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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    2012-003101-10
    Trial protocol
    GB   DE   FR   IT   ES   NL  
    Global end of trial date
    02 Nov 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Aug 2016
    First version publication date
    14 Aug 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CD-ON-CAT-8015-1036
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02227108
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    MedImmune LLC
    Sponsor organisation address
    One MedImmune Way, Gaithersburg, United States, MD 20878
    Public contact
    Information Centre, AstraZeneca, Information.centre@astrazeneca.com, MedImmune LLC, +1 3013980000 x,
    Scientific contact
    Information Centre, AstraZeneca, Information.centre@astrazeneca.com, MedImmune LLC, +1 3013980000 x,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    21 Dec 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Nov 2015
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the efficacy of moxetumomab pasudotox as measured by the composite complete response (CRc) defined as achieving complete response (CR), or complete response with incomplete count recovery (CRi) in pediatric subjects with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma.
    Protection of trial subjects
    The conduct of this clinical study met all local legal and regulatory requirements. The study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and the International Conference on Harmonization guideline E6: Good Clinical Practice. Participating participant signed informed consent form and could withdraw from the study at any time without any disadvantage and without having to provide a reason for this decision. Only investigators qualified by training and experience were selected as appropriate experts to investigate the study drug.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    11 Aug 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 21
    Country: Number of subjects enrolled
    Australia: 3
    Country: Number of subjects enrolled
    France: 4
    Country: Number of subjects enrolled
    Italy: 2
    Worldwide total number of subjects
    32
    EEA total number of subjects
    8
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    22
    Adolescents (12-17 years)
    10
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    A total of 37 participants were screened, of which 5 did not meet eligibility criteria and were considered as screen failures; the remaining 32 participants entered into the study and 30 participants were treated with moxetumomab pasudotox.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Arm description
    Participants received 6 doses of moxetumomab pasudotox 40 mcg/kg intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.
    Arm type
    Experimental

    Investigational medicinal product name
    Moxetumomab Pasudotox
    Investigational medicinal product code
    CAT-8015
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received 6 doses of moxetumomab pasudotox 40 mcg/kg intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.

    Number of subjects in period 1
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Started
    32
    Treated
    30
    Completed
    1
    Not completed
    31
         Progressive disease
             17
         Investigator discretion
             1
         Did not receive treatment
             2
         Initiation of alternative therapy
             2
         Adverse event, non-fatal
             6
         Unspecified
             3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Reporting group description
    Participants received 6 doses of moxetumomab pasudotox 40 mcg/kg intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.

    Reporting group values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg) Total
    Number of subjects
    32 32
    Age categorical
    Units: Subjects
        Children (2-11 years)
    18 18
        Adolescents (12-17 years)
    14 14
    Age Continuous |
    Units: Years
        arithmetic mean (standard deviation)
    10.5 ± 3.9 -
    Gender, Male/Female
    Units: Participants
        Female
    13 13
        Male
    19 19

    End points

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    End points reporting groups
    Reporting group title
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Reporting group description
    Participants received 6 doses of moxetumomab pasudotox 40 mcg/kg intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.

    Primary: Percentage of Participants with Composite Complete Response (CRc)

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    End point title
    Percentage of Participants with Composite Complete Response (CRc) [1]
    End point description
    The CRc is defined as achieving complete response (CR), or CR with incomplete count recovery [CRi]) in participants with relapsed or refractory B-cell ALL or B-cell lymphoblastic lymphoma. The efficacy assessments were as per investigator assessment.
    End point type
    Primary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No inferential statistical analysis was planned for this end-point.
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28 [2]
    Units: percentage of participants
        number (confidence interval 95%)
    10.7 (2.3 to 28.2)
    Notes
    [2] - Evaluable efficacy population
    No statistical analyses for this end point

    Secondary: Overall Response Rate (ORR)

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    End point title
    Overall Response Rate (ORR)
    End point description
    The ORR, defined as the percentage of participants with CRc or partial response (PR), was estimated; the Clopper Pearson (Exact) 95% CI was calculated. The CRc is defined as complete response (CR), or complete response with incomplete count recovery (CRi). Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: percentage of participants
        number (confidence interval 95%)
    28.6 (13.2 to 48.7)
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Minimal Residual Disease (MRD)-negative CRc rate

