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    Summary
    EudraCT Number:2010-020113-85
    Sponsor's Protocol Code Number:CSLCT-BIO-10-67
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2011-06-16
    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 ConcernedFrance - ANSM
    A.2EudraCT number2010-020113-85
    A.3Full title of the trial
    A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to evaluate Plasma-Derived Antihaemophilic Factor/von Willebrand Factor Concentrate (Biostate®) for Immune Tolerance Induction in Male Paediatric Subjects with Haemophilia A (≤2%) who have Developed High-titre Antibodies to Factor VIII (Factor VIII Inhibitors)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. The antibodies are directed against the clotting factor that is given for replacement therapy and usually make therapy unsuccessful. The treatment used in this study is called immune tolerance therapy
    A.3.2Name or abbreviated title of the trial where available
    SWIFT-ITI
    A.4.1Sponsor's protocol code numberCSLCT-BIO-10-67
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/164/2009
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCSL Behring GmbH
    B.1.3.4CountryGermany
    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 supportCSL Behring GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring GmbH
    B.5.2Functional name of contact pointSimone Lang
    B.5.3 Address:
    B.5.3.1Street AddressEmil-von-Behring-Str. 76
    B.5.3.2Town/ cityMarburg
    B.5.3.3Post code35041
    B.5.3.4CountryGermany
    B.5.4Telephone number+496421397060
    B.5.5Fax number+496421393870
    B.5.6E-mailSimone.Lang@cslbehring.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBiostate
    D.3.4Pharmaceutical form Lyophilisate 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 INNHUMAN COAGULATION FACTOR VIII/VON WILLEBRAND FACTOR COMPLEX
    D.3.9.3Other descriptive nameHuman Coagulation factor VIII (+ human von Willebrand factor)
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBiostate
    D.3.4Pharmaceutical form Lyophilisate 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 INNHUMAN COAGULATION FACTOR VIII/VON WILLEBRAND FACTOR COMPLEX
    D.3.9.3Other descriptive nameHuman Coagulation factor VIII (+ human von Willebrand factor)
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    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
    Male paediatric subjects with Haemophilia A who develped high titre antibodies to human coagulation Factor VIII
    E.1.1.1Medical condition in easily understood language
    Treatment of patients with hereditary deficiency of blood coagulation factor VIII that developed neutralizing antibodies against Factor VIII is investigated
    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 14.0
    E.1.2Level LLT
    E.1.2Classification code 10018941
    E.1.2Term Haemophilia NOS
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the proportion of subjects who achieve a complete response (success) following ITI treatment with Biostate.
    E.2.2Secondary objectives of the trial
    Secondary Objective:
    To assess the safety of Biostate when administered to subjects with haemophilia A and inhibitors to FVIII in an ITI treatment regimen.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male subjects diagnosed with haemophilia A (≤2% FVIII level in the absence of factor replacement, according to their medical history).
    2. Age 28 days to <12 years.
    3. ITI treatment can be initiated if:
    a. An inhibitor level of >5 and <200 BU/mL was confirmed in 2 repeated tests by the central laboratory during the screening period and no waiting period is required as judged by the investigator.
    b. An inhibitor level of >5 and <200 BU/mL was confirmed in 2 repeated tests by the central laboratory during the screening period and the inhibitor titre decreased from the peak titre during a waiting period (maximum 11 months) added at the discretion of the investigator.
    c. A subject has pre-existing inhibitors, determined no more than 11 months prior to the Screening visit, which decreased from the peak titre, and an inhibitor level of >5 and <200 BU/mL was confirmed in 2 repeated tests by the central laboratory during the screening period. A waiting period could still be added if the total duration from inhibitor diagnosis until start of ITI treatment (ie, including the screening period) does not exceed 12 months.
    4. The subject and/or his legally acceptable representative understand the nature of the study and have given written informed consent to participate in the study.
