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    Clinical Trial Results:
    A Phase 1/2 Study of CAT-8015 in Adult Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia

    Summary
    EudraCT number
    		 2010-020820-23
    Trial protocol
    		 DE  
    Global end of trial date
    04 Mar 2013

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Jan 2017
    First version publication date
    06 Jan 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    MI-CP218
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01030536
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    MedImmune LLC
    Sponsor organisation address
    Milstein Building, Granta Park, Cambridge, United Kingdom, CB21 6GH
    Public contact
    Mohammed Dar, MD, MedImmune LLC, 011 301 398 0000,
    Scientific contact
    Mohammed Dar, MD, MedImmune LLC, 011 301 398 0000,
    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
    04 Mar 2013
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Mar 2013
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Mar 2013
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objectives of this study were to determine the maximum tolerated dose (MTD) or optimal biologic dose (OBD), and to determine the safety profile of moxetumomab pasudotox in participants with relapsed or refractory advanced B-cell non-Hodgkin lymphoma (NHL) (diffuse large B-cell lymphoma [DLBCL], follicular lymphoma [FL], mantle cell lymphoma [MCL]) or chronic lymphocytic leukemia (CLL), including small lymphocytic lymphoma (SLL).
    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
    01 Mar 2010
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 23
    Worldwide total number of subjects
    23
    EEA total number of subjects
    0
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    12
    From 65 to 84 years
    11
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Study was started on 01 March 2010 and was terminated on 12 September 2012. The Product Development Team authorized early termination of this study due to prioritization of resources and the need to prioritize allocation of investigational product across the studies in the moxetumomab pasudotox clinical development program.

    Pre-assignment
    Screening details
    		 A total of 23 participants were enrolled and treated at 6 sites in the United States.

    Period 1
    Period 1 title
    Subject status at the end of study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 CAT-8015 20 microgram per kilogram (mcg/kg)
    Arm description
    		 Participants received 20 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Moxetumomab pasudotox
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    20 mcg/kg

    Arm title
    		 CAT-8015 30 microgram per kilogram (mcg/kg)
    Arm description
    		 Participants received 30 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Moxetumomab pasudotox
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    30 mcg/kg

    Arm title
    		 CAT-8015 40 microgram per kilogram (mcg/kg)
    Arm description
    		 Participants received 40 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Moxetumomab pasudotox
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    40 mcg/kg

    Arm title
    		 CAT-8015 50 microgram per kilogram (mcg/kg)
    Arm description
    		 Participants received 50 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Moxetumomab pasudotox
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    50 mcg/kg

    Arm title
    		 CAT-8015 60 microgram per kilogram (mcg/kg)
    Arm description
    		 Participants received 60 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Moxetumomab pasudotox
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    60 mcg/kg

    Number of subjects in period 1
    		CAT-8015 20 microgram per kilogram (mcg/kg) CAT-8015 30 microgram per kilogram (mcg/kg) CAT-8015 40 microgram per kilogram (mcg/kg) CAT-8015 50 microgram per kilogram (mcg/kg) CAT-8015 60 microgram per kilogram (mcg/kg)
    Started
    7
    6
    6
    3
    1
    Completed
    0
    0
    0
    0
    0
    Not completed
    7
    6
    6
    3
    1
         Adverse event, serious fatal
    -
    1
    -
    -
    -
         Consent withdrawn by subject
    -
    -
    1
    -
    -
         Adverse event, non-fatal
    -
    3
    1
    -
    1
         undefined
    1
    -
    1
    1
    -
         Progressive disease
    6
    2
    3
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    CAT-8015 20 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 20 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 30 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 30 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 40 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 40 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 50 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 50 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 60 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 60 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group values
    		 CAT-8015 20 microgram per kilogram (mcg/kg) CAT-8015 30 microgram per kilogram (mcg/kg) CAT-8015 40 microgram per kilogram (mcg/kg) CAT-8015 50 microgram per kilogram (mcg/kg) CAT-8015 60 microgram per kilogram (mcg/kg) Total
    Number of subjects
    		 7 6 6 3 1 23
    Age categorical
    Units: Subjects
    Age Continuous | Age
    Units: years
        arithmetic mean (standard deviation)
    		 63.4 ± 8.7 53.7 ± 16.9 63.5 ± 10 52.3 ± 14.6 66 ± 0 -
    Gender, Male/Female
    Units: participants
        Female
    		 2 1 0 0 1 4
        Male
    		 5 5 6 3 0 19
    Subject analysis sets

