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    The EU Clinical Trials Register currently displays   37756   clinical trials with a EudraCT protocol, of which   6186   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2012-003101-10
    Sponsor's Protocol Code Number:CD-ON-CAT8015-1036
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-06-10
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2012-003101-10
    A.3Full title of the trial
    A Phase 2, Multicenter, Single-arm Study of Moxetumomab Pasudotox in Pediatric Subjects with Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin
    Estudio de fase 2, multicéntrico y de un solo brazo de moxetumomab pasudotox en pacientes pediátricos con leucemia linfoblástica aguda infantil o linfoma linfoblástico B refractarios o en recidiva
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Multicenter Study in Pediatric Subjects with Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma
    Estudio de fase 2, multicéntrico en pacientes pediátricos con leucemia linfoblástica aguda infantil o linfoma linfoblástico B refractarios o en recidiva
    A.4.1Sponsor's protocol code numberCD-ON-CAT8015-1036
    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 SponsorMedImmune, LLC
    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 supportMedImmune, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedImmune, LLC
    B.5.2Functional name of contact pointClinical Trial Enquiries
    B.5.3 Address:
    B.5.3.1Street AddressOne MedImmune Way
    B.5.3.2Town/ cityGaithersburg, Maryland
    B.5.3.3Post code20878
    B.5.3.4CountryUnited States
    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 nameMoxetumomab Pasudotox
    D.3.2Product code CAT-8015
    D.3.4Pharmaceutical form Solution 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.3.8INN - Proposed INNMoxetumomab Pasudotox
    D.3.9.1CAS number 1020748-57-5
    D.3.9.2Current sponsor codeCAT-8015
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number0.85
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin
    Leucemia linfoblástica aguda infantil o linfoma linfoblástico B refractarios o en recidiva
    E.1.1.1Medical condition in easily understood language
    Pediatric Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma
    Leucemia linfoblástica aguda infantil o linfoma linfoblástico
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level LLT
    E.1.2Classification code 10000845
    E.1.2Term Acute lymphoblastic leukemia
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level LLT
    E.1.2Classification code 10003939
    E.1.2Term B-lymphoblastic lymphoma (Kiel Classification)
    E.1.2System Organ Class 100000004864
    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 efficacy defined as Composite Complete Response (CRc)
    Evaluar la eficacia definida como respuesta completa combinada (RCc)
    E.2.2Secondary objectives of the trial
    - Assess safety and tolerability
    - Assess immunogenicity and pharmacokinetics
    - Progression-free survival (PFS), overall survival (OS), Duration of complete response (DOCR), Duration of overall response (DOR)
    - Minimal residual disease negative CRc rate
    - Number eligible for stem cell transplant
    - Overall response rate (ORR)
    - Hematologic activity
    - Evaluar la seguridad y tolerabilidad
    - Evaular la ihnmunogenicidad y la farmacocinética
    - Supervivencia si progresión (SSP), supervivencia global (SG), duración de la respuesta completa (DRC), duración de la respuesta global (DRG)
    - Tasa de enfermedad residual mínima sin ERM
    - Número eligible para transplante de células madre
    - Tasa de respuesta global (TRG)
    - Actividad hematológica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria:
    1) Between the ages of >/= 6 months and < 18 years of age
    2) Written informed consent and written informed assent (if applicable)
    3) Must have histologically proven B-cell ALL or B-cell lymphoblastic lymphoma
    4) Measurable or evaluable disease
    5) Disease status:
    - Subjects must have relapsed or refractory disease and received at least 1 standard chemotherapy and either 1 salvage regimen or allogeneic HSCT.
    - In event of relapse after prior allogeneic HSCT, subjects must be at least 3 months post-transplant, have no evidence of active graft-vs-host disease, and been off immunosuppression for at least 4 weeks
    - Must have resolution of the acute toxic effects to </= Grade 2 from prior chemotherapy before entry, in the opinion of the investigator
    6) For non-leukemic subjects, an absolute neutrophil count (ANC) > 1000/µL, and platelet count > 50,000/mm2 are required, unless cytopenias are judged by the investigator to be due to underlying disease (ie, potentially reversible with anti-neoplastic therapy).
    7) Adequate Performance status.
    8) Subjects with central nervous system (CNS) status 1 or 2 are eligible only in the absence of neurologic symptoms, such as cranial nerve palsy, suggestive of CNS leukemia.
    9) Sexually active subjects must agree to use an approved method of contraception or abstinence to meet eligibility criteria.
