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    EudraCT Number:2011-000181-34
    Sponsor's Protocol Code Number:OBI-1-301
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-02-07
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSweden - MPA
    A.2EudraCT number2011-000181-34
    A.3Full title of the trial
    Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of a new factor VIII replacement for patients with acquired hemophilia A
    A.4.1Sponsor's protocol code numberOBI-1-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01178294
    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 SponsorBaxter Innovations GmbH
    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 supportBaxter Innovations GmbH
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxter Innovations GmbH
    B.5.2Functional name of contact pointGuido Wuerth, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressWagramer Strasse 17-19
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1220
    B.5.4Telephone number+43120100 2473489
    B.5.5Fax number+43120100 717
    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/3/10/784
    D.3 Description of the IMP
    D.3.1Product nameOBI-1
    D.3.2Product code OBI-1
    D.3.4Pharmaceutical form Powder and solvent for solution 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 INNobyoctocog alfa
    D.3.9.1CAS number 1339940-90-7
    D.3.9.2Current sponsor codeOBI-1
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number350 to 650
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Haemophilia A
    E.1.1.1Medical condition in easily understood language
    Blood disorder characterized by bleeding episodes, spontaneous or in response to minimal trauma that usually occurs in the elderly
    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 16.0
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Main objectives:
    To evaluate the efficacy of OBI-1 for the treatment of serious bleeding episodes in subjects with acquired hemophilia with autoimmune inhibitors to human factor VIII.
    E.2.2Secondary objectives of the trial
    1)To determine the proportion of serious bleeding episodes controlled with OBI-1.
    2)To assess the efficacy of OBI-1 at designated time points after the initiation of therapy.
    3)To determine the frequency, total dose, and total number of infusions of OBI-1 required to control all serious bleeding episodes.
    4)To assess the correlation between response to anti-OBI-1 therapy at specified assessment time points and eventual control of serious bleeding episodes.
    5)To assess the correlation between the preinfusion anti-OBI-1 inhibitor titer, the total dose of OBI-1, the outcome at 24 hours and the eventual control of the bleeding episode.
    6)To assess the anti-OBI-1 inhibitor level preinfusion, at specified time points during treatment, and at 90 days post final infusion.
    7)To evaluate the safety of OBI-1.
    8)To assess drug exposure using extensive(non-bleeding state) or sparse sampling(bleeding state), and a population PK approach with sparse data in subjects treated with OBI-1.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males or females ≥18 years of age.
    2. Written informed consent from subject, trusted person or person who is legally authorized to sign on behalf of the subject (Legal representative in U.S.), depending on local regulations.
    3. Subjects with acquired hemophilia with autoimmune inhibitors to human factor VIII with a clinical diagnosis established by the following criteria:
    a) Prolonged activated partial thromboplastin time (aPTT)
    b) Prothrombin time (PT) ≤ULN + 2 seconds and platelet count within normal range
    c) Abnormal aPTT mixing study (patient normal control 1:1) consistent with a factor VIII inhibitor
    d) Reduced factor VIII activity level (below 10%)
    4. Has a serious bleeding episode, as documented by the investigator.
    5. Be willing and able to follow all instructions and attend all study visits.
    6. Subjects taking antithrombotics (such as clopidogrel, heparin or heparin analogue) may be included provided three half lives of the agent have elapsed since the last dose of the agent.
    7. Life expectancy of at least 90 days prior to the onset of the bleeding episode.
    8. Subjects of reproductive age will be required to use acceptable methods of birth control and, if female, undergo pregnancy testing as part of the screening process.
    E.4Principal exclusion criteria
    1. Hemodynamically unstable after blood transfusion, fluid resuscitation and pharmacologic or volume replacement pressor therapy.
    2. Has an established reason for bleeding that is not correctable.
    3. Bleeding episode assessed likely to resolve on its own if left untreated.
    4. Anti-OBI-1 inhibitor that exceeds 20 BU (prospectively or retrospectively).
    5. Subsequent bleeding episode at the site of the initial qualifying bleeding episode within two weeks following the final OBI-1 dose for the initial qualifying bleeding episode, or subsequent bleeding episode at a different site than the initial qualifying bleeding episode within 1 week following the final OBI-1 dose for the initial qualifying bleeding episode will not be considered “new” qualifying bleeding episodes.