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 20 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 20 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 30 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 30 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 40 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 40 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 50 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 50 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 60 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    CLL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants with Chronic Lymphocytic Leukemia (CLL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    DLBCL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants with Diffuse Large B cell Lymphoma (DLBCL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    MCL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants with Mantle Cell Lymphoma (MCL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    FL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants wiht Follicular Lymphoma (FL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    CLL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants with Chronic Lymphocytic Leukemia were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis set title
    DLBCL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants with Diffuse Large B cell Lymphoma were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis set title
    MCL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants with Mantle Cell Lymphoma were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis set title
    FL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants wiht Follicular Lymphoma (FL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis sets values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg CLL (Safety Population) DLBCL (Safety Population) MCL (Safety Population) FL (Safety Population) CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects
    7
    6
    6
    3
    1
    10
    7
    2
    4
    10
    6
    1
    3
    Age categorical
    Units: Subjects
    Age Continuous | Age
    Units: years
        arithmetic mean (standard deviation)
    63.4 ± 8.7
    53.7 ± 16.9
    63.5 ± 10
    52.3 ± 14.6
    66 ± 0
    ±
    ±
    ±
    ±
    ±
    ±
    ±
    ±
    Gender, Male/Female
    Units: participants
        Female
    2
    1
    0
    0
    1
        Male
    5
    5
    6
    3
    0

    End points

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    End points reporting groups
    Reporting group title
    CAT-8015 20 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 20 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 30 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 30 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 40 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 40 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 50 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 50 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 60 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 60 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 20 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 20 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 30 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 30 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 40 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 40 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 50 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 50 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants received 60 mcg/kg Moxetumomab pasudotox as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Subject analysis set title
    CLL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants with Chronic Lymphocytic Leukemia (CLL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    DLBCL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants with Diffuse Large B cell Lymphoma (DLBCL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    MCL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants with Mantle Cell Lymphoma (MCL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    FL (Safety Population)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants wiht Follicular Lymphoma (FL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The safety population included all subjects who received Moxetumomab pasudotox. The safety population was used to evaluate baseline characteristics as well as all endpoints for safety.

    Subject analysis set title
    CLL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants with Chronic Lymphocytic Leukemia were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis set title
    DLBCL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants with Diffuse Large B cell Lymphoma were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis set title
    MCL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants with Mantle Cell Lymphoma were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Subject analysis set title
    FL (Evaluable for Efficacy)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Participants wiht Follicular Lymphoma (FL) were included. Moxetumomab pasudotox was administered at doses of 20, 30, 40, 50, or 60 mcg/kg on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60mcg/kg. The evaluable population for efficacy included all subjects who received any treatment of moxetumomab pasudotox and completed at least one post-baseline disease assessment. The evaluable population for efficacy was used to evaluate the endpoints for the efficacy profile.