    Los sujetos deben presentar todos los criterios siguientes:
    1) Edad >/= 6 meses y < 18 años en el momento de la selección.
    2) Otorgamiento del consentimiento informado y de toda autorización que se exija
    3) Leucemia linfoblástica aguda de células B o linfoma linfoblástico de células B con afectación medular, confirmado mediante examen histopatológico
    4) Enfermedad evaluable o mensurable.
    5) Estado de la enfermedad:
    - Recidiva o resistencia al tratamiento después de haber recibido al menos una quimioterapia convencional además de una pauta de rescate o un trasplante alogénico de progenitores hematopoyéticos.
    - En caso de recidiva después de un alo-TPH, deben haber transcurrido >/= 3 meses del trasplante; no debe haber signos de enfermedad del injerto contra el huésped; y el sujeto debe llevar >/= 4 semanas sin inmunosupresores.
    - Resolución de los efectos tóxicos agudos de la última quimioterapia a grado </= 2 antes de la inclusión, a juicio del investigador.
    6) Únicamente en el caso de los sujetos que no leucémicos (o sea, con linfoma), cifra de neutrófilos (CAN) >/= 1000 /µL y cifra de plaquetas > 50.000 /mm2, a menos que el investigador considere que las citopenias obedecen al proceso neoplásico (o sea, que pueden corregirse con el tratamiento antineoplásico).
    7) Estado funcional adecuado
    8) Podrán participar los sujetos que presenten las categorías relativas al sistema nervioso central 1 ó 2 (SNC) únicamente si no presentan síntomas neurológicos indicativos de leucemia del SNC, como una parálisis de pares craneales:
    9) Para cumplir los criterios de inclusión, los sujetos sexualmente activos deben aceptar utilizar un método anticonceptivo autorizado o evitar las relaciones sexuales
    E.4Principal exclusion criteria
    Any of the following would exclude the subject from participation in the study:
    1) Any condition that, in the opinion of the investigator, would interfere with evaluation of the investigational product or interpretation of subject safety or study results
    2) Concurrent enrollment in another clinical study, unless it is a non-interventional, observational study or the subject is in the follow-up period from a previous study, or prior sponsor approval is obtained
    3) Employees of the study site directly involved with the study at the site of enrollment, or immediate family members of such individuals
    4) Isolated testicular or CNS ALL
    5) Inadequate hepatic function
    6) Inadequate renal function
    7) Radiologically-detected CNS lymphoma
    8) Laboratory findings consistent with Grade >/= 3 disseminated intravascular coagulation (DIC) or any Grade 2 DIC that does not correct
    9) Hyperleukocytosis (WBC >/= 50,000/µL) or rapidly progressive disease that in the estimation of the investigator and sponsor would compromise ability to complete study therapy
    10) Pregnant or breast-feeding females
    11) Prior treatment with CAT-3888 (BL22), moxetumomab pasudotox, or any pseudomonas-exotoxin-containing compound
    12) Prior treatment with any anticancer biologic therapy within 2 weeks prior to enrollment, including but not limited to therapeutic monoclonal antibodies or antibody-drug conjugates
    13) Systemic chemotherapy </= 2 weeks (6 weeks for nitrosoureas) and radiation therapy </=3 weeks prior to starting study drug with exceptions per protocol
    14) Other investigational antineoplastic agents within 30 days prior to Cycle 1, Dose of study treatment
    15) Human immunodeficiency virus (HIV) positive serology
    16) Seropositivity for hepatitis B (HBsAg) or hepatitis C (HCV antibody)
    17) Clinically significant ophthalmologic findings during the screening
    18) Uncontrolled, symptomatic, intercurrent illness that would limit study compliance
    19) Presence of a second invasive malignancy
    20) Any physical, social, or psychiatric condition, or any other condition which in the opinion of the investigator would prevent effective study cooperation or participation
    21) Uncontrolled pulmonary infection, presence of pulmonary edema
    22) Inadequate oxygen saturation
    23) Serum albumin < 2 g/dL
    24) Radioimmunotherapy within 2 years prior to study enrollment
    25) Subject with prior history of thrombotic microangiopathy or HUS.
    El sujeto no debe presentar ninguno de los criterios siguientes para poder participar en el ensayo.
    -Cualquier enfermedad que a juicio del investigador dificulte la evaluación del producto en estudio o la interpretación de los resultados de seguridad o del ensayo.