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    End point title
    Percentage of Participants with Minimal Residual Disease (MRD)-negative CRc rate
    End point description
    The MRD-negative CRc rate was defined as the percentage of participants who achieved CRc and became MRD-negative as determined by flow cytometry performed by a central analysis laboratory. The CRc is defined as complete response (CR), or complete response with incomplete count recovery (CRi). Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: percentage of participants
    0
    No statistical analyses for this end point

    Secondary: Time to Overall Response

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    End point title
    Time to Overall Response
    End point description
    Time to overall response was evaluated using the Kaplan-Meier method. Duration of overall response was defined as the duration from the first documentation of overall response to the first documented disease progression. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: months
        median (confidence interval 95%)
    0.66 (0.53 to 0.76)
    No statistical analyses for this end point

    Secondary: Best Overall Response (BOR)

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    End point title
    Best Overall Response (BOR)
    End point description
    Best overall response was calculated, based upon disease assessments recorded during study visits, and summarized with percentage of participants for following categories: CRc,PR,HA,SD,PD, and not evaluable. Overall best response is best response observed for a participant during study based on International Working Group (IWG) Response Criteria for malignant lymphoma.Complete response (CR) is complete disappearance of all detectable clinical evidence of disease and disease-related symptoms.PR is a minimum of 50% decrease in sum of product of diameters of up to 6 of largest dominant nodes or nodal masses and no increase in size of other nodes and in size of liver or spleen.Stable disease (SD) is when a participant fails to attain criteria needed for a CR or PR, but does not fulfill those for PD.Efficacy evaluable population included all participants who received any amount of study drug and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: percentage of participants
    number (not applicable)
        Composite complete response
    10.7
        Partial response
    17.9
        Hematological activity
    7.1
        Stable disease
    21.4
        Progressive disease
    39.3
        No evaluation available
    3.6
    No statistical analyses for this end point

    Secondary: Bone Marrow Blast Percentage Change

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    End point title
    Bone Marrow Blast Percentage Change
    End point description
    Change in bone marrow blast percentage from baseline was evaluated. The intent to treat (ITT) population included all participants who entered the study. Here, number of participants analysed, "N" included evaluable participants for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    26
    Units: percentage of participants
    number (not applicable)
        Baseline M1, post-baseline M1
    0
        Baseline M1, post-baseline M2
    0
        Baseline M1, post-baseline M3
    3.8
        Baseline M2, post-baseline M1
    0
        Baseline M2, post-baseline M2
    0
        Baseline M2, post-baseline M3
    7.7
        Baseline M3 post-baseline M1
    3.8
        Baseline M3 post-baseline M2
    11.5
        Baseline M3 post-baseline M3
    73.1
    No statistical analyses for this end point

    Secondary: Percentage of Participants who Became Eligible to Receive an Stem Cell Transplant (SCT) After Treatment with Moxetumomab Pasudotox as Well as the Time to Transplant

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    End point title
    Percentage of Participants who Became Eligible to Receive an Stem Cell Transplant (SCT) After Treatment with Moxetumomab Pasudotox as Well as the Time to Transplant
    End point description
    The percentage of participants who became eligible for SCT after treatment with moxetumomab pasudotox were provided. The time to SCT was defined as the duration from the start of treatment with moxetumomab pasudotox until the date when the participant became eligible for SCT. The Clopper Pearson (Exact) 95% CI was calculated. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: percentage of participants
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants who Were Neutropenic at Study Entry and who Experienced Hematologic Activity (HA)

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    End point title
    Percentage of Participants who Were Neutropenic at Study Entry and who Experienced Hematologic Activity (HA)
    End point description
    The percentage of participants who were neutropenic at study entry and experienced HA after treatment with moxetumomab pasudotox was evaluated. The Clopper Pearson (Exact) 95% CI was calculated. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: percentage of participants
        number (not applicable)
    3.6
    No statistical analyses for this end point

    Secondary: Duration of Complete Response (DOCR)

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    End point title
    Duration of Complete Response (DOCR)
    End point description
    DOCR was defined as the duration from the first documentation of CRc to the first documented disease progression.The CRc is defined as achieving complete response (CR), or CR with incomplete count recovery [CRi]) in participants with relapsed or refractory B-cell ALL or B-cell lymphoblastic lymphoma. Kaplan-Meier method was used for evaluation. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment. Here, number of participants analysed, "N" included evaluable participants for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    1
    Units: months
        number (not applicable)
    1.97
    No statistical analyses for this end point