    Primary: Maximum Tolerated Dose (MTD)

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    End point title
    		 Maximum Tolerated Dose (MTD) [1]
    End point description
    MTD reflects highest dose of drug that did not cause an unacceptable side effect (= Dose Limiting Toxicity (DLT) in more than 30% of patients.
    End point type
    Primary
    End point timeframe
    Days 1, 3, 5 every 28 days
    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: statistical analysis not provided as outcome measures reported as counts.
    End point values
    		 CAT-8015 20 microgram per kilogram (mcg/kg) CAT-8015 30 microgram per kilogram (mcg/kg) CAT-8015 40 microgram per kilogram (mcg/kg) CAT-8015 50 microgram per kilogram (mcg/kg) CAT-8015 60 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Number of Participants With Dose Limiting Toxicities (DLTs)

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    End point title
    		 Number of Participants With Dose Limiting Toxicities (DLTs) [2]
    End point description
    Any Grade 3 or greater, non-hematological toxicity (including capillary leak syndrome [CLS] and thrombotic microangiopathy/ hemolytic uremic syndrome (HUS), Grade 3 or higher treatment-related hematologic toxicities and only ≥ Grade 3 thrombotic microangiopathy /HUS constituted a DLT with few exceptions
    End point type
    Primary
    End point timeframe
    Days 1, 3, 5 every 28 days
    Notes
    [2] - 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: statistical analysis not provided as outcome measures reported as counts.
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
    0
    2
    1
    0
    1
    No statistical analyses for this end point

    Primary: 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) [3]
    End point description
    Treatment-emergent adverse event (TEAEs) were defined as events that occur following the first injection of study treatment, or that started prior to the first injection and worsened during treatment. An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An Serious Adverse Event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent adverse events were defined as adverse events/serious adverse events that started or worsened after the study drug treatment.
    End point type
    Primary
    End point timeframe
    From Screening (Day -28) to Post Therapy Day 30
    Notes
    [3] - 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: statistical analysis not provided as outcome measures reported as counts.
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
        TEAEs
    7
    6
    6
    3
    1
        TESAEs
    0
    3
    2
    0
    1
    No statistical analyses for this end point

    Primary: Number of Participants with Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Participants with Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [4]
    End point description
    Treatment-emergent adverse event (TEAEs) were defined as events that occur following the first injection of study treatment, or that started prior to the first injection and worsened during treatment. An adverse event (AE) was any untoward medical occurrence in a subject who received study drug without regard to possibility of causal relationship. Treatment-emergent adverse events were defined as adverse events/serious adverse events that started or worsened after the study drug treatment.
    End point type
    Primary
    End point timeframe
    From Screening (Day -28) to Post Therapy Day 30
    Notes
    [4] - 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: statistical analysis not provided as outcome measures reported as counts.
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
        Anaemia
    0
    3
    3
    0
    1
        Neutrophil count decreased
    1
    0
    0
    1
    1
        Neutropenia
    1
    1
    0
    0
    0
        Platelet count decreased
    0
    0
    1
    0
    1
        Thrombocytopenia
    0
    2
    0
    0
    0
        Leukocytosis
    0
    1
    0
    0
    0
        Hypoalbuminaemia
    2
    1
    2
    0
    1
        Hypertriglyceridaemia
    0
    3
    0
    1
    1
        Hyponatraemia
    0
    1
    3
    0
    1
        Alanine aminotransferase increased
    1
    1
    0
    1
    1
        Blood creatinine increased
    1
    2
    0
    0
    1
        Hypernatraemia
    2
    0
    1
    0
    1
        Hypocalcaemia
    0
    1
    2
    0
    1
        Aspartate aminotransferase increased
    1
    1
    0
    1
    0
        Hypokalaemia
    0
    0
    1
    1
    1
        Blood alkaline phosphatase increased
    0
    0
    0
    1
    1
        Hypercalcaemia
    0
    1
    0
    0
    1
        Hyperglycaemia
    0
    1
    1
    0
    0
        Hyperkalaemia
    0
    1
    1
    0
    0
        Hyperuricaemia
    1
    0
    0
    0
    1
        Hypophosphataemia
    0
    0
    1
    1
    0
        Blood albumin decreased
    1
    0
    0
    0
    0
        Blood bicarbonate increased
    0
    0
    0
    0
    1
        Blood bilirubin increased
    1
    0
    0
    0
    0
        Blood triglycerides increased
    0
    1
    0
    0
    0
        Haptoglobin decreased
    0
    0
    0
    0
    1
        Hyperchlorhydria
    0
    0
    1
    0
    0
        Hyperlipidaemia
    0
    0
    1
    0
    0
        Hypermagnesaemia
    0
    0
    0
    0
    1
        Hematuria
    0
    1
    1
    0
    1
    No statistical analyses for this end point

