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    The EU Clinical Trials Register currently displays   36358   clinical trials with a EudraCT protocol, of which   5990   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
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    EudraCT Number:2012-003101-10
    Sponsor's Protocol Code Number:CD-ON-CAT-8015-1036
    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:2013-04-24
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    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
    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
    A.4.1Sponsor's protocol code numberCD-ON-CAT-8015-1036
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1150
    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 typerange
    D.3.10.3Concentration number0.90 to 1.10
    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
    Pediatric Subjects with Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) of B-cell Origin
    E.1.1.1Medical condition in easily understood language
    Pediatric Acute Lymphoblastic Leukemia
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10000845
    E.1.2Term Acute lymphoblastic leukemia
    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)
    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
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    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 with marrow involvement
    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) Adequate Performance status.
    7) Subjects with the following 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.
    8) Female subjects of childbearing potential and post-pubertal male
    subjects must agree to use an approved method of contraception for the study (if sexually active) or abstinence to meet eligibility criteria.
    E.4Principal exclusion criteria
    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 conduct of the study at the site of proposed enrollment, or immediate family members of any such individuals
    4) Isolated testicular or CNS ALL
    5) Subjects with mixed-lineage leukemia (MLL) gene rearrangement
    6) Inadequate hepatic function
    7) Inadequate renal function
    8) Radiologically-detected CNS lymphoma
    9) Laboratory findings or clinical evidence consistent with Grade ≥ 3 disseminated intravascular coagulation (DIC) or any Grade 2 DIC that does not correct
    10) 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
    11) Pregnant or breast-feeding females
    12) A QTcF interval (manually overread) of ≥ 481 msec (ie, ≥ Grade 2) confirmed by medically qualified staff on at least 2 separate electrocardiograms (ECGs) performed at least 5 min apart within 28 days prior to enrollment
    13) Prior treatment with CAT-3888 (BL22), moxetumomab pasudotox, or any pseudomonas-exotoxin-containing compound
    14) 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
    15) Systemic chemotherapy ≤ 2 weeks (6 weeks for nitrosoureas) and radiation therapy ≤ 3 weeks prior to starting study drug with exceptions per protocol
    16) Other investigational antineoplastic agents within 30 days prior to Cycle 1, Dose of study treatment
    17) Seropositivity for Human immunodeficiency virus (HIV)
    18) Seropositivity for hepatitis B (HBsAg) or hepatitis C (HCV antibody)
    19) Clinically significant ophthalmologic findings (evidence of retinal damage or injury) during the screening
    20) Uncontrolled, symptomatic, intercurrent illness including, but not
    limited to infection, congestive heart failure, cardiac arrhythmia, malaria
    infection or any other condition that would limit compliance with study requirements
    21) Presence of a second invasive malignancy
    22) Any physical, social, or psychiatric condition, or any other condition which in the opinion of the principal investigator or designee would prevent effective study cooperation or participation in the study
    23) Uncontrolled pulmonary infection, presence of pulmonary edema
    24) Inadequate oxygen saturation
    25) Serum albumin < 2 g/dL
    26) Radioimmunotherapy within 2 years prior to enrollment in study
    27) Subject with prior history of thrombotic microangiopathy or HUS.
    28) Previous life-threatening anaphylactic reactions to prior monoclonal antibody-based immunotherapy or any component of the moxetumomab pasudotox formulation
    29) T-cell ALL or T-cell lymphoblastic lymphoma
    30) History of known congenital hypercoaguable condition
    31) Subjects receiving high-dose estrogen therapy
    32) For German Investigative Sites: Subjects with B-cell lymphoblastic lymphoma will be excluded.
    E.5 End points
    E.5.1Primary end point(s)
    Assess the efficacy defined as composite complete response (CRc)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Disease assessments will be completed prior to the first cycle and subsequent cycles
    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
    E.5.2.1Timepoint(s) of evaluation of this end point
    Duration of study
    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 concerned6
    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%
    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 months9
    E.8.9.1In the Member State concerned days9
    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
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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
    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 Decision2013-11-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-08-01
    P. End of Trial
    P.End of Trial StatusCompleted
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