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    Summary
    EudraCT Number:2011-006329-42
    Sponsor's Protocol Code Number:CAD0111
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-10-30
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2011-006329-42
    A.3Full title of the trial
    Short course of Bortezomib in anemic patients with refractory cold agglutinin disease
    Ciclo breve di Bortezomib in pazienti anemici con crioagglutinemia refrattaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Short treatment with a drug for patients affected by a type of a treatment-resistant anaemia, where the red cells are destroyed by some cold cells (autoantibodies).
    Breve trattamento con un farmaco per pazienti con una forma di anemia che non si risolve con i normali trattamenti, in cui sono distrutti dei globuli rossi, a causa della presenza di alcune cellule (autoanticorpi) fredde.
    A.4.1Sponsor's protocol code numberCAD0111
    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 SponsorG.I.M.E.M.A. GRUPPO ITALIANO MALATTIE EMATOLOGICHE DELL'ADULTO
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportA.I.L. Associazione Italiana contro le Leucemie
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportJanssen-Cilag
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione G.I.M.EM.A.
    B.5.2Functional name of contact pointCentro Dati
    B.5.3 Address:
    B.5.3.1Street Addressvia Casilina,5
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00182
    B.5.3.4CountryItaly
    B.5.4Telephone number0670390526
    B.5.5Fax number0670390540
    B.5.6E-mailgimema@gimema.it
    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 Yes
    D.2.1.1.1Trade name VELCADE*EV 1FL 3,5MG 1MG/ML
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.4Pharmaceutical form Powder for infusion
    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.3.9.1CAS number 179324-69-7
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.3
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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
    Anaemia with refractory cold agglutinin disease
    Anemia con crioagglutinemia refrattaria
    E.1.1.1Medical condition in easily understood language
    Type of red cell-destruction anaemia caused by cold autoantibodies. The refractory state is due to the lack of recovery from anaemia after first-line treatment.
    Forma di anemia da distruzione dei globuli rossi causata da autoanticorpi freddi. La refrattarietà è dovuta alla mancanza di risoluzione dell’anemia dopo trattamenti di prima linea.
    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 PT
    E.1.2Classification code 10009868
    E.1.2Term Cold type haemolytic anaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the trial is to evaluate the achievement of transfusion independence after Bortezomib therapy or of a significant rise of hemoglobin concentration in patients with moderate to severe anemia related to CAD.
    L’obiettivo primario dello studio è valutare il raggiungimento dell’indipendenza da trasfusione dopo la terapia con Bortezomib o di un aumento significativo della concentrazione di emoglobina in pazienti con anemia da moderata a grave correlata a criogglutinemia (CAD, cold agglutinin disease).
    E.2.2Secondary objectives of the trial
    - Evaluation of Bortezomib therapy in terms of safety. - Evaluation of the duration of transfusion independence. - Evaluation of the effects of treatment on the underlying clonal B cell disorder.
    - Valutazione della terapia con Bortezomib in termini di sicurezza; - Valutazione della durata dell’indipendenza da trasfusione; - Valutazione degli effetti del trattamento sulle patologie correlate alle sottostanti linee cellulari B.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patients with chronic cold agglutinin disorder requiring transfusion or with a hemoglobin concentration below 10g/L determined at least monthly during the two months before entering the trial; - Failure of at least one previous treatment attempt; - Hemoglobin level assessment; - Cold agglutinin (CA) titer at 4°C of 64 or higher; - Written informed consent.
    - Pazienti con patologia di crioagglutinemia che richieda una trasfusione o con una concentrazione di emoglobina al di sotto di 10g/L determinata almeno mensilmente durante i due mesi precedenti l’entrata nello studio; - Fallimento o almeno un precedente tentativo di trattamento; - Valutazione dei livelli di emoglobina; - Titolo della crioagglutinina a 4°C ≥ 64; - Consenso informato scritto.
    E.4Principal exclusion criteria
    - Presence of a concomitant lymphoproliferative disorder requiring specific treatment for reasons other than cold agglutinin related hemolytic anemia. - Preexisting peripheral neuropathy. - Known hypersensitivity to Bortezomib. - Non-cooperative behaviour or non-compliance. - Psychiatric diseases or conditions that might impair the ability to give informed consent. - Patients who are pregnant (women of childbearing potential must have a negative serum pregnancy test). Post-menopausal women must be amenorrhoic for at least 24 months to be considered of non-childbearing potential. Male and female patients must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug
    - Presenza di una concomitante patologia linfoproliferativa, richiedente un trattamento specifico per ragioni diverse dall’anemia emolitica correlata alla crioagglutinemia; - Preesistente neuropatia periferica; - Conosciuta ipersensibilità al Bortezomib; - Comportamento non collaborativo o mancata aderenza alla terapia; - Disordini psichiatrici o condizioni che possano compromettere la capacità di esprimere il proprio consenso informato. - Donne in gravidanza. (Le donne potenzialmente fertili devono eseguire un test di gravidanza che risulti negativo). Le donne in menopausa non devono aver avuto il ciclo mestruale nei 24 mesi prima dell’inizio della terapia per essere considerate come non potenzialmente fertili. Uomini e donne devono essere d’accordo ad utilizzare una barriera contraccettiva efficace durante lo svolgimento dello studio e per 3 mesi dopo l’ultima somministrazione del farmaco sperimentale.
    E.5 End points
    E.5.1Primary end point(s)
    Cumulative proportion of patients transfusion-free at three months after Bortezomib therapy or, in non-transfused patients, with a > 2g hemoglobin rise compared to baseline.
    Proporzione cumulata di pazienti liberi da trasfusione a 3 mesi dopo terapia con bortezomib o, nei pazienti non trasfusi, con un aumento di emoglobina > 2g paragonata al basale.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During and at the end of the study
    Durante e alla fine dello studio
    E.5.2Secondary end point(s)
    • Evaluation of the incidence of CTC grade 3 and 4 adverse events during and after treatment. • Duration of transfusion independence • Effect of treatment on the underlying clonal B cell disorder.
    • Valutazione dell'incidenza degli eventi avversi di CTC di grado 3 e 4 durante e alla fine del trattamento • Durata dell'indipendenza da trasfusione • Effetto del trattamento sulla patologia delle sottostanti linee cellulari B.
    E.5.2.1Timepoint(s) of evaluation of this end point
    During and at the end of the study
    Durante e alla fine dello studio
    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 No
    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) 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 concerned12
    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 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
    LVLS
    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 months18
    E.8.9.1In the Member State concerned 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: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 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 state25
    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)
    NOT Applicable
    NOT Applicable
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation G.I. M.EM.A.
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-10-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-10-02
    P. End of Trial
    P.End of Trial StatusCompleted
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