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    Summary
    EudraCT Number:2022-001940-36
    Sponsor's Protocol Code Number:TAK-755-2001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-12-22
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2022-001940-36
    A.3Full title of the trial
    A Phase 2b, multicenter, randomized, double-blind study of safety and efficacy of TAK-755 (rADAMTS13) with minimal to no plasma exchange (PEX) in the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP)
    Estudio de fase IIb, multicéntrico, aleatorizado y con doble enmascaramiento de la seguridad y la eficacia de TAK-755 (rADAMTS13) con plasmaféresis (PF) mínima o nula en el tratamiento de la púrpura trombocitopénica trombótica inmunitaria (PTTi)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2b study of TAK-755 (rADAMTS13) with minimal to no PEX in the treatment of patients with iTTP
    Estudio de fase IIb de TAK-755 (rADAMTS13) con PF mínima o nula en el tratamiento de pacientes con PTTi
    A.4.1Sponsor's protocol code numberTAK-755-2001
    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 SponsorTakeda Development Center Americas, Inc.
    B.1.3.4CountryUnited States
    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 supportTakeda Development Center Americas, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Development Center Americas, Inc.
    B.5.2Functional name of contact pointJose Gonzalez
    B.5.3 Address:
    B.5.3.1Street AddressPaseo de la Castellana, 95
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28046
    B.5.3.4CountrySpain
    B.5.4Telephone number34917904461
    B.5.6E-mailJose.Gonzalez@takeda.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 (rADAMTS13)
    D.3.2Product code TAK-755
    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 INNApadamtase alfa
    D.3.9.2Current sponsor codeTAK-755
    D.3.9.3Other descriptive nameBAX930 - RECOMBINANT HUMAN ADAMTS13
    D.3.9.4EV Substance CodeSUB195523
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1500
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder and solution for solution for injection
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    immune-mediated thrombotic thrombocytopenic purpura (iTTP)
    púrpura trombocitopénica trombótica inmunitaria (PTTi)
    E.1.1.1Medical condition in easily understood language
    immune blood disorder
    trastorno inmunológico de la sangre
    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 20.0
    E.1.2Level SOC
    E.1.2Classification code 10005329
    E.1.2Term Blood and lymphatic system disorders
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the safety of TAK-755 in the treatment of iTTP
    Evaluar la seguridad de TAK-755 en el tratamiento de la PTTi
    E.2.2Secondary objectives of the trial
    1. Assess the efficacy of TAK-755 in achieving clinical response with and without on-study PEX following an acute TTP event.
    2. Assess treatment failures.
    3. Assess the efficacy of TAK-755 in prevention of iTTP recurrence, exacerbation, and relapse.
    4. Assess changes in organ damage biomarkers.
    5. Evaluate the durability of clinical response.
    6. Evaluate the ADAMTS13 antigen and activity over time.
    7. Evaluate VWF antigen and activity over time.
    1. Evaluar la eficacia de TAK-755 en la consecución de una respuesta clínica con y sin PF durante el estudio después de un acontecimiento agudo de púrpura trombocitopénica trombótica (PTT).
    2. Evaluar los fracasos de tratamiento.
    3. Evaluar la eficacia de TAK-755 en la prevención de la recidiva de la PTTi, la reagudización y la recaída.
    4. Evaluar los cambios en los biomarcadores de daño orgánico.
    5. Evaluar la durabilidad de la respuesta clínica.
    6. Evaluar el antígeno y la actividad de ADAMTS13 a lo largo del tiempo.
    7. Evaluar el antígeno y la actividad del factor de von Willebrand (VWF) con el tiempo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must provide a signed informed consent form. A fully recognized proxy may be used per local laws for subjects unable to provide consent.
    2. Subject is 18 years or older at time of screening.
    3. Subject has been diagnosed with de novo or relapsed iTTP based on the following criteria:
    a. Thrombocytopenia [platelet count <100,000/μL].
    b. Microangiopathic hemolytic anemia [elevation of LDH >2×ULN or by the presence or an increase of schistocytes in peripheral blood smear].
    4. Subject must be willing to fully comply with study procedures and requirements.
    5. Female subjects of childbearing potential must present with a negative pregnancy test and agree to employ highly effective birth control measures for duration of study. Sexually active male subjects must agree to use an effective method of contraception for the duration of the study.
    1. El participante debe proporcionar un documento de consentimiento informado firmado. Se puede usar un representante totalmente reconocido para los participantes que no pueden dar su consentimiento.
