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    Summary
    EudraCT Number:2007-006081-15
    Sponsor's Protocol Code Number:Sym001-03
    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:2008-02-07
    Trial 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 number2007-006081-15
    A.3Full title of the trial
    A multi-centre, single intravenous dose, exploratory dose-finding, open label trial on the safety and efficacy of Sym001 in the treatment of Immune Thrombocytopenic Purpura (ITP) in RhD positive, non-splenectomized adult subjects
    A.3.2Name or abbreviated title of the trial where available
    N/A
    A.4.1Sponsor's protocol code numberSym001-03
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberN/A
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSymphogen A/S
    B.1.3.4CountryDenmark
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 namesym001
    D.3.2Product code sym001
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codesym001
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.6
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    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
    Immune Thrombocytopenic Purpura (ITP) in RhD positive, non-splenectomized adult subjects
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10043561
    E.1.2Term Thrombocytopenic purpura
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety of Sym001 following a single intravenous dose, at multiple dose levels, in the treatment of ITP in RhD positive, non-splenectomized adult subjects
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of Sym001 following a single intravenous dose in the treatment of ITP in RhD positive, non-splenectomized adult subjects.
    • To evaluate the binding of Sym001 to RBCs following a single intravenous dose of Sym001 in the treatment of ITP in RhD positive, non-splenectomized adult subjects.
    • To evaluate an appropriate dose of Sym001 for Phase 3.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Informed consent obtained before any trial-related procedures.
    2. Age >18 years.
    3. Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit. In each cohort< 200 µg/kg a maximum of 3 subjects will be included with platelet counts < 10,000/mm3, and in cohorts > 200 µg/kg a maximum of 1 subject with platelet counts < 10,000/mm3 will be included in order to limit variability. Two individual pre-dose platelet counts taken on the dosing day within > 1 hour interval between each other and both being < 30,000/mm3 will be used to confirm thrombocytopenia.
    4. History of isolated ITP (thrombocytopenia with no known aetiology, blood smear showing normal appearing platelets or, if performed, a bone marrow showing adequate thrombopoïesis and normal erythroid and myeloid morphology).
    5. RhD-positive serology.
    6. Previous treatment and response to first line therapy for ITP (corticosteroids, anti-D or IVIg), with response being defined as an increase in platelet count to ≥30,000/mm3.
    7. If female and of child-bearing potential, subject has a negative pregnancy test at screening.
    8. If subject is a female of child-bearing potential, subject agrees to use a medically accepted form of contraception from the time of enrolment (at screening) to completion of all follow-up trial visits.
    E.4Principal exclusion criteria
    Known clinical picture suggestive of other causes of thrombocytopenia, especially systemic lupus erythematosus, antiphospholipid syndrome, Evans syndrome, immunodeficiency states, lymphoproliferative disorders, liver disease, ingestion of drugs such as quinidine/quinine, heparin and sulfonamides and hereditary thrombocytopenia confirmed by relevant laboratory findings.
    2. Suspected infection with HIV, hepatitis C, H. pylori unless corresponding laboratory test are negative.
    3. Clinical splenomegaly (the spleen should not be palpable at more than 1 finger breadth below the costal margin).
    4. History of abnormal bone marrow examination (except ITP-typical megacaryocytosis).
    5. At pre-dose visit: an ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale.
    6. History of anaphylaxis or hypersensitivity reactions following anti-D treatment.
    7. Current immune haemolytic anaemia.
    8. Underlying haemolytic condition (e.g. reticulocyte count > 3%).
    9. Planned surgery during the 15 days post dosing.
    10. Haemoglobin pre-dose value lower than 2.0 g/dL below the lower limit of the laboratory normal range for gender and age.
    11. History of splenectomy.
    12. Known current malignancy (except basal cell carcinoma).
    13. Received other investigational agent within 3 months prior to enrolment.
    14. Positive DAT (direct Coombs-test) at screening unless subject has received treatment with IVIg or anti-D products within 3 months prior to screening with prior negative DAT.
    15. Known non-responders to most recent anti-D treatment (despite any initial response to treatment).
    16. Any other current treatment for ITP except corticosteroids (Prednisone or Dexamethasone) at doses equivalent to ≤30 mg prednisone/day if the daily dose has been constant for 2 weeks or more before trial drug administration.
    17. Therapy with IVIg within 2 weeks prior to enrollment or with anti-D or any other treatment of ITP within 4 weeks prior to enrolment.
    18. Therapy with tranexamic acid, alkylating agents or any anti-CD20 antibodies within 8 weeks prior to enrolment
    19. Any antithrombotic treatment (except acetyl salicylic acid at doses up to 150 mg daily) within 7 days prior to pre-dosing visit or planned during the trial.
    20. PT/INR and aPTT out of normal range at the pre-dose visit.
    21. History of venous or arterial thrombosis or know thrombophilia.
    22. Diseases other than ITP that may influence the result of the trial as judged by the Investigator.
    23. Creatinine 25% or more above normal range value, alanine aminotransferase (ALT) and alkaline phosphatase (ALP) 100% above normal range value, and albumin 25% or more below normal range value (according to the local laboratory) at pre-dose visit.
    24. Current or planned treatment with erythropoietin.
    25. Subject is pregnant, breast feeding or intends to become pregnant.
    26. Previous dosing with Sym001 in this trial.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence and severity of AEs, including SAEs and Adverse Events of Special Interest (AESIs) during the 6-week trial period.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    E.7.1.3.1Other trial type description
    exploratory dose-finding study
    E.7.2Therapeutic exploratory (Phase II) Yes
    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 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    6 weeks after Sym001 has been administered to the last patient
    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 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 months6
    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.1.1.1In 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.1.3Elderly (>=65 years) Yes
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 66
    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)
    There are no plans for treatment or care after the subject has ended his/her participation in the trial. However it would be investigators discretion to provide additional treatment if need be.
    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 Decision2008-05-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-04-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2011-08-08
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