    5. Sufficient peripheral venous access or central venous line.
    E.4Principal exclusion criteria
    Exclusion Criteria:
    1. The subject has received ITI previously.
    2. Subjects with a historical peak inhibitor titre of ≥200 BU/mL.
    3. Concomitant treatment with drugs with immunosuppressive side effects (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin or the use of a protein A column or plasmapheresis and interferons.
    4. High risk of cardiovascular, cerebrovascular, or other thromboembolic events (excluding catheter thrombosis) as judged by the investigator.
    5. Subjects who are human immunodeficiency virus (HIV)-1 or HIV-2 positive (as reported in the medical records or determined at screening).
    6. The subject has evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit.
    7. The subject has a known or suspected hypersensitivity or has previous evidence of severe side effects to von Willebrand factor (VWF)/FVIII or FVIII concentrates or human albumin.
    8. The subject has participated in a clinical study or used an investigational compound (eg, a new chemical entity not approved for clinical use) in the past 3 months, unless the study was for haemophilia A and the subject developed an inhibitor (then, a wash-out period of at least 4 weeks must be applied), or is planning to enter such a study during the study period.
    9. Subjects or legal guardians/ representatives with suspected inability (eg, language problems) or unwillingness to comply with study procedures.
    10. The subject has an acute or chronic medical condition other than haemophilia A, which may, in the opinion of the investigator, affect the conduct of the study.
    11. Mental condition rendering the subject (or the subject’s legally acceptable representative) unable to understand the nature, scope and possible consequences of the study).
    12. Any condition that is likely to interfere with evaluation of the investigational medicinal product (IMP) or satisfactory conduct of the study.
    13. Employee at the study site, or spouse/partner or relative of the investigator or subinvestigators.
    Study Product, Dose, and Mode of Administration:
    The IMP Biostate will be intravenously (i.v.) administered at a daily dose of 200 international units (IU)/kg body weight (b.w.), preferably split into 2 doses of 100 IU/kg b.w. per day.
    For subjects with an undetectable inhibitor titre (<0.6 BU/mL) at 2 consecutive assessments, tested at intervals of 2 weeks (±3 days), and normal recovery (≥66% of predicted) for 6-8 weeks, the daily Biostate dose will be reduced by 20 IU/kg b.w. (ie, by 10% of the initial daily ITI dose), if possible, every 2-4 weeks down to a dose of 100 IU/kg b.w., provided the recovery remains normal during that time (assessed weekly).
    After further gradual reduction of the dose and extension of the administration interval (at the discretion of the investigator), Biostate will be administered as prophylaxis at a daily dose of 50 IU/kg b.w. on 3 days per week (about every second day).
    Any daily dose >100 IU/kg b.w. should preferably be evenly split into 2 doses per day. Daily doses of 100 IU/kg b.w. or lower will be administered once daily.
    Bleeding complications during the tolerization period should be treated according to centre’s standard of care. Study treatment should not be interrupted for surgical procedures.
    E.5 End points
    E.5.1Primary end point(s)
    Study endpoints:
    Efficacy endpoints:
    • Response to ITI treatment: complete response (success), partial response (partial success), or ITI failure.
    • FVIII inhibitor titre.
    • Time to complete response (success).
    • Time to inhibitor <0.6 BU/mL for the first time.
    Safety endpoints:
    • Adverse events (AEs).
    • Clinical laboratory testing (haematology, biochemistry).
    • Thrombogenic risk assessment: markers of activation of coagulation (eg, fibrin fragments, thrombin-antithrombin complex [TAT]).
    • Frequency and nature of bleeding events.
    • Catheter-related complications (eg, line infections).
    • Physical examination.
    • Vital signs (blood pressure, heart rate, and body temperature).
    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 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.8.3 The trial involves single site in the Member State concerned Information not present in EudraCT
    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 EEA14
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months5
    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: 20
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 13
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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 study participation the patients will be treated with the current standard therapy on the discretion of their treating doctor.It is expected at the end of the study Biostate will be licensed.

    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-10-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-06-12
    P. End of Trial
    P.End of Trial StatusOngoing
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