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    Summary
    EudraCT Number:2019-004430-42
    Sponsor's Protocol Code Number:AHA-EMI
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-04-22
    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 ConcernedAustria - BASG
    A.2EudraCT number2019-004430-42
    A.3Full title of the trial
    Emicizumab in Patients with Acquired Hemophilia A: Multicenter, Single-Arm, Open-Label Clinical Trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of the effect of emicizumab in patients suffering from acute bleeds with previously no family history.
    A.4.1Sponsor's protocol code numberAHA-EMI
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04188639
    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 SponsorGWT-TUD GmbH
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    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 organisationGWT-TUD GmbH
    B.5.2Functional name of contact pointMedical Consulting
    B.5.3 Address:
    B.5.3.1Street AddressFreiberger Str. 33
    B.5.3.2Town/ cityDresden
    B.5.3.3Post code01067
    B.5.3.4CountryGermany
    B.5.4Telephone number+4935125933172
    B.5.5Fax number+4935125933198
    B.5.6E-mailmedical.consulting@g-wt.de
    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.3.4Pharmaceutical 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.3.9.3Other 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 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
    Acquired Hemophilia A (AHA)
    E.1.1.1Medical condition in easily understood language
    aquired bleeding disorder
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.1
    E.1.2Level LLT
    E.1.2Classification code 10053761
    E.1.2Term Acquired hemophilia with anti FVIII, XI, or XIII
    E.1.2System Organ Class 100000004851
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053760
    E.1.2Term Acquired hemophilia
    E.1.2System Organ Class 100000004851
    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 present study is to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
    E.2.2Secondary objectives of the trial
    - To study the safety of emicizumab in patients with AHA
    - To compare bleeding and adverse events with the historic GTH-AH 01/2010 cohort
    - To compare bleeding and adverse events with a parallel US study
    - To evaluate the pharmacokinetics (PK) of emicizumab in patients with AHA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Patients diagnosed with AHA based on a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.6 BU/ml) at the time of diagnosis (local laboratory)
    2) Signed informed consent form by the participant or a person who is legally authorized to sign on behalf of the participant before any study specific tests or procedures
    3) Male or female patients aged 18 years or older at the time of informed consent
    4) Ability to understand and follow study-related instructions
    5) Current bleeds due to AHA at the time of screening
    E.4Principal exclusion criteria
    1) Congenital hemophilia A
    2) Partial or complete remission of AHA (defined as FVIII:C =50% and no bleeding and no hemostatic therapy) at the time of screening
    3) Treatment with aPCC within the last 48 h before first study treatment or planned treatment with aPCC during the course of the study
    4) Treatment of AHA within the days before study enrollment with more than 100 mg prednisolone (or equivalent) per day or prednisolone for more than 2 days or with other immunosuppressive drugs (e.g. rituximab, cyclophosphamide). IST for other concomitant disorders (e.g. autoimmune disorders) is not an exclusion criterion and can be continued at the investigator’s discretion.
    5) Therapy (current or planned during the emicizumab treatment period) with immunosuppressive or immune modulating drugs that were not already given on a regular basis before first diagnosis of AHA
    6) Positive lupus anticoagulant at the time of screening
    7) Severe uncontrolled infection at the time of screening
    8) Signs of active disseminated intravascular coagulation at the time of screening
    9) Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening
    10) Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator’s judgment
    11) Known severe congenital or acquired thrombophilia
    12) Life expectancy <3 months at the time of screening
    13) Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator
    14) Contraindications according to the Investigator’s Brochure of emicizumab
    15) Current treatment with emicizumab at time of screening
    16) History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator
    17) 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 local investigator, preclude the
    patient’s safe participation in and completion of the study
    18) Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator
    19) Pregnant or breast-feeding women
    20) Women of childbearing potential unless women who meet the following criteria:
    a. Post-menopausal (12 months natural amenorrhea or 6 months amenorrhea with serum FSH > 40 U/mL)
    b. Postoperatively (six weeks after bilateral ovariectomy with or without hysterectomy)
    c. Regular and correct use of a contraceptive method with error rate <1% per year such as implants, depot injections, oral contraceptives or intrauterine devices
    d. Sexual abstinence
    e. Vasectomy of the partner
    21) Subject is in custody by order of an authority or a court of law
    22) Receipt of an investigational drug concurrently or within 5 half-lives before administration of the study drug
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy objective is to evaluate the efficacy of prophylactic emicizumab which will be evaluated by determination of the rate of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 weeks
    E.5.2Secondary end point(s)
    The secondary study objective to study the safety of emicizumab in patients with AHA will be evaluated on the basis of the incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab treatment and mortality in the 24 weeks after starting emicizumab.

    Further secondary endpoints include the bleeding-free survival in the 12 weeks after starting emicizumab treatment, mortality and cause of death, days of treatment with and dose of bypassing agents, days in hospital and number of patients archieving partial remission in the 24 weeks after starting emicizumab treatment.

    The additional exploratory objective to compare bleeding and adverse events with the historic GTH-AH 01/2010 cohort will be evaluated on basis of 1) the number of clinically significant bleeds per patient-week until death or week 12 after starting treatment, whatever occurs first; 2) the incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy until week 12 after starting treatment and mortality after 24 weeks, and 3) bleeding-free survival until week 12 after starting treatment.

    The additional exploratory objective to compare bleeding and adverse events with a parallel US study will be evaluated on basis of 1) the number of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first; 2) the incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy until week 12 after starting emicizumab treatment and mortality after 24 weeks, and 3) bleeding-free survival until week 12 after starting emicizumab treatment.

    PK parameters derived by emicizumab plasma levels will be analyzed and descriptively summarized.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 weeks
    24 weeks
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 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 EEA30
    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 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
    As for this study, the primary outcome will be analyzed after the last patient has completed the therapy, thus, the end of the study will be the date when the clean database is available. However, the primary completion event of the study will be the date of last patient last visit (LPLV).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 23
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 47
    F.4.2.2In the whole clinical trial 47
    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)
    After conclusion of the clinical study, patients will continue the IST therapy as medically indicated and receive further standard medical care as usual for this kind of disease. Emicizumab will not be provided to the patients for further treatment.
    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 Decision2021-06-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-01-04
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