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    Summary
    EudraCT Number:2012-003221-19
    Sponsor's Protocol Code Number:281101
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-12-07
    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 ConcernedGermany - PEI
    A.2EudraCT number2012-003221-19
    A.3Full title of the trial
    BAX 930 (rADAMTS13)
    A PHASE 1 PROSPECTIVE, UNCONTROLLED, OPEN-LABEL, MULTICENTER, DOSE-ESCALATION STUDY EVALUATING THE SAFETY AND PHARMACOKINETICS IN HEREDITARY TTP (THROMBOTIC THROMBOCYTOPENIC PURPURA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 1 clinical study to assess safety of single infusions of rADAMTS13 (BAX930) in humans and to evaluate distribution and elimination of rADAMTS13 (BAX930) from the human body after administration at 3 different dose levels in patients diagnosed with severe hereditary TTP
    A.3.2Name or abbreviated title of the trial where available
    Phase 1 dose escalation, single dose, to assess safety and PK of BAX930 in hTTP
    A.4.1Sponsor's protocol code number281101
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/48/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxalta 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 supportBaxalta Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta Innovations GmbH
    B.5.2Functional name of contact pointManfred Rieger
    B.5.3 Address:
    B.5.3.1Street AddressDonau City Strasse 7
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1221
    B.5.3.4CountryAustria
    B.5.4Telephone number+431201002473438
    B.5.5Fax number+43120100717
    B.5.6E-mailManfred.Rieger@baxalta.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/3/08/588
    D.3 Description of the IMP
    D.3.1Product nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
    D.3.2Product code BAX930
    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 INNRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
    D.3.9.2Current sponsor codeBAX930
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number40
    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
    Subjects diagnosed with hereditary thrombotic thrombocytopenic purpura (TTP)
    E.1.1.1Medical condition in easily understood language
    Subjects diagnosed with hereditary thrombotic thrombocytopenic purpura (TTP)
    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 18.0
    E.1.2Level LLT
    E.1.2Classification code 10043562
    E.1.2Term Thrombocytopenic purpura, thrombotic
    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
    To evaluate the safety of BAX930 following single infusions at doses of 5, 20, and 40 U/kg BW, including the occurrence of adverse events (serious and non-serious) and formation of binding and inhibitory antibodies to BAX 930.
    E.2.2Secondary objectives of the trial
    -To evaluate the pharmacokinetics of BAX 930 following single infusions of rADAMTS13 at doses of 5, 20, and 40 U/kg BW
    -To evaluate the effect of BAX930 on plasma VWF levels and multimeric patterns
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Subject is between 18 and 65 years of age, inclusive.
    -The subject has a documented diagnosis of severe hereditary ADAMTS13 deficiency
    -Cryoprecipitate, FFP, or other ADAMTS13 containing products interfering with ADAMTS13 PK have to be paused at least 10 days prior infusion of the IP.
    -The subject is not displaying any severe TTP symptoms at screening.
    -Subjects ≥18 years of age have a Karnofsky score ≥ 60%.
    -If female of childbearing potential, subject presents with a negative serum pregnancy test and agrees to employ adequate birth control measures for the duration of the study.

    For the entire list of inclusion criteria refer to protocol section 9.1
    E.4Principal exclusion criteria
    -The subject has been diagnosed with any other TTP-like disorder (for eg, microangiopathic hemolytic anemia), including acquired TTP.
    -The subject has a medical history or presence of a functional neutralizing ADAMTS13 inhibitor at screening.
    -The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/mild asthma, food allergies or animal allergies.
    -The subject has a medical history of hematological disorders, in particular systemic lupus erythematosus, amyloidosis, antiphospholipid antibody syndrome, vasculitis, other hemolytic anemia, disseminated intravascular coagulation, and systemic scleroderma.
    -The subject has a history of significant neurological events, such as major stroke, indicating that a relapse might have severe consequences, as judged by the investigator.
    -The subject has been diagnosed with a cardiovascular disease [New York Heart Association (NYHA) classes 3-4].
    -The subject has been diagnosed with severe liver disease, as evidenced by, but not limited to, any of the following: serum ALT 3 times the upper limit of normal, INR > 1.5, hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices).
    -The subject has been diagnosed with severe glomerular disease, with gross proteinuria and a serum creatinine level ≥ 2.5 mg/dL.
    -If female, subject is pregnant or lactating at the time of study enrollment.

    For the entire list of inclusion criteria refer to protocol section 9.2
    E.5 End points
    E.5.1Primary end point(s)
    Occurrence of adverse events (serious and non-serious), including the incidence of binding and inhibitory antibody formation, occurring up to 28 ± 3 days after the last investigational product infusion.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At specified timepoints following IP infusion and at study completion (28 ± 3 days after the last investigational product infusion)in each dose cohort.
    E.5.2Secondary end point(s)
    -Standard pharmacokinetic parameters for ADAMTS13 activity and ADAMTS13:Ag after single infusions of rADAMTS13 in Dose Cohorts 1, 2 and 3;
    -Measurement of plasma VWF:RCo, VWF:Ag and VWF structure analysis prior to and following a single infusion of rADAMTS13.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At specified timepoints following IP infusion and at study completion (28 ± 3 days after the last investigational product infusion) in each dose cohort.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    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) Yes
    E.7.1.1First administration to humans Yes
    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) 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 Yes
    E.8.1.7.1Other trial design description
    Dose escalation
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Japan
    Switzerland
    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 years0
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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 Yes
    F.1.1Number of subjects for this age range: 3
    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: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Adolescents
    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 8
    F.4.2.2In the whole clinical trial 14
    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)
    None
    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 Decision2015-03-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-03
    P. End of Trial
    P.End of Trial StatusCompleted
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