    Secondary: Duration of Overall Response (DOR)

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    End point title
    Duration of Overall Response (DOR)
    End point description
    DOR was defined as the duration from the first documentation of overall response to the first documented disease progression. ''99999'' indicates data was not available as upper limit of 95% confidence interval could not be determined. Kaplan-Meier method was used for evaluation. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: months
        median (confidence interval 95%)
    0.95 (0.72 to 99999)
    No statistical analyses for this end point

    Secondary: Progression-Free Survival (PFS)

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    End point title
    Progression-Free Survival (PFS)
    End point description
    PFS was measured from the start of treatment with moxetumomab pasudotox until the first documentation of disease progression or death due to any cause, whichever occurred first. Kaplan-Meier method was used for evaluation. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: months
        median (confidence interval 95%)
    1.4 (0.7 to 1.5)
    No statistical analyses for this end point

    Secondary: Overall Survival (OS)

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    End point title
    Overall Survival (OS)
    End point description
    OS was determined as the time from the start of treatment with moxetumomab pasudotox until death due to any cause. For participants who were alive at the end of the study or lost to follow-up, OS was censored on the last date when the participant was known be alive. ''99999'' indicates data was not available as upper limit of 95% confidence interval could not be determined. Kaplan-Meier method was used for evaluation. Efficacy evaluable population included all participants who received any amount of moxetumomab pasudotox and completed a baseline disease assessment and had at least one post-baseline disease assessment.
    End point type
    Secondary
    End point timeframe
    Baseline to end of study or last contact date
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    28
    Units: months
        median (confidence interval 95%)
    3.6 (2.4 to 99999)
    No statistical analyses for this end point

    Secondary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

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    End point title
    Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
    End point description
    Treatment-emergent adverse events (TEAEs), were defined as events present at baseline that worsened in intensity after administration of investigational product or events absent at baseline that emerged after administration of study drug. Safety population includes all participants who received any amount of moxetumomab pasudotox.
    End point type
    Secondary
    End point timeframe
    Baseline up to 30 days after the last dose of study drug
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    30
    Units: participants
        TEAEs
    30
        TESAEs
    16
    No statistical analyses for this end point

    Secondary: Number of Participants With Abnormal Clinical Laboratory Parameters Reported as Treatment-Emergent Adverse Events (TEAE)

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    End point title
    Number of Participants With Abnormal Clinical Laboratory Parameters Reported as Treatment-Emergent Adverse Events (TEAE)
    End point description
    Laboratory tests were grouped according to hematology, serum chemistry, and urinalysis. Laboratory abnormalities with toxicity grades according to NCI CTCAE Version 4.03 were derived according to laboratory values and reported as treatment-emergent adverse events. Safety population includes all participants who received any amount of moxetumomab pasudotox.
    End point type
    Secondary
    End point timeframe
    Baseline up to 30 days after the last dose of study drug
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    30
    Units: participants
        Anaemia
    11
        Febrile neutropenia
    7
        Haemolytic uraemic syndrome
    4
        Thrombocytopenia
    1
        Platelet count decreased
    9
        Neutrophil count decreased
    6
        White blood cell count decreased
    5
        Lymphocyte count decreased
    2
        International normalised ratio increased
    1
        Alanine aminotransferase increased
    11
        Aspartate aminotransferase increased
    8
        Gamma-glutamyltransferase increased
    4
        Blood bilirubin increased
    2
        Blood creatinine increased
    2
        Blood lactate dehydrogenase increased
    2
        Bilirubin conjugated
    1
        Blood albumin decreased
    1
        Blood urea increased
    1
        Glucose tolerance increased
    1
        Hypoalbuminaemia
    6
        Hypocalcaemia
    6
        Hypokalaemia
    5
        Hyponatraemia
    5
        Hyperglycaemia
    4
        Hypophosphataemia
    3
        Hypermagnesaemia
    1
        Hypomagnesaemia
    1
        Hepatic function abnormal
    1
        Proteinuria
    1
    No statistical analyses for this end point

    Secondary: Number of participants with clinically significant Electrocardiogram (ECG) abnormalities