    -Participación simultánea en otra investigación, a menos que sea de tipo observacional y no intervencionista, o bien que el sujeto se halle en una fase de seguimiento posterior al tratamiento o que se cuente con el visto bueno del promotor.
    -Ser trabajador del centro donde se realiza el ensayo clínico e intervenir directamente en su realización, o ser familiar directo de dichas personas.
    -LLA testicular o del SNC aislada.
    -Alteración de la función hepática:
    -Alteración de la function renal
    -Linfoma del SNC detectado mediante radiología.
    -Alteraciones analíticas compatibles con un cuadro de coagulación intravascular diseminada (CID) de grado >/= 3 o una CID de grado 2 que no sea corregible.
    -Hiperleucocitosis (>/= 50.000 leucocitos por microlitro) o progresión rápida de la enfermedad que a juicio del investigador y del promotor pongan en entredicho la capacidad de finalizar el tratamiento en estudio.
    -Embarazo o lactancia materna en el caso de las chicas.
    -Antecedentes de tratamiento con CAT-8888 (BL22), con moxetumomab pasudotox o con una molécula que contenga exotoxinas de pseudomonas.
    -Antecedentes de tratamiento antineoplásico con un producto biológico en las últimas 2 semanas, incluidos los anticuerpos monoclonales y los conjugados anticuerpo-fármaco.
    -Quimioterapia sistémica en las </= 2 semanas (6 semanas en el caso de las nitrosoureas) y radioterapia en las </=3 semanas antes de comenzar el tratamiento en estudio, con las excepciones incluidas en el protocolo
    -Otros antineoplásicos en fase de investigación en los 30 días anteriores a la 1.ª dosis del 1.er ciclo del fármaco en estudio,
    -Seropositividad del virus de la inmunodeficiencia humana (VIH).
    -Seropositividad de la hepatitis B (HBsAg) o la hepatitis C (anticuerpos del VHC)
    -Alteraciones oftalmológicas de relevancia clínica durante la selección.
    -Enfermedad concomitante sintomática e incontrolada que menoscabaría el cumplimiento de los requisitos del ensayo.
    -Presencia de una segunda neoplasia invasora;
    -Situación física, social, psiquiátrica o de otra índole que a juicio del investigador impida la colaboración efectiva o la participación en el ensayo.
    -Infección pulmonar no controlada; presencia de edema pulmonar.
    -Saturación de oxígeno en reposo inadecuada
    -Albúmina sérica < 2 g/dl.
    -Radioinmunoterapia en los 2 años antes de la inclusión.
    -Antecedentes de microangiopatía trombótica o síndrome urémico hemolítico
    E.5 End points
    E.5.1Primary end point(s)
    Assess the efficacy defined as composite complete response (CRc)
    Evaluar la eficacia definida como la respuesta completa combinada (RCc)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Disease assessments will be completed prior to the first cycle and subsequent cycles
    La evaluación de la enfermedad se llevará a cabo antes del primer ciclo y de los subsecuentes
    E.5.2Secondary end point(s)
    - Assess safety and tolerability
    - Assess immunogenicity and pharmacokinetics
    - PFS, OS, DOCR, DOR
    - Minimal residual disease negative CRc rate
    - Number eligible for stem cell transplant
    - ORR
    - Hematologic activity
    - Evaluar seguridad y tolerabilidad
    - Evaluar inmunogenicidad y farmacocinética
    - SSP, SG, DRC, DRG
    - Tasa de RCc sin enfermedad residual minima
    - Numero elegible para transplante de células hematopoyéticas
    - TRG
    - Actividad hematológica
    E.5.2.1Timepoint(s) of evaluation of this end point
    Duration of study
    Duración del estudio
    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 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. 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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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.8.3 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 EEA29
    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
    United Kingdom
    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
    12 months after last subject begins treatment or earlier if interim analysis performed after enrollment of the first 33 evaluable subjects shows CRc </= 21%
    12 meses después de que el último sujeto haya iniciado el tratamiento o antes si los análisis intermedios realizados tras el reclutamiento de los primeros 33 sujetos evaluables muestra una RCc </= 21%
    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 months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days9
    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: 76
    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. of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 35
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 35
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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. of subjects incapable of giving consent
    Pediatric subjects
    Pacientes pediátricos
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 33
    F.4.2.2In the whole clinical trial 76
    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)
    Standard of Care
    Cuidados habituales
    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 Innovative Therapies for Children with Cancer (ITCC)
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-07-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-11-03
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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