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Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia

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ClinicalTrials.gov Identifier: NCT01829711
Recruitment Status : Completed
First Posted : April 11, 2013
Results First Posted : August 27, 2018
Last Update Posted : April 8, 2020
Sponsor:
Information provided by (Responsible Party):
MedImmune LLC

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Leukemia, Hairy Cell
Interventions Drug: Moxetumomab pasudotox
Drug: IV Bag Protectant for Moxetumomab pasudotox
Enrollment 80
Recruitment Details The study was conducted across 14 countries (the USA, France, Italy, Germany, Belgium, Canada, Czech Republic, Ireland, Israel, Norway, Poland, Serbia, Spain, and United Kingdom).
Pre-assignment Details  
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Period Title: Overall Study
Started 80
Completed 37
Not Completed 43
Reason Not Completed
Lost to Follow-up             2
Withdrawal by Subject             3
Death             4
Started new therapies             17
Adverse Event             3
Due to progression of disease             13
Due to lack of response             1
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Baseline Participants 80
Hide Baseline Analysis Population Description
Intent-to-treat (ITT) population included participants who entered into the study and treated with moxetumomab pasudotox.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 80 participants
60.3  (11.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 80 participants
Female
17
  21.3%
Male
63
  78.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 80 participants
Hispanic or Latino
5
   6.3%
Not Hispanic or Latino
67
  83.8%
Unknown or Not Reported
8
  10.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 80 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   1.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   1.3%
White
70
  87.5%
More than one race
0
   0.0%
Unknown or Not Reported
8
  10.0%
1.Primary Outcome
Title Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review
Hide Description Durable CR was defined as overall response that meets blood, bone marrow and imaging criteria for CR, followed by a >180 day duration of hematologic remission (HR). CR requires all of the following to be present: No evidence of leukemic cells in peripheral blood and/or by routine H/E staining of bone marrow; Resolution of any hepatomegaly, splenomegaly, and abnormal (>= 2 cm minimum length) lymphadenopathy by CT or MRI (maximum diameter of spleen should be either < 17 cm or have decreased by >25% from its baseline.); HR requires normal complete blood count (CBC) as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Time Frame Full disease assessment (CBC, bone marrow and imaging) at end of treatment (EOT; up to 24 weeks) and post EOT Day 181; CBC monthly for 6 months post EOT, every 3 months post Day 181 for first 2 years and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
36.3
(25.8 to 47.8)
2.Primary Outcome
Title Percentage of Participants With Durable CR by Investigator's Assessment
Hide Description Durable CR was defined as overall response that meets blood, bone marrow and imaging criteria for CR, followed by a >180 day duration of HR. CR requires all of the following to be present: No evidence of leukemic cells in peripheral blood and/or by routine H/E staining of bone marrow; Resolution of any hepatomegaly, splenomegaly, and abnormal (>= 2 cm minimum length) lymphadenopathy by CT or MRI (maximum diameter of spleen should be either < 17 cm or have decreased by >25% from its baseline.); HR requires normal complete blood count (CBC) as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Time Frame Full disease assessment (CBC, bone marrow and imaging) at end of treatment (EOT; up to 24 weeks) and post EOT Day 181; CBC monthly for 6 months post EOT, every 3 months post Day 181 for first 2 years and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
48.8
(37.4 to 60.2)
3.Secondary Outcome
Title Percentage of Participants With Minimal Residual Disease (MRD) Positive or MRD Negative CR Assessed by Blinded Independent Central Review
Hide Description

The MRD status by blinded independent review refers specifically to results of central pathologist read of bone marrow biopsy by immunohistochemistry.

The CR with Positive or Negative MRD requires all of the following to be present:

  • No evidence of leukemic cells in the peripheral blood and/or by routine H/E staining of bone marrow. Minimal Residual Disease: CR with HCL evident in blood or in bone marrow biopsy by immunohistochemistry.
  • Resolution of any hepatomegaly, splenomegaly, and abnormal (>= 2 cm minimum length) lymphadenopathy by CT or MRI. Although a normal spleen size is not defined, the maximum diameter of the spleen should be either < 17 cm or have decreased by >25% from its baseline.
  • Normal CBC as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
MRD negative CR
33.8
(23.6 to 45.2)
MRD positive CR
7.5
(2.8 to 15.6)
4.Secondary Outcome
Title Percentage of Participants With MRD Positive or MRD Negative CR by Investigator's Assessment
Hide Description

The MRD status by investigator refers to results of investigator assessment of bone marrow biopsy or bone marrow aspirate by immunohistochemistry and/or flow cytometry.