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    End point title
    Number of participants with clinically significant Electrocardiogram (ECG) abnormalities
    End point description
    Participants were evaluated for ECG abnormalities. No clinically significant ECG abnormalities were detected and clinically significant prolongation of the median QT interval was not observed. Safety population includes all participants who received any amount of moxetumomab pasudotox.
    End point type
    Secondary
    End point timeframe
    Baseline up to 30 days after the last dose of study drug
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    30
    Units: participants
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)
    End point description
    Participants who experienced vital signs abnormalities recorded as TEAEs were reported. Safety population includes all participants who received any amount of moxetumomab pasudotox.
    End point type
    Secondary
    End point timeframe
    Baseline up to 30 days after the last dose of study drug
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    30
    Units: participants
        Pyrexia
    16
        Dyspnoea
    6
        Hypoxia
    4
        Hypertension
    8
        Hypotension
    4
        Weight decreased
    1
        Weight increased
    10
        Bradycardia
    7
        Sinus Bradycardia
    1
        Sinus Tachycardia
    3
        Tachycardia
    3
    No statistical analyses for this end point

    Secondary: Number of Participants With Positive Anti-drug Antibody (ADA) and Neutralizing Antibodies (NAb)

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    End point title
    Number of Participants With Positive Anti-drug Antibody (ADA) and Neutralizing Antibodies (NAb)
    End point description
    Immunogenicity assessment included determination of anti-drug (moxetumomab pasudotox) antibodies and neutralizing antidrug antibodies in serum samples. Titers and specificity were determined for NAb-positive participants. Specificity were observed in participants who had ADAs directed to the PE38 domain of moxetumomab pasudotox and increase in titers were observed in participants who tested ADA-positive at baseline. Safety population includes all participants who received any amount of moxetumomab pasudotox. Here, number of participants analysed, "N" included participants with at least one post-baseline sample.
    End point type
    Secondary
    End point timeframe
    Prior to the Start of Each Cycle for Cycles 1, 2, 3, and Subsequent Odd-Numbered Cycles, End of Treatment, and 30 Day Follow-up Visit
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    23
    Units: participants
        Anti-drug Antibody (ADA)
    13
        Neutralizing Antibodies (NAb)
    13
        Increase in Titers
    3
        Specificity
    13
    No statistical analyses for this end point

    Secondary: Area Under the Plasma Concentration Time Curve From Time 0 to Infinity (AUC0-inf) After the First Dose of Cycle 1

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    End point title
    Area Under the Plasma Concentration Time Curve From Time 0 to Infinity (AUC0-inf) After the First Dose of Cycle 1
    End point description
    AUC (0-infinity) = Area under the serum concentration versus time curve from time zero (pre-dose) to extrapolated infinite time (0-infinity). It is obtained from AUC (0-t) plus AUC (tinfinity). It was calculated by extrapolating the concentrationtime curve from time zero to infinity using the linear/log trapezoidal rule. Safety population who provided at least one measurable Pharmacokinetic concentration. Here, number of participants analysed, "N" included evaluable participants for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion of Day 1 of Cycle 1
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    7
    Units: hour*nanogram per milliliter (hr*ng/mL)
        arithmetic mean (standard deviation)
    1200 ± 224
    No statistical analyses for this end point

    Secondary: Area Under the Concentration Versus Time Curve From Time Zero to Last Quantifiable Concentration [AUC0-last] After the First Dose of Cycle 1

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    End point title
    Area Under the Concentration Versus Time Curve From Time Zero to Last Quantifiable Concentration [AUC0-last] After the First Dose of Cycle 1
    End point description
    AUC is a measure of systemic drug exposure, which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample. AUC0-last is defined as AUC from time zero to the last data point above the lower limit of quantification. Safety population who provided at least one measurable Pharmacokinetic concentration. Here, number of participants analysed, "N" included evaluable participants for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    21
    Units: hour*nanogram per milliliter (hr*ng/mL)
        arithmetic mean (standard deviation)
    794 ± 239
    No statistical analyses for this end point

    Secondary: Maximum Observed Drug Concentration in Plasma (Cmax) After the First Dose of Cycle 1

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    End point title
    Maximum Observed Drug Concentration in Plasma (Cmax) After the First Dose of Cycle 1
    End point description
    Cmax refers to the highest measured drug concentration which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample. Safety population who provided at least one measurable Pharmacokinetic concentration.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    26
    Units: nanogram per milliliter
        arithmetic mean (standard deviation)
    457 ± 128
    No statistical analyses for this end point