    6. Prior history of bleeding disorder other than acquired hemophilia.
    7. Known major sensitivity (anaphylactoid reactions) to therapeutic products of porcine or hamster origin; examples include therapeutics of porcine origin (e.g. previously marketed porcine factor VIII, Hyate-C®) and recombinant therapeutics prepared from hamster cells (e.g. Humira®, Advate® and Enbrel®).
    8. Use of hemophilia medication: rFVIIa within 3 hours prior to OBI-1 administration, or aPCC treatment within 6 hours prior to OBI-1 administration.
    9. Participation in any other clinical study within 30 days of the first OBI-1 treatment.
    10. Anticipated need for treatment or device during the study that may interfere with the evaluation of the safety or efficacy of OBI-1, or whose safety or efficacy may be affected by OBI-1.
    11. Is currently pregnant, or breastfeeding or planning to become pregnant or father a child during the study.
    12. Abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the subject’s safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
    13. Inability or unwillingness to comply with the study design, protocol requirements, or the follow-up procedures.
    14. Patient of majority age under legal protection.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the proportion of serious bleeding episodes responsive to OBI-1 therapy at 24 hours after the initiation of treatment. Response to OBI-1 therapy will be based on assessment of effectiveness and factor VIII blood levels.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 24 hours
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints:
    1) The overall proportion of serious bleeding episodes successfully controlled with OBI-1 therapy, as assessed by the investigator.
    2) The proportion of bleeding episodes responsive to OBI-1 therapy at designated assessment time points after the initiation of therapy, as assessed by the investigator.
    3) Frequency, total dose, and total number of infusions of OBI-1 required to successfully control qualifying bleeding episodes.
    4) Correlation between response to OBI-1 therapy at specified time points and eventual control of serious bleeding episodes.
    5) Correlation between the pre-infusion anti-OBI-1 antibody titers, the total dose of OBI-1, the outcome at 24 hours and the eventual control of the bleeding episode.
    6) Drug exposure will be determined by means of population PK analysis for the sparse (bleeding state data) and compartmental analysis for the complete (non-bleeding) data. At least the following PK parameters will be estimated: Cl, Vd, area under the concentration-time curve (AUC) and Cmax/Dose.

    Safety endpoints:
    1) TEAEs and serious adverse events (SAEs) throughout the study.
    2) Biochemistry, hematology, urinalyses and vital signs.
    3) Anti-human factor VIII antibody titer.
    4) Anti-OBI-1 antibody titer.
    5) Anti-host cell protein (BHK) antibody titer.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1)-5)Each dose or q8h to 24h;24h;each dose or q12h from 24- 120h;each dose or q24h thereafter until last OBI-1 dose or withdrawal.
    6)screening; 10-20min;each dose or q8h to 24h;24h;each dose or q12h from 24-120h;each dose or q24h thereafter until last OBI-1 dose or withdrawal;optional:last OBI-1 dose
    1)Ongoing basis throughout study including follow-up
    2)Vital signs:screening;10-20min after end of first dose; each dose or q8h up to 24h;24h;each dose or q12h from 24h-120h;each dose or q24h thereafter until last OBI-1 dose or withdrawal;follow-up
    Analyses:screening;at each dose or q8h to 24h;24h;follow-up
    3)-4)Screening;q5 days during treatment;follow-up
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    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 No
    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.8.3 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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    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
    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 months6
    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 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 No
    F.1.1Number of subjects for this age range: 0
    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: 4
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    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. of subjects incapable of giving consent
    As the bleeding episodes may be life threatening bleeding episodes, the patient may not be in a position to consent. A trusted person/legal representative may do so on the patient's behalf.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 28
    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)
    It is expected that the need for treatment will be completed, i.e. the bleed will have been managed. Most subjects will then be immunosuppressed and in the majority the acquired antibody will be suppressed and there will be no need for future 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 Decision2012-02-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-05-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-10-09
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