    Primary: Number of Participants with Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Participants with Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [5]
    End point description
    Treatment-emergent adverse event (TEAEs) were defined as events that occur following the first injection of study treatment, or that started prior to the first injection and worsened during treatment. An adverse event (AE) was any untoward medical occurrence in a subject who received study drug without regard to possibility of causal relationship. Treatment-emergent adverse events were defined as adverse events/serious adverse events that started or worsened after the study drug treatment.
    End point type
    Primary
    End point timeframe
    From Screening (Day -28) to Post Therapy Day 30
    Notes
    [5] - 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: statistical analysis not provided as outcome measures reported as counts.
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
        Hypotension
    1
    1
    1
    0
    1
        Hypertension
    0
    2
    1
    0
    0
        Pyrexia
    0
    1
    1
    0
    0
        Weight Increased
    1
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Number of Participants with Electrocardiogram (ECG) Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Participants with Electrocardiogram (ECG) Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [6]
    End point description
    Treatment-emergent adverse event (TEAEs) were defined as events that occur following the first injection of study treatment, or that started prior to the first injection and worsened during treatment. An adverse event (AE) was any untoward medical occurrence in a subject who received study drug without regard to possibility of causal relationship. Treatment-emergent adverse events were defined as adverse events/serious adverse events that started or worsened after the study drug treatment.
    End point type
    Primary
    End point timeframe
    From Screening (Day -28) to Post Therapy Day 30
    Notes
    [6] - 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: statistical analysis not provided as outcome measures reported as counts.
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
        Sinus Tachycardia
    0
    1
    0
    0
    0
        ECG QT Prolonged
    2
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Complete Response (CR)

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    End point title
    		 Percentage of Participants with Complete Response (CR)
    End point description
    The CR rate was defined as the proportion of subjects who had achieved CR based on both the evaluable population for efficacy.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Percentage of Participants
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Duration of Complete Response

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    End point title
    		 Duration of Complete Response
    End point description
    Duration of CR was measured from the first documentation of a CR to the time of relapse for the subgroup of participants with CR. Duration of CR was calculated using the Kaplan Meier method. Here '99999' indicates the parameter was not evaluated at that time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Months
        median (full range (min-max))
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Partial Response (PR)

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    End point title
    		 Percentage of Participants with Partial Response (PR)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Participants
    1
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Objective Response (OR)

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    End point title
    		 Percentage of Participants with Objective Response (OR)
    End point description
    OR was defined as the proportion of participants with CR or partial response (PR).
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Percentage of Participants
    1
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Time to Response (TTR)

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    End point title
    		 Time to Response (TTR)
    End point description
    TTR was measured from the start of moxetumomab pasudotox administration to the first documentation of response (CR or PR) and was only assessed in subjects who had achieved objective response (OR). Here '99999' indicates the parameter was not evaluated at that time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Months
        median (full range (min-max))
    0.79 (0.79 to 0.79)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Secondary: Duration of Objective Response (DOR)

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    End point title
    		 Duration of Objective Response (DOR)
    End point description
    DOR was measured from the first documentation of OR to the event of relapse. DOR was calculated using the Kaplan-Meier method. Here '99999' indicates the parameter was not evaluated at that time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Months
        median (full range (min-max))
    7.66 (7.66 to 7.66)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Secondary: Duration of Stable Disease (SD)