    Secondary: Time to Reach Maximum Drug Concentration in Plasma (tmax) After the First Dose of Cycle 1

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    End point title
    Time to Reach Maximum Drug Concentration in Plasma (tmax) After the First Dose of Cycle 1
    End point description
    Tmax refers to the time after dosing when a drug attains its highest measurable concentration (Cmax). It is obtained by collecting a series of blood samples at various times after dosing, and measuring them for drug content. Safety population who provided at least one measurable Pharmacokinetic concentration.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    26
    Units: hour
        arithmetic mean (standard deviation)
    0.54 ± 0.05
    No statistical analyses for this end point

    Secondary: Terminal Phase Elimination Half Life (t1/2) After the First Dose of Cycle 1

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    End point title
    Terminal Phase Elimination Half Life (t1/2) After the First Dose of Cycle 1
    End point description
    Terminal phase elimination half-life is the time measured for the serum/plasma concentration to decrease by one half, calculated as natural logarithmic (log)-transformed (ln) value of 2 divided by elimination rate constant (lambda); that is [ln(2)/lambda]. Elimination rate constant (lambda) was estimated via linear regression of the time versus log concentration. Safety population who provided at least one measurable Pharmacokinetic concentration. Here, number of participants analysed, "N" included evaluable participants for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    7
    Units: hour
        median (full range (min-max))
    1.02 (0.82 to 1.44)
    No statistical analyses for this end point

    Secondary: Systemic Clearance (CL) After the First Dose of Cycle 1

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    End point title
    Systemic Clearance (CL) After the First Dose of Cycle 1
    End point description
    CL is a quantitative measure of the rate at which a drug substance is removed from the body. The total systemic clearance after intravenous dose was estimated by dividing the total administered dose by the plasma Area Under the Plasma Concentration-Time Curve From Time Zero to Infinite Time (AUC[0-infinity]). Safety population who provided at least one measurable Pharmacokinetic concentration. Here, number of participants analysed, "N" included evaluable participants for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
    End point values
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    7
    Units: milliliter per hour per kilogram
        arithmetic mean (full range (min-max))
    34.4 (24.4 to 41.8)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    The period immediately following the time that written informed consent is obtained through Day 30 after the last dose
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Reporting group description
    Participants received 6 doses of moxetumomab pasudotox 40 mcg/kg intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.