The CR with Positive or Negative MRD requires all of the following to be present:

  • No evidence of leukemic cells in the peripheral blood and/or by routine H/E staining of bone marrow. Minimal Residual Disease: CR with HCL evident in blood or marrow by flow cytometry.
  • Resolution of any hepatomegaly, splenomegaly, and abnormal (>= 2 cm minimum length) lymphadenopathy by CT or MRI. Although a normal spleen size is not defined, the maximum diameter of the spleen should be either < 17 cm or have decreased by >25% from its baseline.
  • Normal CBC as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Time Frame Prior to each treatment cycle, end of treatment, and at follow-up visits every 3 months for the next 24 months and every 6 months thereafter (Approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
MRD negative CR
32.5
(22.4 to 43.9)
MRD positive CR
7.5
(2.8 to 15.6)
5.Secondary Outcome
Title Time to CR Assessed by Blinded Independent Central Review
Hide Description Time to CR was defined as the time from the start of moxetumomab pasudotox administration to the first documentation of CR.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Time to CR was evaluated for participants who achieved CR per independent central review.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (Full Range)
Unit of Measure: Months
5.9
(1.8 to 13.2)
6.Secondary Outcome
Title Duration of CR Assessed by Blinded Independent Central Review
Hide Description Duration of CR was defined as the duration from documentation of CR to the time of relapse from CR. Relapse from CR was defined as any CR criteria (blood counts, imaging or bone marrow) no longer consistent with CR. CR requires all of the following to be present: No evidence of leukemic cells in peripheral blood and/or by routine H/E staining of bone marrow; Resolution of any hepatomegaly, splenomegaly, and abnormal (>= 2 cm minimum length) lymphadenopathy by CT or MRI (maximum diameter of spleen should be either < 17 cm or have decreased by >25% from its baseline); normal CBC as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Duration of CR was evaluated for participants who achieved CR per independent central review.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (95% Confidence Interval)
Unit of Measure: Months
62.8
(35.7 to 62.8)
7.Secondary Outcome
Title Duration of Hematologic Remission
Hide Description

Duration of HR was defined as the duration from documentation of HR to the time of relapse. HR was defined as the blood counts required for CR as normal CBC as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.

Duration of HR was censored on the date of the last hematologic assessment for participants who have no documented relapse based on blood count prior to data cutoff, dropout, or initiation of alternative anticancer therapy.

Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Duration of HR was evaluated for participants who achieved HR.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (95% Confidence Interval)
Unit of Measure: Months
45.8
(25.9 to 71.5)
8.Secondary Outcome
Title Time to Hematologic Remission
Hide Description Time to HR was defined as the time from the start of moxetumomab pasudotox administration to the first documentation of HR. HR was defined as the blood counts required for CR as normal CBC as exhibited by: Neutrophils >= 1.5 x 10^9/L, Platelets >= 100 x 10^9/L, and hemoglobin >= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Time to HR was evaluated for participants in the ITT population who achieved HR.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (Full Range)
Unit of Measure: Months
1.1
(0.2 to 12.9)
9.Secondary Outcome
Title Percentage of Participants With Objective Response (OR) Assessed by Blinded Independent Central Review
Hide Description The OR was defined as number of participants with a best response of CR or PR. CR requires all of following to be present: No evidence of leukemic cells in peripheral blood and/or by routine H/E staining of bone marrow; Resolution of any hepatomegaly, splenomegaly, and abnormal (>=2 cm minimum length) lymphadenopathy by CT or MRI (maximum diameter of spleen should be either <17 cm or have decreased by >25% from its baseline); normal CBC (Neutrophils >=1.5 x 10^9/L, Platelets >=100 x 10^9/L, and hemoglobin >=11.0 g/dL) without transfusions or growth factors for at least 4 weeks. PR requires all of following for a period of at least 4 weeks: >=50% decrease or normalization (<5.0 x 10^9/L) in peripheral blood lymphocyte count and >=50% reduction in lymphadenopathy and in abnormal haepatosplenomegaly by CT or MRI from pre-treatment baseline value; normal CBC as mentioned above or 50% improvement in CBC values over baseline without transfusions or growth factors for at least 4 weeks.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
75.0
(64.1 to 84.0)
10.Secondary Outcome
Title Percentage of Participants With Objective Response by Investigator's Assessment
Hide Description The OR was defined as number of participants with a best response of CR or PR. CR requires all of following to be present: No evidence of leukemic cells in peripheral blood and/or by routine H/E staining of bone marrow; Resolution of any hepatomegaly, splenomegaly, and abnormal (>=2 cm minimum length) lymphadenopathy by CT or MRI (maximum diameter of spleen should be either <17 cm or have decreased by >25% from its baseline); normal CBC (Neutrophils >=1.5 x 10^9/L, Platelets >=100 x 10^9/L, and hemoglobin >=11.0 g/dL) without transfusions or growth factors for at least 4 weeks. PR requires all of following for a period of at least 4 weeks: >=50% decrease or normalization (<5.0 x 10^9/L) in peripheral blood lymphocyte count and >=50% reduction in lymphadenopathy and in abnormal haepatosplenomegaly by CT or MRI from pre-treatment baseline value; normal CBC as mentioned above or 50% improvement in CBC values over baseline without transfusions or growth factors for at least 4 weeks.
Time Frame Prior to each treatment cycle, end of treatment, and at follow-up visits every 3 months for the next 24 months and every 6 months thereafter (Approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
78.8
(68.2 to 87.1)
11.Secondary Outcome
Title Time to Objective Response Assessed by Blinded Independent Central Review
Hide Description Time to OR was defined as the time from the start of moxetumomab pasudotox administration to the first documentation of OR (CR or PR).
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Time to OR was evaluated for participants who achieved OR per independent central review.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (Full Range)
Unit of Measure: Months
5.7
(1.8 to 12.9)
12.Secondary Outcome
Title Duration of Objective Response Assessed by Blinded Independent Central Review
Hide Description

Duration of OR was defined as the time from the first documentation of objective response (CR or PR) to the date of relapse.

Duration of OR was censored on the date of last disease assessment or hematologic assessment for participants who have no documented relapse prior to data cut-off, dropout, or the initiation of alternative anticancer therapy.

Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Duration of OR was evaluated for participants who achieved OR per independent central review.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (95% Confidence Interval)
Unit of Measure: Months
66.7
(25.4 to 66.7)
13.Secondary Outcome
Title Progression-free Survival (PFS) Assessed by Blinded Independent Central Review
Hide Description The PFS was defined as the time from the start of moxetumomab pasudotox administration to the earliest date of a disease assessment showing a progressive disease/relapse, earliest date of hematologic relapse or date of death, whichever was earlier. The PFS was censored on the date of last disease assessment or hematologic assessment for participants who are alive with no documented relapse or PD prior to data cut-off, dropout, or the initiation of alternative anticancer therapy.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (95% Confidence Interval)
Unit of Measure: Months
41.5
(28.1 to 71.7)
14.Secondary Outcome
Title Time to Treatment Failure (TTF) Assessed by Blinded Independent Central Review
Hide Description The TTF was defined as the time from the start of moxetumomab pasudotox administration to the date of the first of relapse, progressive disease, initiation of alternative anticancer therapy, or death due to disease or disease-related complication. The TTF was censored on the date of last disease assessment or hematologic assessment for participants who are alive with no documented relapse or PD prior to data cut-off, dropout, or the initiation of alternative anticancer therapy and also censored for death not accompanied by relapse.
Time Frame Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years)
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included participants who entered into the study and treated with moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (95% Confidence Interval)
Unit of Measure: Months
41.5
(28.1 to 71.7)
15.Secondary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
Hide Description An Adverse Event (AE) is any unfavourable and unintended sign, symptoms, or diseases temporally associated with use of study drug, whether or not considered related to study drug. A serious adverse event (SAE) is an AE that results in death, initial or prolonged inpatient hospitalization, life-threatening, persistent or significant disability/incapacity, congenital anomaly/birth defect, or an important medical event. TEAEs and TESAEs are defined as AEs and SAEs present at baseline that worsened in intensity after administration of study drug, or events absent at baseline that emerged after administration of study drug.
Time Frame From the start of study treatment (Day 1) through 4-6 weeks after last dose of Cycle 6 (28-day cycle) (approximately 7 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included participants who received at least 1 dose of moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Count of Participants
Unit of Measure: Participants
Any TEAEs
79
  98.8%
Any TESAEs
28
  35.0%
16.Secondary Outcome
Title Number of Participants With Abnormal Clinical Laboratory Results Reported as TEAEs
Hide Description An abnormal laboratory finding which required an action or intervention by the investigator, or a finding judged by the investigator to represent a change beyond the range of normal physiologic fluctuation were reported as AEs. The TEAEs are defined as AEs present at baseline that worsened in intensity after administration of study drug, or events absent at baseline that emerged after administration of study drug, up to 30 days after the last dose of study drug (approximately 7 months).
Time Frame From the start of study treatment (Day 1) through 4-6 weeks after last dose of Cycle 6 (28-day cycle) (approximately 7 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included participants who received at least 1 dose of moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Count of Participants
Unit of Measure: Participants
Anaemia
17
  21.3%
Disseminated intravascular coagulation
1
   1.3%
Febrile neutropenia
5
   6.3%
Iron deficiency anaemia
1
   1.3%
Leukopenia
2
   2.5%
Lymphopenia
1
   1.3%
Neutropenia
4
   5.0%
Thrombocytopenia
3
   3.8%
Activated partial thromboplastin time prolonged
1
   1.3%
Lymphocyte count decreased
16
  20.0%
Lymphocyte count increased
1
   1.3%
Neutrophil count decreased
6
   7.5%
Platelet count decreased
9
  11.3%
White blood cell count decreased
8
  10.0%
Aspartate aminotransferase increased
15
  18.8%
Blood albumin decreased
1
   1.3%
Blood alkaline phosphatase increased
4
   5.0%
Blood bicarbonate decreased
2
   2.5%
