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    Summary
    EudraCT Number:2011-001899-18
    Sponsor's Protocol Code Number:OBI-1-302
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2011-07-18
    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 ConcernedUK - MHRA
    A.2EudraCT number2011-001899-18
    A.3Full title of the trial
    Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Patients with Congenital Hemophilia A With Factor VIII Inhibitors
    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 congenital
    hemophilia A
    A.4.1Sponsor's protocol code numberOBI-1-302
    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.1.3.4CountryAustria
    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.4.2CountryAustria
    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.5.3.4CountryAustria
    B.5.4Telephone number+43120100 2473489
    B.5.5Fax number+43120100 717
    B.5.6E-mailguido_wuerth@baxter.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/3/10/784
    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.3.9.3Other descriptive namerecombinant porcine coagulation factor VIII (B-domain deleted)
    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.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
    Congenital Hemophilia A with factor VIII inhibitors
    E.1.1.1Medical condition in easily understood language
    Haemophilia bleeding disorder present at birth, characterized by spontaneous bleeding episodes, as well as prolonged bleeding after trauma or surgery
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10018938
    E.1.2Term Haemophilia A (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
    To evaluate the efficacy of OBI-1 for the treatment of serious bleeding episodes in subjects with congenital hemophilia A with inhibitors to human factor VIII and with a history of inadequate response to bypassing agents.
    E.2.2Secondary objectives of the trial
    1) To determine the proportion of serious bleeding episodes controlled with OBI-1 therapy
    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 serious bleeding episodes
    4) To assess the correlations between response to OBI-1 therapy at specified assessment time points and eventual control of serious bleeding episodes.
    5) To assess the correlations between the pre-infusion anti-OBI-1 inhibitor titer, the total dose of OBI-1, the outcome at 24 hours and the eventual control of the bleeding disorder
    6) To assess the anti-OBI-1 inhibitor level pre-infusion, at specified time points during treatment and at the end of the follow-up period at 90 days post final infusion
    7) To evaluate the safety of OBI-1
    8) To assess the simple recovery and elimination rate parameters of OBI-1 in subjects with inhibitors treated with OBI-1 therapy
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Males or females ≥ 6 years of age.
    2) Written informed consent from subject or subject’s legal representative.
    3) Subjects with congenital hemophilia A with a human factor VIII inhibitor ≤30 BU assessed within 30 days prior to study entry and no documented human factor VIII inhibitor >30 BU within the prior 90 days..
    4) Has previously or is currently demonstrating suboptimal hemostatic response to bypassing agents for treatment of bleeding episodes; suboptimal response is determined by the investigator, but minimally includes no or minimal evidence of response after at least two doses of bypassing agents, either for the current or a historic bleeding episode.
    5) Has an anti-OBI-1 titer ≤10 BU.
    6) Has any serious or life-threatening bleeding episode; or requires a major or minor surgical procedure that could lead to a serious bleeding episode if not well controlled.
    7) Is willing and able to follow all instructions and attend all study visits.
    8) Has no other significant hemostatic abnormality and:
    a) Platelets >100,000/mm^3
    b) Prothrombin time <15 seconds
    c) INR ≤1.3
    9) Subjects taking anti-thrombotics (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.
    E.4Principal exclusion criteria
    1) Hemodynamically unstable after blood transfusion, fluid resuscitation and pharmacologic or volume replacement pressor therapy. This hemodynamic instability is characterized by symptomatic hypotension resulting in vital organ dysfunction, such as cardiac ischemia, oliguria (urine volume <0.5 mL/kg in the previous six hours), central nervous system hypoperfusion manifested by mental status change such as confusion (unless head injury or intracranial hemorrhage is present), pulmonary compromise, and/or acidosis (manifested by pH and lactate levels).
    2) Bleeding episode assessed likely to resolve on its own if left untreated.
    3) Prior history of bleeding disorder other than congenital hemophilia A.
    4) 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®).
    5) Received any other investigational treatment within 30 days of the first OBI-1 treatment.
    6) Anticipated need for treatment or device during the study that may interfere with the evaluation of the safety or efficacy of OBI-1, whose safety or efficacy may be affected by OBI-1.
    7) Is planning to father a child during the study.
    8) Has 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.
    9) Inability or unwillingness to comply with the study design, protocol requirements, or the follow-up procedures.
    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
    24 hours after initiation of treatment
    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. Correlation between the pre-infusion anti-OBI-1 antibody titers and the recovery of OBI-1.

    Safety endpoints:
    7. TEAEs and serious adverse events (SAEs) throughout the study.
    8. Biochemistry, hematology, urinalyses and vital signs.
    9. Anti-human factor VIII antibody titer.
    10. Anti-OBI-1 antibody titer.
    11. Anti-host cell protein (BHK) antibody titer.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy:
    1,3,4,5,6. Ongoing basis throughout study
    2. 24h after end of first dose;each dose or q8h to 24h, q12h from 24-120h and q24h thereafter until last OBI-1 dose or withdrawal;all f-up visits

    Safety:
    7. 0, 10-20min and 24h after end of first dose;at each dose or q8h to 24h, q12h from 24-120h and q24h thereafter until last OBI-1 dose or withdrawal;all f-up visits;termination
    8. Biochemistry, hematology and urinalyses: screening;24h after end of first dose;f-up visits 2-5;
    Vital signs: screening;10-20min and 24h after end of first dose;at each dose or q8h to 24h, q12h from 24-120h and q24h thereafter until last OBI-1 dose or withdrawal;all f-up visits
    9-10: Screening;q5 days during treatment and healing;all f-up visits;termination
    11: Screening;f-up visit 5;termination
    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.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 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 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 concerned4
    E.8.5The trial involves multiple Member States No
    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
    Canada
    South Africa
    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
    LVLS
    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 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 Yes
    F.1.1Number of subjects for this age range: 18
    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: 9
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 9
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    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)
    For patients who respond to OBI-1 and do not develop inhibitors, a follow on study (expanded access) is being developed to allow them to receive OBI-1 for subsequent bleeds that can not be treated under this protocol.
    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 Decision2011-07-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-10-25
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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