    Serious adverse events
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    16 / 30 (53.33%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    1
    Vascular disorders
    Capillary leak syndrome
         subjects affected / exposed
    5 / 30 (16.67%)
         occurrences causally related to treatment / all
    5 / 5
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary oedema
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    1 / 1
    Respiratory distress
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Haemolytic uraemic syndrome
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences causally related to treatment / all
    4 / 4
         deaths causally related to treatment / all
    0 / 0
    Febrile neutropenia
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Encephalopathy
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Headache
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Somnolence
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Vision blurred
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pyrexia
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences causally related to treatment / all
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Bone pain
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Device related infection
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infection
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Staphylococcal sepsis
         subjects affected / exposed
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Moxetumomab Pasudotox 40 microgram per kilogram (mcg/kg)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    30 / 30 (100.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    8 / 30 (26.67%)
         occurrences all number
    12
    Hypotension
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    4
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    3
    Face oedema
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    5
    Oedema peripheral
         subjects affected / exposed
    5 / 30 (16.67%)
         occurrences all number
    8
    Pyrexia
         subjects affected / exposed
    12 / 30 (40.00%)
         occurrences all number
    40
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    3
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    11 / 30 (36.67%)
         occurrences all number
    45
    Aspartate aminotransferase increased
         subjects affected / exposed
    8 / 30 (26.67%)
         occurrences all number
    34
    Blood bilirubin increased
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    4
    Blood creatinine increased
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    3
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Lymphocyte count decreased
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    4
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    18
    Platelet count decreased
         subjects affected / exposed
    8 / 30 (26.67%)
         occurrences all number
    59
    Neutrophil count decreased
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    21
    White blood cell count decreased
         subjects affected / exposed
    5 / 30 (16.67%)
         occurrences all number
    24
    Weight increased
         subjects affected / exposed
    10 / 30 (33.33%)
         occurrences all number
    21
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    3
    Sinus tachycardia
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    6
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    13
    Anaemia
         subjects affected / exposed
    11 / 30 (36.67%)
         occurrences all number
    29
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    9
    Cough
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    3
    Hypoxia
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    6
    Epistaxis
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Wheezing
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    12 / 30 (40.00%)
         occurrences all number
    22
    Paraesthesia
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Eye disorders
    Periorbital oedema
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    3
    Vision blurred
         subjects affected / exposed
    5 / 30 (16.67%)
         occurrences all number
    6
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    5
    Constipation
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    5
    Diarrhoea
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    3
    Vomiting
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    4
    Nausea
         subjects affected / exposed
    8 / 30 (26.67%)
         occurrences all number
    10
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    6
    Bone pain
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    3
    Groin pain
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Myalgia
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Musculoskeletal chest pain
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Pain in extremity
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    6
    Neck pain
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Pain in jaw
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2
    Metabolism and nutrition disorders
    Hypoalbuminaemia
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    11
    Hyperglycaemia
         subjects affected / exposed
    4 / 30 (13.33%)
         occurrences all number
    12
    Hypocalcaemia
         subjects affected / exposed
    6 / 30 (20.00%)
         occurrences all number
    28
    Hypokalaemia
         subjects affected / exposed
    5 / 30 (16.67%)
         occurrences all number
    27
    Hyponatraemia
         subjects affected / exposed
    5 / 30 (16.67%)
         occurrences all number
    12
    Hypophosphataemia
         subjects affected / exposed
    3 / 30 (10.00%)
         occurrences all number
    13
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    2 / 30 (6.67%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Feb 2013
    Enrollment of any relapsed participant with acute lymphoblastic leukemia (ALL) was allowed. B-cell lineage disease (that is, B-cell ALL and B-cell lymphoblastic lymphoma) were also targeted. -The secondary objective of “to evaluate the participant’s quality of life using the parent proxy-reported health-related quality of life (HRQOL) assessment, specifically using PedsQL4.0 Generic Core Scale, the PedsQL 3.0 Cancer Module, and the PedsQL Multidimensional Fatigue Scales” was deleted. - Dose changed from 50 to 40 mcg/kg due to a change from Process 2 to Process 3 and from 6 to 10 doses per cycle. - Study-stopping criteria modified. - Clarified that cluster of differentiation-22 (CD22) positivity was not required for inclusion. – Inclusion and exclusion criteria were revised. - Withdrawal criteria were added and revised. - This is in reference to the amendment change for clarifying that other agents could have been used in addition to or in lieu of allopurinol. - Allopurinol was to be administered during Cycle 1. - Clarified dosing delay within a cycle of > 5 Days would result in withdrawal from the study. - Clarified additional concomitants being allowed. – Clarified DMC change. – Clarified that investigators should consider G6PD or other screening tests for conditions that may cause hemolysis. - Dosing was changed to not be capped at a certain weight. – Treatment administration details were clarified. - Conditions for treatment continuation were revised. - Clarification of study procedures timings were done. - Statistical definitions and clarifications were included.