Blood bilirubin increased
5
   6.3%
Blood creatinine increased
9
  11.3%
Blood triglycerides increased
1
   1.3%
Gamma-glutamyltransferase increased
1
   1.3%
Lipase increased
2
   2.5%
Hyperglycaemia
8
  10.0%
Hyperkalaemia
6
   7.5%
Hypermagnesaemia
3
   3.8%
Hypernatraemia
4
   5.0%
Hypertriglyceridaemia
2
   2.5%
Hypoalbuminaemia
16
  20.0%
Hypocalcaemia
19
  23.8%
Hypoglycaemia
2
   2.5%
Hypokalaemia
13
  16.3%
Hypomagnesaemia
6
   7.5%
Hyponatraemia
9
  11.3%
Haematuria
6
   7.5%
Haemoglobinuria
2
   2.5%
Proteinuria
1
   1.3%
17.Secondary Outcome
Title Number of Participants With Abnormal Vital Signs Reported as TEAEs
Hide Description An abnormal vital signs that were judged by the investigator to be medically significant were reported as AEs. The TEAEs are defined as AEs present at baseline that worsened in intensity after administration of study drug, or events absent at baseline that emerged after administration of study drug, up to 30 days after the last dose of study drug (approximately 7 months).
Time Frame From the start of study treatment (Day 1) through 4-6 weeks after last dose of Cycle 6 (28-day cycle) (approximately 7 months)
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Safety population included participants who received at least 1 dose of moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
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Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Count of Participants
Unit of Measure: Participants
Dyspnoea
9
  11.3%
Dyspnoea exertional
3
   3.8%
Hypertension
12
  15.0%
Hypotension
6
   7.5%
Pyrexia
25
  31.3%
18.Secondary Outcome
Title Number of Participants With Abnormal Electrocardiogram (ECG) Reported as TEAEs
Hide Description An abnormal ECG findings that were judged by the investigator to be medically significant were reported as AEs. The TEAEs are defined as AEs present at baseline that worsened in intensity after administration of study drug, or events absent at baseline that emerged after administration of study drug, up to 30 days after the last dose of study drug (approximately 7 months).
Time Frame From the start of study treatment (Day 1) through 4-6 weeks after last dose of Cycle 6 (28-day cycle) (approximately 7 months)
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Safety population included participants who received at least 1 dose of moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Count of Participants
Unit of Measure: Participants
Angina pectoris
2
   2.5%
Atrial fibrillation
1
   1.3%
Atrioventricular block first degree
3
   3.8%
Bundle branch block left
1
   1.3%
Left ventricular dysfunction
1
   1.3%
Palpitations
1
   1.3%
Pericardial effusion
1
   1.3%
Sinus bradycardia
2
   2.5%
Sinus tachycardia
6
   7.5%
Supraventricular tachycardia
1
   1.3%
Tachycardia
1
   1.3%
Ventricular arrhythmia
1
   1.3%
19.Secondary Outcome
Title Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox
Hide Description The Tmax of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Pharmacokinetic (PK) population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Median (Full Range)
Unit of Measure: Hours
Cycle 1 Day 1 Number Analyzed 75 participants
0.567
(0.433 to 1.30)
Cycle 1 Day 5 Number Analyzed 71 participants
0.550
(0.417 to 2.45)
Cycle 2 Day 1 Number Analyzed 69 participants
0.583
(0.500 to 1.75)
20.Secondary Outcome
Title Maximum Observed Plasma Concentration (Cmax) of Moxetumomab Pasudotox
Hide Description The Cmax of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
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Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: ng/mL
Cycle 1 Day 1 Number Analyzed 75 participants
192  (162)
Cycle 1 Day 5 Number Analyzed 71 participants
435  (233)
Cycle 2 Day 1 Number Analyzed 69 participants
379  (262)
21.Secondary Outcome
Title Time of Last (Tlast) Measurable Concentration of Moxetumomab Pasudotox
Hide Description The Tlast of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
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Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: Hours
Cycle 1 Day 1 Number Analyzed 75 participants
0.841  (0.866)
Cycle 1 Day 5 Number Analyzed 71 participants
3.37  (2.38)
Cycle 2 Day 1 Number Analyzed 69 participants
2.16  (1.43)
22.Secondary Outcome
Title Area Under the Plasma Concentration-time Curve From Time Zero to Time of the Last Quantifiable Concentration (AUC0-last) of Moxetumomab Pasudotox
Hide Description The AUC0-last of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
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Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