    2. El participante tiene 18 años o más en el momento de la selección.
    3. Al participante se le ha diagnosticado PTTi de novo o presenta una recidiva de acuerdo con los siguientes criterios:
    a. Trombocitopenia (recuento de plaquetas <100 000/μl).
    b. Anemia hemolítica microangiopática [aumento de la LDH >2 × LSN o presencia o un aumento de esquistocitos en frotis de sangre periférica].
    4. El participante debe estar dispuesto a cumplir completamente con los procedimientos y requisitos del estudio.
    5. Las participantes en edad fértil deben presentar una prueba de embarazo negativa y acceder a utilizar métodos anticonceptivos muy eficaces durante todo el estudio. Los hombres sexualmente activos deben aceptar el uso de un método anticonceptivo eficaz durante todo el estudio.
    E.4Principal exclusion criteria
    1. Subject has received more than 2 pre-study PEX prior to randomization.
    2. Subject has been diagnosed with cTTP or another cause of TMA, including: disseminated intravascular coagulation (DIC), disseminated malignancy, malignant hypertension, solid organ and hematopoietic stem cell transplantation, shiga toxin–related and atypical hemolytic uremic syndrome, drug toxicity (eg, gemcitabine, mitomycin C, clopidogrel) and pregnancy-related thrombocytopenia syndromes (eg, hemolysis, elevated liver enzymes and low platelets [HELLP]; eclampsia), serum creatinine >2.25 mg/dL, active cancer within the last year.
    3. Subject has been exposed to another investigational product within 30 days prior to enrollment or is scheduled to participate in another clinical study involving investigational product or investigational device during the course of the study.
    4. Subject has received caplacizumab within 30 days prior to study enrollment.
    5. Subject has had a previous iTTP event within the past 30 days.
    6. Subject is positive for human immunodeficiency virus (HIV) with unstable disease or CD4+ count ≤200 cells/mm3 within 3 months of screening.
    7. Subject has condition of severe immunodeficiency.
    8. Subject has a severe systemic acute infection.
    9. Subject has another underlying progressive fatal disease and/or life expectancy <3 months.
    10. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
    11. Subject suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude. However, a fully recognized medical proxy will be permitted to provide consent, under the circumstances that the subject is unable to provide consent due to the effects of iTTP.
    12. Subject is pregnant or lactating.
    13. Subject is a family member or employee of the sponsor or investigator.
    14. Subject has any condition in which methylprednisolone or other streroide equivalent is contraindicated as per prescribing information.
    15.Subject has known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS13, CHO cell proteins, or other constituents of TAK-755.
    1. El participante ha recibido más de 2 PF previas al estudio antes de la aleatorización.
    2. El participante con diagnóstico de PTTh u otra causa de microangiopatía trombótica (MAT), como por ejemplo: coagulación intravascular diseminada (CID), neoplasia maligna diseminada, hipertensión maligna, trasplante de víscera maciza o de células madre hematopoyéticas, síndrome hemolítico urémico atípico y relacionado con la toxina Shiga, toxicidad farmacológica (p. ej., gemcitabina, mitomicina C, clopidogrel) y síndromes de trombocitopenia relacionados con el embarazo (p. ej., hemólisis, niveles elevados de enzimas hepáticas y bajas plaquetas [HELLP]; eclampsia), creatinina sérica >2,25 mg/dl, cáncer activo en el último año.
    3. El participante ha estado expuestos a otro producto en investigación en los 30 días anteriores a la inscripción o está programado que participen en otro estudio clínico con un producto en investigación o un dispositivo en investigación durante el transcurso del estudio.
    4. El participante ha recibido caplacizumab en los 30 días anteriores a la inscripción en el estudio.
    5. El participante ha sufrido un acontecimiento de PTTi en los últimos 30 días.
    6. El participante es positivo para el virus de la inmunodeficiencia humana (VIH) con enfermedad inestable o recuento de CD4+ ≤200 células/mm3 en los 3 meses anteriores a la selección.
    7. El participante presenta una afección de inmunodeficiencia grave.
    8. El participante tiene una infección aguda sistémica grave.
    9. El participante tiene otra enfermedad mortal progresiva subyacente y/o una esperanza de vida <3 meses.
    10. El investigador determina que el participante no es capaz de colaborar en las actividades del estudio o no está dispuesto a hacerlo.
    11. El participante padece una enfermedad mental que lo incapacita para entender la naturaleza, el alcance y las posibles consecuencias del estudio, o bien muestra indicios de una actitud poco colaboradora. Sin embargo, se permitirá que un representante médico totalmente reconocido dé su consentimiento, en las circunstancias en las que el participante es incapaz de dar su consentimiento debido a los efectos de la PTTi.