    19 Apr 2013
    -Added Exclusion criteria, to exclude participants with mixed-lineage leukemia (MLL) gene rearrangement. - Added Exclusion criteria, to exclude participants with a QTcF interval above a defined threshold. - Added text that participants will also be queried for ocular symptoms throughout the study; and that additional ophthalmologic exams must be conducted to follow up on any reported ocular symptoms. - Clarified that bone marrow aspirate with or without a biopsy would be performed. - Updated schedule of assessments on 24-hour urine protein excretion and creatinine clearance, and electrocardiograms (ECGs). - Management guidelines for hemolytic uremic syndrome (HUS) added.
    13 Jan 2014
    - CAT-8015” was replaced with “moxetumomab pasudotox” where appropriate. – Change of definitions of primary and secondary objectives, assessment of endpoints, and power calculations. - Change from dosing with 40 mcg/kg of Process 2 material to its bioequivalent of 32 mcg/kg of Process 3 material. - Wording was changed “must be reported within 24 hours of the Principal Investigator or designee obtaining knowledge of event. - Interim monitoring of treatment-related death was added to study-stopping criteria. – Exclusion criteria were modified. - Withdrawal criteria was amended. - Dosing delays were included for events of hypercalcemia. - Exclusion of concomitant medication of high-dose estrogen therapy was added. - Added: “If participants develop any grade of HUS or capillary leak syndrome (CLS) during study treatment, PK and immunogenicity samples, serum complement factors, and serum immunoglobulin levels will be collected within 24 hours of time of first diagnosis, worsening of event, and at time of improvement and full resolution of event.” - Added: Appendix 4: Performance Status Scales/Scores (Eastern Cooperative Oncology Group (ECOG)/Karnofsky/Lansky), Appendix 5: Schwartz Formula, and Appendix 6: Measured Corrected Creatinine Clearance. - Treatment administration: Wording for option to pre-medicate participants with acetaminophen, diphenhydramine, and ranitidine at Principal Investigator’s discretion and as clinically indicated was added to this section. - Wording was changed regarding administration of allopurinol, corticosteroid, and fluid hydration. - Added: HLA typing and anti-Factor H antibodies, serum immunoglobulins, serum complement factors, twice daily serum creatinine measurements on specified days during Cycle 1. - Twice daily serum creatinine collections and assessments for ocular symptoms were added for inpatient admission days during Cycle 1. - A change was made to make any grade of CLS or thrombotic microangiopathy/HUS immediately reportable.
    17 Jun 2014
    - Dosing was changed from 32 mcg/kg of Process 3 material for 10 doses in each 21-day cycle to 40 mcg/kg of Process 3 material for 6 doses (Days 1, 3, 5, 7, 9, and 11) of each 21-day cycle until completion of a maximum of 6 cycles. - The statement regarding retinal damage or abnormalities was simplified to retinal abnormalities. - The start time for allopurinol was specified as at least 8 hours prior to the first dose of moxetumomab pasudotox. - The dose of methylprednisolone for treatment of infusion reaction was changed from a fixed dose to a weight based dose. - The number of days for a delay in the start of a cycle to accommodate schedule conflicts was reduced from 14 days to 7 days. - In the event that a subject developed any grade of HUS or CLS, or had an ocular event with new abnormality by ophthalmologic examination during treatment, collection of samples for plasma cytokines was added in addition to PK and immunogenicity samples, serum complement factors, and serum immunoglobulin levels. - The requirement for collection of samples for CD22 expression if subjects develop HUS, CLS, or ocular events during study treatment was removed as CD22 expression was to be collected at screening only. - The threshold for withdrawal due to an event of thrombotic microangiopathy/HUS was lowered from >= Grade 3 to >= Grade 2; The duration for concomitant allopurinol during Cycle 1 was changed from “until disease burden is reduced” to “until at least 24 hours after the sixth dose of moxetumomab pasudotox in Cycle 1”. - A provision requiring admission for inpatient management for participants with Grade 2 or higher HUS.
    06 Mar 2015
    -Text was modified to specify that treatment would not continue if the start of a cycle was delayed more than 7 days (that is ie, beyond 28 days after the first dose of the previous cycle). - Text was updated to include HUS as an identified risk, and information regarding mitigation and monitoring for this risk was added. - Inclusion criteria 4 was clarified to specify that all participants (both ALL and subjects with lymphoblastic lymphoma) must have M2 or M3 bone marrow classification to be eligible for the study. - Modified exclusion criteria 2 to indicate that the participant must not be concurrently enrolled in another clinical study for cancer treatment, unless the subject is in the follow-up period of the previous study. - Added vincristine as an exception to the medication exclusions in exclusion criterion 15. - Deleted “within 3 months prior to enrollment” from exclusion criterion 26. - Myoglobin was deleted from the list of cardiac biomarkers. - Schistocyte count was included as part of the peripheral blood smear at selected time points. - Added the following to exclusion criterion 24: Albumin infusions for correction of hypoalbuminemia are allowed, but cannot have been administered within 7 days prior to start of study drug. - Modified exclusion criterion 30 to indicate that participants who received high-dose estrogen therapy within 2 weeks prior to enrollment in the study were excluded. Use of high-dose estrogen therapy was added as a withdrawal criterion. - Added “or HUS-like event” to withdrawal criterion 10.
    28 Apr 2015
    -Clarification of language regarding the severity of CLS.

    Interruptions (globally)

    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    01 Sep 2015
    The study was terminated prior to a planned interim analysis based on lack of required efficacy in the first 32 participants enrolled.
    -

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    The study was terminated due to lack of required efficacy because review of response data suggested that the number of clinical responses required to proceed to Stage 2 would not be met.
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