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    Summary
    EudraCT Number:2016-000072-17
    Sponsor's Protocol Code Number:BH30071
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-05-30
    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
    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 number2016-000072-17
    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
    WITHOUT INHIBITORS
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Effectiveness and Safety of Prophylactic Emicizumab Versus no Prophylaxis in Haemophilia A Patients Without Inhibitors
    A.4.1Sponsor's protocol code numberBH30071
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02847637
    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.4.1Name of organisation providing supportChugai Pharmaceutical Co. Ltd
    B.4.2CountryJapan
    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 code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@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 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/3/13/1221
    D.3 Description of the IMP
    D.3.1Product nameACE910
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEmicizumab
    D.3.9.2Current sponsor codeRo 553-4262/F03
    D.3.9.3Other descriptive nameRO5534262 EMICIZUMAB
    D.3.9.4EV Substance CodeSUB168081
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 typeRO5534262 (ACE910) 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
    Haemophilia A without factor VIII inhibitors
    E.1.1.1Medical condition in easily understood language
    Haemophilia A is a genetic deficiency of blood clotting factor VIII (FVIII), which causes increased bleeding. It is treated with frequent, intravenous FVIII infusion
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053753
    E.1.2Term Hemophilia A without inhibitors
    E.1.2System Organ Class 100000004850
    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 (1.5 mg/kg/week or 3 mg/kg/2weeks) compared with no prophylaxis in patients with haemophilia A without FVIII inhibitors on the basis of the number of bleeds over time (primary endpoint is based on treated bleeds)
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of prophylactic emicizumab on the basis of number of bleeds over time compared with the patient's historical bleed rate (treated and all bleeds)
    • To evaluate the efficacy of prophylactic emicizumab (1.5 mg/kg/week or 3 mg/kg/2weeks) on the basis of:
    - All bleeds over time
    - Spontaneous bleeds over time (spontaneous bleed rate)
    - Joint bleeds over time
    - Target joint bleeds over time
    - Health-related quality of life (HRQoL) of patients according to Haem A QoL (aged >= 18) or Haemo-QoL-Short Form (aged 12-17) scores after 24 weeks
    - Health status of patients according to EuroQoL Five Dimension Five Levels Questionnaire (EQ-5D-5L) scores after 24 weeks
    • Maintaining adequate control of bleeding in patients previously treated with factor VIII prophylaxis by evaluation of the bleed rate (treated and all bleeds)
    • To evaluate overall safety of prophylactic emicizumab in patients with haem. A without inhibitors
    • To evaluate pharmacokinetic of emicizumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Aged 12 years or older at the time of informed consent
    - Body weight >= 40 kg at the time of screening
    - Diagnosis of severe congenital haemophilia A (intrinsic FVIII level < 1%)
    - A negative test for inhibitor (i.e., < 0.6 Bethesda units [BU]) within 8 weeks of enrollment
    - No documented inhibitor (i.e., < 0.6 BU), FVIII half-life < 6 hours, or FVIII recovery < 66% in the last 5 years
    - Patients who completed successful immune tolerance induction (ITI) at least 5 years before screening are eligible, provided they have had no evidence of inhibitor recurrence (permanent or temporary) as may be indicated by detection of an inhibitor, FVIII half-life < 6 hrs, or FVIII recovery < 66% since completing ITI
    - Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks
    - For patients on no prophylaxis (episodic treatment) pre-study, >= 5 bleeds in the last 24 weeks prior to study entry
    - Patients who were on FVIII prophylaxis for at least the last 24 weeks, can be enrolled regardless of the number of bleeds during this period. Eligibility will be based on investigator's attestation of adequate prophylaxis regimen.
    - At least 40 patients who were on FVIII prophylaxis pre-enrolment will have been enrolled for minimum of 24 weeks in Study BH29768 (non-interventional)
    - Adequate haematologic function, defined as platelet count >= 100,000/ microliter and haemoglobin >= 8 gram/decilitre (4.97 millimoles/liter) at the time of screening
    - Adequate hepatic and renal function
    - Agreement to remain abstinent or use contraceptive methods specified in the study
    E.4Principal exclusion criteria
    - Inherited or acquired bleeding disorder other than haemophilia A
    - Previous (in the past 12 months) or current treatment for thromboembolic disease (except previous catheter-associated thrombosis for which anti thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
    - Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
    - Known human immunodeficiency virus infection with CD4 count < 200 cells/microlitre within 24 weeks prior to screening
    - Use of systemic immunomodulators at enrolment or planned use during the study (except anti-retroviral therapy)
    - Patients who are at high risk for thrombotic microangiopathy (e.g., have a previous medical or family history of TMA), in the investigator's judgment
    - Receipt of emicizumab in a prior investigational study, an investigational drug to treat or reduce the risk of haemophilic bleeds within 5 half-lives of last drug administration, and a non-haemophilia-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
    - Women who are not postmenopausal (>=48 weeks of non-therapy-induced amenorrhea) or surgically sterile must have a negative pregnancy test result within 7 days prior to initiation of study drug
    E.5 End points
    E.5.1Primary end point(s)
    1. Number of bleeds over time (i.e., bleed rate)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 2 years
    E.5.2Secondary end point(s)
    Efficacy:
    1. Change in the number of bleeds over time compared with the patient'shistorical bleed rate over the last 24 weeks prior to study entry
    2. All bleeds over time (treated and not treated)
    3. Spontaneous bleeds over time (spontaneous bleed rate)
    4. Joint bleeds over time
    5. Target joint bleeds over time
    6. HRQoL of patients according to Haem-A-QoL (aged >=18) or Haemo-QoL-Short Form (aged 12-17) scores
    7. Health status of patients according to EQ-5D-5L scores
    8. Maintaining adequate control of bleeding by evaluation of the bleed rate in patients previously on factor VIII prophylaxis

    Safety:
    9. Incidence of adverse events
    10. Incidence of thromboembolic events
    11. Incidence of new physical examination abnormalities
    12. Incidence of new vital signs abnormalities
    13. Incidence of laboratory abnormalities
    14. Incidence injection-site reactions
    15. Incidence of adverse events leading to drug discontinuation
    16. Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions
    17. Incidence and severity of thrombotic microangiopathy
    18. Incidence and clinical significance of anti-emicizumab antibodies
    19. Incidence of de novo development of FVIII inhibitors in patients receiving emicizumab prophylaxis

    Pharmacokinetic (PK):
    20. Trough plasma concentrations of emicizumab 9. Incidence of adverse events
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-20. Up to 2 years
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    armC: no prophylaxis (standard of care rescue medication); historical intrapatient control
    E.8.2.4Number of treatment arms in the trial4
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Australia
    Costa Rica
    France
    Germany
    Ireland
    Italy
    Japan
    Korea, Republic of
    Poland
    South Africa
    Spain
    Taiwan
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    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 patient completes the last safety follow-up visit 24 weeks after the last dose of emicizumab, enrolls in an emicizumab extension study, or is lost to follow-up.
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 12
    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: 12
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 128
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 145
    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 post-trial access to the study drug (RO5534262)
    free of charge to eligible patients in accordance with the Roche Global
    Policy on Continued Access to Investigational Medicinal Product.
    The Roche Global Policy on Continued Access to Investigational
    Medicinal Product is available at the following Web site:
    http://www.roche.com/policy_continued_access_to_investigational_
    medicines.pdf
    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 Decision2016-08-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-07-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-05-12
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    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
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