Cycle 1 Day 1 Number Analyzed 75 participants
120  (261)
Cycle 1 Day 5 Number Analyzed 71 participants
820  (721)
Cycle 2 Day 1 Number Analyzed 69 participants
626  (610)
23.Secondary Outcome
Title Area Under the Plasma Concentration-time Curve From Time Zero to 3 Hours (AUC0-3hr) Post End of Moxetumomab Pasudotox
Hide Description The AUC0-3hr of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, and 3 hr post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
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Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
Cycle 1 Day 1 Number Analyzed 6 participants
869  (200)
Cycle 1 Day 5 Number Analyzed 54 participants
856  (370)
Cycle 2 Day 1 Number Analyzed 37 participants
1030  (333)
24.Secondary Outcome
Title Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of Moxetumomab Pasudotox
Hide Description The AUC0-inf of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point. Data for Cycle 1 Day 1 was not reported as no evaluable participants for the calculation of the concerned parameters (ie., data were not sufficient).
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
Cycle 1 Day 1 Number Analyzed 0 participants
Cycle 1 Day 5 Number Analyzed 49 participants
1300  (742)
Cycle 2 Day 1 Number Analyzed 22 participants
1470  (541)
25.Secondary Outcome
Title Area Under the Plasma Concentration-time Curve Extrapolated (AUCExt) of Moxetumomab Pasudotox
Hide Description The AUCExt of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
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Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
Cycle 1 Day 1 Number Analyzed 6 participants
13.2  (3.04)
Cycle 1 Day 5 Number Analyzed 49 participants
14.3  (5.72)
Cycle 2 Day 1 Number Analyzed 22 participants
20.2  (7.62)
26.Secondary Outcome
Title Systemic Clearance (CL) of Moxetumomab Pasudotox
Hide Description The CL of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point. Data for Cycle 1 Day 1 was not reported as no evaluable participants for the calculation of the concerned parameters (ie., data were not sufficient).
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: mL/hr/kg
Cycle 1 Day 1 Number Analyzed 0 participants
Cycle 1 Day 5 Number Analyzed 49 participants
44.6  (30.5)
Cycle 2 Day 1 Number Analyzed 22 participants
31.8  (13.7)
27.Secondary Outcome
Title Terminal Half Life (t1/2) of Moxetumomab Pasudotox
Hide Description The t1/2 of moxetumomab pasudotox is reported.
Time Frame Cycle 1 Day 1 (pre-dose; 5 mins and 3 hr post dose); Cycle 1 Day 5 (pre-dose; 5 mins, 1 hr, 3 hr, and 6 hrs post dose); and Cycle 2 Day 1 (pre-dose; 5 mins and 3 hr post dose)
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Hide Analysis Population Description
The PK population included all participants who received at least 1 dose of moxetumomab pasudotox and provided at least 1 baseline and post-baseline concentration-time data point. Data for Cycle 1 Day 1 was not reported as no evaluable participants for the calculation of the concerned parameters (ie., data were not sufficient).
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Mean (Standard Deviation)
Unit of Measure: Hours
Cycle 1 Day 1 Number Analyzed 0 participants
Cycle 1 Day 5 Number Analyzed 49 participants
1.38  (0.632)
Cycle 2 Day 1 Number Analyzed 22 participants
1.39  (0.351)
28.Secondary Outcome
Title Percentage of Participants With Positive Anti-drug Antibodies (ADA), Neutralizing Anti-drug Antibodies (nAb) and Specificity (CD22 and PE38) Positive to Moxetumomab Pasudotox
Hide Description Participants with ADA positive, nAb positive, cluster of differentiation 22 (CD22) positive of ADA positive/NAb positive, and pseudomonas exotoxin 38 (PE38) positive of ADA positive/NAb positive to moxetumomab pasudotox at any visit are reported.
Time Frame Pre-infusion on Day 1 of Cycles 1, 2, 3, and 5; at the End of Treatment (4 to 6 weeks after the last dose; approximately 7 months)
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Hide Analysis Population Description
Safety population included participants who received at least 1 dose of moxetumomab pasudotox.
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description:
Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
Overall Number of Participants Analyzed 80
Measure Type: Number
Unit of Measure: Percentage of Participants
ADA positive 87.5
ADA and NAb positive 83.8
Specificity CD22 positive of ADA+/NAb+ 55.2
Specificity PE38 positive of ADA+/NAb+ 98.5
Time Frame For TEAEs and TESAEs: From the start of study treatment (Day 1) through 4-6 weeks after last dose of Cycle 6 (28-day cycle) (approximately 7 months) All-cause mortality data: From the start of study treatment (Day 1) through end of study (approximately 6 years)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Moxetumomab Pasudotox 40 µg/kg
