionnaire Total Score at Baseline and Week 25 in Participants 12 to 17 Years of Age
    9) European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) Questionnaire Visual Analog Scale (VAS) Score at Week 25
    10) EQ-5D-5L Index Utility Score at Week 25
    11) Incidence and severity of adverse events
    12) Incidence and severity of thromboembolic events
    13) Incidence and severity of thrombotic microangiopathy
    14) Incidence and severity of injection-site reactions
    15) Incidence of adverse events leading to drug discontinuation
    16) Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions
    17) Incidence of laboratory abnormalities
    18) Changes in vital signs
    19) Incidence of Anti-Emicizumab Antibodies
    20) Trough Plasma Concentration of Emicizumab
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) to 6) From Baseline to at least 24 weeks
    7) to 10) Baseline and Week 25
    11) to 17) From Baseline until end of study (up to 7 years, 4 months)
    18) Baseline, Weeks 5, 25, 49, and at study completion
    19) At prespecified timepoints from Baseline until end of study (up to 7 years, 4 months)
    20) At prespecified timepoints from Baseline until end of study (up to 7 years, 4 months)
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Standard of care episodic therapy with Factor VIII or bypassing agents
    E.8.2.4Number of treatment arms in the trial4
    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
    Malaysia
    Hong Kong
    China
    Thailand
    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
    The end of this study is defined as the date when the last remaining patient has completed the last visit (LPLV), as defined by any of the following criteria:

    - Completion of 24 weeks of emicizumab treatment and transfer to a future extension study to receive further emicizumab
    - Completion of the end-of-study safety follow-up visit 24 weeks after discontinuing emicizumab
    - Withdrawal of consent
    - Lost to follow-up
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days0
    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: 26
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 11
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 58
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    This study included pediatric patients (<18 years old). Investigators will obtain the informed assent of the patient and informed consent from a guardian or legally authorized representative of the patient in accordance with applicable law.
    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 85
    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)
    The Sponsor will offer continued access to emicizumab free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product at: https://assets.roche.com/f/176343/x/1b18e080e0/2025-revision-roche_global_policy_on_continued_access_to_investigational_interventions.pdf

    A patient's eligibility to receive emicizumab after completing the study is detailed in the Protocol section 4.3.4 "Post-Trial Access to Emicizumab".
    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: China
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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