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    End point title
    		 Duration of Stable Disease (SD)
    End point description
    Duration of SD was defined as time from start of moxetumomab pasudotox administration to the event of progressive disease (PD)/relapse. Duration of SD was only calculated for the subgroup of subjects with best response of CR, PR, or SD, and was calculated using the Kaplan-Meier method. Here '99999' indicates the parameter was not evaluated at that time point.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Months
        median (full range (min-max))
    1.87 (0.79 to 8.41)
    99999 (99999 to 99999)
    0.95 (0.95 to 0.95)
    4.67 (3.71 to 5.62)
    No statistical analyses for this end point

    Secondary: Maximum Plasma Concentration (Cmax) of Moxetumomab pasudotox

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    End point title
    		 Maximum Plasma Concentration (Cmax) of Moxetumomab pasudotox
    End point description
    Maximum observed drug concentration of Moxetumomab pasudotox in plasma.
    End point type
    Secondary
    End point timeframe
    Pre-dose and End of infusion on Day 1, 3 and 5 of each cycle; 1, 3 and 6 hour after the end of infusion on Day 1 of each cycle
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    6
    6
    6
    3
    1
    Units: nanogram per milliliter (ng/ml)
        arithmetic mean (standard deviation)
    283 ± 159
    452 ± 141
    589 ± 366
    769 ± 221
    818 ± 99999
    No statistical analyses for this end point

    Secondary: Area Under Concentration-Time Curve From Dosing Extrapolated to Infinity (AUCinf) of Moxetumomab pasudotox

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    End point title
    		 Area Under Concentration-Time Curve From Dosing Extrapolated to Infinity (AUCinf) of Moxetumomab pasudotox
    End point description
    Area under the concentration versus time curve from zero to infinity (AUC) of Moxetumomab pasudotox in Plasma.
    End point type
    Secondary
    End point timeframe
    Pre-dose and End of infusion on Day 1, 3 and 5 of each cycle; 1, 3 and 6 hour after the end of infusion on Day 1 of each cycle
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    5
    5
    5
    3
    1
    Units: hour*nanogram per milligram (hr•ng/mL)
        arithmetic mean (standard deviation)
    1120 ± 928
    1640 ± 682
    2050 ± 1150
    2320 ± 1010
    1810 ± 99999
    No statistical analyses for this end point

    Secondary: Clearance (CL) of Moxetumomab pasudotox

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    End point title
    		 Clearance (CL) of Moxetumomab pasudotox
    End point description
    CL of drug is rate at which drug is metabolized or eliminated by normal biological processes and is influenced by fraction of dose absorbed.
    End point type
    Secondary
    End point timeframe
    Pre-dose and End of infusion on Day 1, 3 and 5 of each cycle; 1, 3 and 6 hour after the end of infusion on Day 1 of each cycle
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    5
    5
    5
    3
    1
    Units: milliliter per hour per kilogram
        arithmetic mean (standard deviation)
    31.6 ± 25.5
    22.2 ± 12.5
    26.9 ± 18.6
    25.2 ± 12.9
    33.1 ± 99999
    No statistical analyses for this end point

    Secondary: Elimination Half Life (t1/2) of Moxetumomab pasudotox

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    End point title
    		 Elimination Half Life (t1/2) of Moxetumomab pasudotox
    End point description
    Plasma decay half life is the time measured for the plasma concentration to decrease by one half.
    End point type
    Secondary
    End point timeframe
    Pre-dose and End of infusion on Day 1, 3 and 5 of each cycle; 1, 3 and 6 hour after the end of infusion on Day 1 of each cycle
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    5
    5
    5
    3
    1
    Units: hour
        arithmetic mean (standard deviation)
    1.55 ± 1.1
    1.87 ± 0.985
    1.68 ± 0.726
    1.87 ± 0.586
    0.901 ± 99999
    No statistical analyses for this end point

