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    Summary
    EudraCT Number:2016-001094-33
    Sponsor's Protocol Code Number:BO39182
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-02-14
    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 number2016-001094-33
    A.3Full title of the trial
    A MULTICENTER, OPEN-LABEL, PHASE III STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB GIVEN EVERY 4 WEEKS (Q4W) IN PATIENTS WITH HEMOPHILIA A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients with Hemophilia A
    A.4.1Sponsor's protocol code numberBO39182
    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
    Hemophilia A
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is a genetic deficiency of blood clotting factor VIII (FVIII), which causes increased bleeding.
    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 10018938
    E.1.2Term Haemophilia A (Factor VIII)
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053751
    E.1.2Term Hemophilia A with anti factor VIII
    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
    There is no formal hypothesis testing in this study:

    Expansion phase:
    Efficacy objectives
    •To evaluate the efficacy of prophylactic emicizumab in maintaining
    adequate control of bleeding
    •To evaluate the clinical effect of prophylactic emicizumab on the number of joint bleeds over time, target joint bleeds over time, all bleeds over time, spontaneous bleeds over time
    •To evaluate the health-related quality of life, health status and patient preference

    Safety Objectives
    •To evaluate the overall safety of emicizumab given Q4W in patients with hemophilia A
    Pharmacokinetic Objective
    •To characterize the pharmacokinetics of multiple Q4W doses of 6mg/kg emicizumab
    E.2.2Secondary objectives of the trial
    Please note, there is no formal hypothesis testing in this study

    Run-in phase objectives:
    •To investigate the pharmacokinetics (PK) of emicizumab after single and multiple (every 4 weeks [Q4W]) subcutaneous (SC) administration of 6 milligram per Kilogram (mg/kg)
    •To assess the safety and tolerability of emicizumab after Q4W SC administration of 6 mg/kg
    •To explore the prophylactic effect of emicizumab in maintaining adequate control of bleeding
    •To investigate the effect of emicizumab on pharmacodynamic (PD) markers including (but not limited to) activated partial thromboplastin time, thrombin generation, and factor VIII (FVIII) activity
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Aged >=12 years
    •Body weight >= 40 kg at screening
    •Have severe congenital hemophilia A or hemophilia A with FVIII inhibitors
    •Patients using rFVIIa or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds
    •FVIII inhibitor test during screening with titer results available prior to first administration of study drug
    •Patients without FVIII inhibitors (< 0.6 Bethesda Units [BU]/milliliter
    [mL], < 1.0 BU/mL only for laboratories with an historical sensitivity
    cutoff for inhibitor detection of 1.0 BU/mL) who completed successful
    immune tolerance induction (ITI) must have done so at least 5 years
    before screening and must have no evidence of inhibitor recurrence
    (permanent or temporary) indicated by detection of an inhibitor > 0.6
    BU/mL (> 1.0 BU/mL only for laboratories with an historical sensitivity
    cutoff for inhibitor detection of 1.0 BU/mL) since ITI
    •For patients to be enrolled into PK run-in cohort: Current episodic treatment (FVIII or bypassing agents) at the time of entry into this study and documentation of details of episodic treatment for at least 24 weeks prior to entry into this study
    •For patients to be enrolled into the expansion cohort: Documentation of details of prophylactic or episodic treatment and the number of bleeding episodes for at least 24 weeks prior to entry into this study. For patients on an episodic regimen, >= 5 bleeds in the prior 24 weeks, regardless of inhibitor status
    •For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods that result in a failure rate of < 1percent per year during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug
    •Adequate values of hematologic, hepatic, and renal function tests
    E.4Principal exclusion criteria
    •Inherited or acquired bleeding disorder other than hemophilia A
    •Ongoing or planned ITI therapy; patient in whom ITI has failed will be eligible with a 72-hour washout period prior to the first emicizumab administration
    •History of illicit drug or alcohol abuse within 48 weeks prior to screening
    •Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA), in the investigator’s judgment
    •Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
    •Other conditions (e.g., certain autoimmune diseases) that may currently increase the risk of bleeding or thrombosis
    •History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
    •Known HIV infection with cluster of differentiation 4 (CD4) counts < 200 cells/microliter (μL) (CD4 counts >= 200 cell/μL will be permitted)
    •Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
    •Concomitant disease, condition, significant abnormality on screening evaluations or laboratory tests, or treatment that could interfere with the conduct of the study or pose an additional unacceptable risk in administering study drug to the patient
    •Women with a positive serum pregnancy test or pregnant/lactating women
    •Receipt of any of the following: 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 within last 30 days or 5 half-lives, whichever is shorter, and any other investigational drug currently being administered or planned to be administered
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy: (Expansion phase)
    1.Number of bleeds over time
    2.Number of joint bleeds over time
    3.Number of target joint bleeds over time
    4.Number of spontaneous bleeds over time
    5.Number of all bleeds over time (treated and untreated)
    6.HRQoL of patients according to Haem-A-QoL (>= 18years) or Haemo-QoL_SF (ages 12−17 years) scores after 24 weeks
    7.EQ-5D-5L scores after 24 weeks
    8.Patient preference (Preference Survey)
    9.Number of bleeds over time compared with the patient’s historical bleed rate over the last 24 weeks prior to study entry
    10.Number of days away from school/work
    11.Number of days hospitalized
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-4. Day 1 to study completion (24 weeks after emicizumab stop OR transfer to other emicizumab trial OR withdrawal)
    5-6. Week 1, 13, 25, then every 12 weeks until study completion (24 weeks after emicizumab stop OR transfer to other emicizumab trial OR withdrawal)
    7-9. Day 1 to study completion (24 weeks after emicizumab stop OR transfer to other emicizumab trial OR withdrawal)
    E.5.2Secondary end point(s)
    PK endpoints: (Run-in and expansion phase)
    1.Time to maximum observed plasma concentration
    2.Area under the plasma concentration (AUC)-time curve over a dosing interval
    3.Pre-dose (trough) plasma concentrations of emicizumab (Expansion phase)

    Safety endpoints: (Run-in and expansion phase)
    4.Incidence and severity of adverse events
    5.Incidence and severity of thromboembolic events
    6.Changes in physical examination findings and vital signs
    7.Incidence of laboratory abnormalities
    8.Incidence and severity of injection-site reactions
    9.Incidence of adverse events leading to drug discontinuation
    10.Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events
    11.Incidence of thrombotic microangiopathy
    12.Incidence of clinically significant levels of anti-emicizumab antibodies
    13.Incidence of de novo development of FVIII inhibitors (non-inhibitor population)

    Efficacy endpoint (Run-in phase):
    14.Maintaining adequate control of bleeding

    Pharmacodynamics (Run-in and expansion phase)
    15.PD marker analyses
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-3. Day 1 until study completion (24 weeks after emicizumab stop OR transfer to other emicizumab trial OR withdrawal)
    4-14. Up to study completion (24 weeks after emicizumab stop OR transfer to other emicizumab trial OR withdrawal)
    15. Day 1 until study completion (24 weeks after emicizumab stop OR transfer to other emicizumab trial OR withdrawal)
    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 No
    E.6.10Pharmacogenetic Yes
    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 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 Yes
    E.8.2.3.1Comparator description
    historical bleed rates
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    Belgium
    Germany
    Japan
    Poland
    Russian Federation
    Spain
    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
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    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: 4
    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: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 41
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 46
    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 Decision2017-06-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-02-27
    P. End of Trial
    P.End of Trial StatusOngoing
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