    12. La participante está embarazada o en periodo de lactancia.
    13. El participante es pariente o empleado del promotor o del investigador.
    14. El participante padece alguna afección en la que la metilprednisolona, u otro corticosteroide equivalente, está contraindicada según la información de prescripción.
    15. El participante tiene una reacción de hipersensibilidad conocida potencialmente mortal, incluida la anafilaxia, a la molécula original ADAMTS13, proteínas de las células ováricas de hámster chino o a otros componentes de TAK-755.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of AEs, SAEs, and AESIs after receiving any dose of IP
    Incidencia de acontecimientos adversos (AA), acontecimientos adversos graves (AAG) y acontecimientos adversos de especial interés (AAEI) tras recibir cualquier dosis del PEI
    E.5.1.1Timepoint(s) of evaluation of this end point
    Variable time point
    Punto de tiempo variable
    E.5.2Secondary end point(s)
    1.Achievement of clinical response without on-study PEX.
    2.Achievement of clinical response with on-study PEX
    3.Time to clinical response.
    4.Occurrence of refractoriness.
    5.Time to first on-study PEX in subjects who achieved clinical response.
    6.Number of days of PEX and total volume of plasma administered.
    7.Occurrence of treatment failure.
    8.Occurrence of iTTP recurrence, exacerbation, or relapse.
    9.Time to iTTP recurrence, exacerbation, or relapse.
    10.Occurrence of any one of the following events: clinical recurrence, iTTP-related death, or major thromboembolic event from time of first IP administration through study completion.
    11.Time to occurrence of any one of the following events: clinical recurrence, iTTP-related death, or major thromboembolic event from
    time of first IP administration through study completion.
    12.Organ damage biomarkers change from baseline at (1) clinical response and (2) study completion
    • LDH
    • Troponin
    13.Achievement of clinical remission (maintaining clinical response for ≥30 days).
    14.ADAMTS13 antigen and activity levels resulting from TAK-755 administration in acute and post-acute phases.
    15.VWF antigen and activity levels resulting from TAK-755 administration in acute and post-acute phases.
    1. Logro de la respuesta clínica sin PF durante el estudio.
    2. Consecución de la respuesta clínica con la PF durante el estudio.
    3. Tiempo hasta la respuesta clínica.
    4. Aparición de resistencia.
    5. Tiempo hasta la primera PF durante el estudio en los participantes que hayan alcanzado una respuesta clínica.
    6. Número de días de PF y volumen total de plasma administrado.
    7. Aparición de fracaso del tratamiento.
    8. Incidencia de la recurrencia, reagudización o recidiva de la PTTi.
    9. Tiempo hasta la recurrencia, reagudización o recidiva de la PTTi.
    10. Aparición de cualquiera de los siguientes acontecimientos: recurrencia clínica, muerte relacionada con la PTTi o acontecimiento tromboembólico importante desde el momento de la primera administración del producto en investigación hasta la finalización del estudio.
    11. Tiempo hasta la aparición de cualquiera de los siguientes acontecimientos: recurrencia clínica, muerte relacionada con la PTTi o acontecimiento tromboembólico importante desde el momento de la primera administración del producto en investigación hasta la finalización del estudio.
    12. Cambio en los biomarcadores de daño orgánico desde el inicio (1) en la respuesta clínica y (2) finalización del estudio
    • LDH
    • Troponina
    13. Consecución de la remisión clínica (que mantenga la respuesta clínica durante ≥30 días).
    14. Niveles de antígeno e actividad de ADAMTS13 derivados de la administración de TAK-755 en las fases aguda y posaguda.
    15. Los niveles de antígeno y actividad del VWF resultantes de la administración de TAK-755 en las fases aguda y posaguda.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Variable time points
    Punto de tiempo variable
    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 Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 Yes
    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 trial2
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    Argentina
    Canada
    Israel
    United States
    Austria
    France
    Poland
    Spain
    Switzerland
    Germany
    Greece
    Italy
    United Kingdom
    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
    LSLV
    última visita del último sujeto
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days12
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days12
    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.1.2.1Number of subjects for this age range: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Proxy will be permitted to provide consent, under the circumstances that the subject is unable to provide consent due to the effects of iTTP.
    Se permitirá que el apoderado brinde su consentimiento, en las circunstancias en que el sujeto no pueda brindar su consentimiento debido a los efectos de la PTTi .
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 40
    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)
    No aftercare is planned for this study.
    No se planean cuidados posteriores para este estudio.
    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 Decision2023-02-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-02-10
    P. End of Trial
    P.End of Trial StatusOngoing
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