    Secondary: Number of Participants With Positive Anti-Drug Antibody

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    End point title
    		 Number of Participants With Positive Anti-Drug Antibody
    End point description
    The moxetumomab pasudotox specific bridging assay using the Meso Scale Discovery platform was employed to detect anti-drug antibodies (ADA).
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of the Treatment (Last dose of Last cycle)
    End point values
    		 Moxetumomab pasudotox (CAT-8015) 20 mcg/kg Moxetumomab pasudotox (CAT-8015) 30 mcg/kg Moxetumomab pasudotox (CAT-8015) 40 mcg/kg Moxetumomab pasudotox (CAT-8015) 50 mcg/kg Moxetumomab pasudotox (CAT-8015) 60 mcg/kg
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
    5
    4
    5
    2
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with CD22 Expression Levels

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    End point title
    		 Number of Participants with CD22 Expression Levels
    End point description
    CD22 Expression was analyzed using Prism® analysis. Flow cytometry was performed to quantitate the CD22 expression for the purpose of evaluating the relationship of CD22 expression with response to treatment.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of the Treatment (Last dose of Last cycle)
    End point values
    		 CLL (Safety Population) DLBCL (Safety Population) MCL (Safety Population) FL (Safety Population)
    Number of subjects analysed
    10
    6
    1
    3
    Units: Participants
    10
    1
    1
    4
    No statistical analyses for this end point

    Secondary: Number of Capillary Leak Syndrome (CLS) Participants with Weight Changes, Albumin, Hypotension, Edema, Hypoxia, and Pulmonary Adverse Events (AEs)

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    End point title
    		 Number of Capillary Leak Syndrome (CLS) Participants with Weight Changes, Albumin, Hypotension, Edema, Hypoxia, and Pulmonary Adverse Events (AEs)
    End point description
    The correlation of CLS and weight changes, albumin, hypotension, edema, hypoxia, and pulmonary AEs were examined.
    End point type
    Secondary
    End point timeframe
    From Screening (Day -28) to Post Therapy Day 30
    End point values
    		 CAT-8015 20 microgram per kilogram (mcg/kg) CAT-8015 30 microgram per kilogram (mcg/kg) CAT-8015 40 microgram per kilogram (mcg/kg) CAT-8015 50 microgram per kilogram (mcg/kg) CAT-8015 60 microgram per kilogram (mcg/kg)
    Number of subjects analysed
    7
    6
    6
    3
    1
    Units: Participants
    1
    0
    1
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Stable Disease (SD)

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    End point title
    		 Percentage of Participants with Stable Disease (SD)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) until Final Study Visit Post Therapy (up to 2 year after the last participant begins study drug treatment)
    End point values
    		 CLL (Evaluable for Efficacy) DLBCL (Evaluable for Efficacy) MCL (Evaluable for Efficacy) FL (Evaluable for Efficacy)
    Number of subjects analysed
    Units: Percentage of Participants
    10
    6
    1
    3
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Screening (Day -28) to Post Therapy Day 30
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    15.1
    Reporting groups
    Reporting group title
    CAT-8015 30 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 30 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 20 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 20 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 50 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 50 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 60 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 60 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Reporting group title
    CAT-8015 40 microgram per kilogram (mcg/kg)
    Reporting group description
    		 Participants received 40 mcg/kg Moxetumomab pasudotox (CAT-8015) as an intravenous (IV) infusion over 30 minutes on Days 1, 3, and 5 of every 28-day cycle. Dose escalation was to be continued to the Maximum tolerated dose (MTD) or Optimal biologic dose (OBD). Subsequent dose levels with a 10 mcg/kg increase from the previous dose level were possible if an MTD or OBD was not reached by 60 mcg/kg.

    Serious adverse events
    		 CAT-8015 30 microgram per kilogram (mcg/kg) CAT-8015 20 microgram per kilogram (mcg/kg) CAT-8015 50 microgram per kilogram (mcg/kg) CAT-8015 60 microgram per kilogram (mcg/kg) CAT-8015 40 microgram per kilogram (mcg/kg)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 6 (50.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    2 / 6 (33.33%)
         number of deaths (all causes)
    5
    2
    1
    0
    0
         number of deaths resulting from adverse events
    2
    0
    0
    0
    0
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Diffuse large b-cell lymphoma
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Tumour associated fever
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Capillary leak syndrome
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory distress syndrome
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal failure acute
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Lung infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 CAT-8015 30 microgram per kilogram (mcg/kg) CAT-8015 20 microgram per kilogram (mcg/kg) CAT-8015 50 microgram per kilogram (mcg/kg) CAT-8015 60 microgram per kilogram (mcg/kg) CAT-8015 40 microgram per kilogram (mcg/kg)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    7 / 7 (100.00%)
    3 / 3 (100.00%)
    1 / 1 (100.00%)
    6 / 6 (100.00%)
    Vascular disorders
    Capillary leak syndrome
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    0
    0
    3
    Haematoma
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    0
    0
    1
    Hypertension
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    2
    0
    0
    0
    1
    Hypotension
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    1
    0
    1
    7
    General disorders and administration site conditions
    Chills
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Face oedema
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 7 (28.57%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    2
    1
    1
    0
    Fatigue
         subjects affected / exposed
    0 / 6 (0.00%)
    5 / 7 (71.43%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    4 / 6 (66.67%)
         occurrences all number
    0
    6
    0
    0
    4
    Oedema peripheral
         subjects affected / exposed
    1 / 6 (16.67%)
    4 / 7 (57.14%)
    2 / 3 (66.67%)
    1 / 1 (100.00%)
    3 / 6 (50.00%)
         occurrences all number
    1
    5
    3
    1
    11
    Pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    3 / 6 (50.00%)
         occurrences all number
    0
    1
    0
    0
    4
    Dyspnoea
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    0
    1
    0
    2
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    0
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 7 (14.29%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    1
    1
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 7 (14.29%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    1
    0
    0
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    Blood creatinine increased
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    8
    1
    0
    0
    0
    Electrocardiogram qt prolonged
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 7 (28.57%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    Neutrophil count decreased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    1
    1
    0
    Platelet count decreased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 7 (14.29%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    1
    1
    0
    2
    Headache
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 7 (14.29%)
    2 / 3 (66.67%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    4
    1
    2
    1
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    3 / 6 (50.00%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    3 / 6 (50.00%)
         occurrences all number
    6
    0
    0
    1
    4
    Neutropenia
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    Thrombocytopenia
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    5
    0
    0
    0
    0
    Eye disorders
    Vision blurred
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    1
    0
    2
    Constipation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    0
    1
    1
    2
    Diarrhoea
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    3 / 6 (50.00%)
         occurrences all number
    0
    1
    0
    0
    4
    Dyspepsia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    0
    1
    Flatulence
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Nausea
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 7 (28.57%)
    2 / 3 (66.67%)
    0 / 1 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    2
    2
    0
    3
    Vomiting
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    0
    1
    0
    3
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    1
    5
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    2
    1
    0
    0
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    1
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    1
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    0
    1
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    0
    2
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 7 (28.57%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    Hypercalcaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    Hyperglycaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Hyperkalaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Hypernatraemia
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 7 (28.57%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    2
    0
    1
    1
    Hypertriglyceridaemia
         subjects affected / exposed
    3 / 6 (50.00%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    9
    0
    2
    1
    0
    Hyperuricaemia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 7 (14.29%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    1
    0
    Hypoalbuminaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 7 (28.57%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    2
    0
    1
    2
    Hypocalcaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    2 / 6 (33.33%)
         occurrences all number
    1
    0
    0
    1
    3
    Hypokalaemia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    1
    1
    Hyponatraemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 7 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    3 / 6 (50.00%)
         occurrences all number
    1
    0
    0
    1
    3
    Hypophosphataemia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 7 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    0
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Nov 2009
    Inclusion Criteria was revised.-Study Stopping Criteria was revised.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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