Hide Arm/Group Description Participants received intravenous infusion of moxetumomab pasudotox 40 µg/kg on Days 1, 3, and 5 of each 28-day cycle for up to 6 cycles, until documentation of complete response, progressive disease, initiation of alternate therapy, or unacceptable toxicity.
All-Cause Mortality
Moxetumomab Pasudotox 40 µg/kg
Affected / at Risk (%)
Total   4/80 (5.00%)    
Hide Serious Adverse Events
Moxetumomab Pasudotox 40 µg/kg
Affected / at Risk (%) # Events
Total   28/80 (35.00%)    
Blood and lymphatic system disorders   
Febrile neutropenia  1  2/80 (2.50%)  3
Haemolytic uraemic syndrome  1  6/80 (7.50%)  6
Cardiac disorders   
Left ventricular dysfunction  1  1/80 (1.25%)  1
Gastrointestinal disorders   
Diarrhoea  1  1/80 (1.25%)  1
Nausea  1  1/80 (1.25%)  1
Neutropenic colitis  1  1/80 (1.25%)  1
Vomiting  1  1/80 (1.25%)  1
General disorders   
Fatigue  1  1/80 (1.25%)  1
Multiple organ dysfunction syndrome  1  1/80 (1.25%)  1
Pain  1  1/80 (1.25%)  1
Pyrexia  1  5/80 (6.25%)  5
Infections and infestations   
Clostridium difficile colitis  1  1/80 (1.25%)  1
Erysipelas  1  1/80 (1.25%)  1
Infection  1  1/80 (1.25%)  1
Lung infection  1  1/80 (1.25%)  1
Pneumonia  1  1/80 (1.25%)  1
Pneumonia fungal  1  1/80 (1.25%)  1
Sepsis syndrome  1  1/80 (1.25%)  1
Septic shock  1  1/80 (1.25%)  1
Upper respiratory tract infection  1  2/80 (2.50%)  2
Injury, poisoning and procedural complications   
Infusion related reaction  1  2/80 (2.50%)  2
Investigations   
Blood creatinine increased  1  1/80 (1.25%)  1
Haptoglobin decreased  1  1/80 (1.25%)  1
Neutrophil count decreased  1  1/80 (1.25%)  1
Platelet count decreased  1  1/80 (1.25%)  1
Weight increased  1  1/80 (1.25%)  1
Musculoskeletal and connective tissue disorders   
Rhabdomyolysis  1  1/80 (1.25%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Glioblastoma  1  1/80 (1.25%)  1
Psychiatric disorders   
Mental status changes  1  1/80 (1.25%)  1
Renal and urinary disorders   
Acute kidney injury  1  1/80 (1.25%)  1
Haematuria  1  1/80 (1.25%)  1
Renal failure  1  1/80 (1.25%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/80 (1.25%)  1
Dyspnoea  1  1/80 (1.25%)  1
Hypoxia  1  2/80 (2.50%)  2
Pharyngeal cyst  1  1/80 (1.25%)  1
Respiratory failure  1  1/80 (1.25%)  1
Tachypnoea  1  1/80 (1.25%)  1
Skin and subcutaneous tissue disorders   
Rash  1  1/80 (1.25%)  1
Vascular disorders   
Capillary leak syndrome  1  4/80 (5.00%)  5
1
Term from vocabulary, MedDRA 19.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Moxetumomab Pasudotox 40 µg/kg
Affected / at Risk (%) # Events
Total   77/80 (96.25%)    
Blood and lymphatic system disorders   
Anaemia  1  17/80 (21.25%)  41
Neutropenia  1  4/80 (5.00%)  5
Cardiac disorders   
Sinus tachycardia  1  6/80 (7.50%)  6
Eye disorders   
Cataract  1  4/80 (5.00%)  4
Dry eye  1  6/80 (7.50%)  6
Vision blurred  1  7/80 (8.75%)  8
Gastrointestinal disorders   
Abdominal distension  1  10/80 (12.50%)  12
Abdominal pain  1  7/80 (8.75%)  7
Abdominal pain upper  1  4/80 (5.00%)  5
Constipation  1  18/80 (22.50%)  21
Diarrhoea  1  17/80 (21.25%)  20
Dyspepsia  1  4/80 (5.00%)  5
Flatulence  1  5/80 (6.25%)  5
Nausea  1  28/80 (35.00%)  48
Vomiting  1  14/80 (17.50%)  17
General disorders   
Asthenia  1  10/80 (12.50%)  30
Chills  1  15/80 (18.75%)  18
Face oedema  1  11/80 (13.75%)  16
Fatigue  1  26/80 (32.50%)  40
Non-cardiac chest pain  1  4/80 (5.00%)  4
Oedema  1  4/80 (5.00%)  5
Oedema peripheral  1  31/80 (38.75%)  47
Peripheral swelling  1  4/80 (5.00%)  5
Pyrexia  1  22/80 (27.50%)  35
Infections and infestations   
Nasopharyngitis  1  4/80 (5.00%)  4
Rhinitis  1  4/80 (5.00%)  4
Sinusitis  1  4/80 (5.00%)  4
Upper respiratory tract infection  1  5/80 (6.25%)  5
Injury, poisoning and procedural complications   
Infusion related reaction  1  5/80 (6.25%)  6
Investigations   
Alanine aminotransferase increased  1  17/80 (21.25%)  43
Aspartate aminotransferase increased  1  15/80 (18.75%)  38
Blood alkaline phosphatase increased  1  4/80 (5.00%)  8
Blood bilirubin increased  1  5/80 (6.25%)  14
Blood creatinine increased  1  8/80 (10.00%)  15
Haptoglobin decreased  1  6/80 (7.50%)  6
Lymphocyte count decreased  1  16/80 (20.00%)  73
Neutrophil count decreased  1  5/80 (6.25%)  10
Platelet count decreased  1  9/80 (11.25%)  18
Weight increased  1  6/80 (7.50%)  7
White blood cell count decreased  1  8/80 (10.00%)  15
Metabolism and nutrition disorders   
Decreased appetite  1  11/80 (13.75%)  12
Hyperglycaemia  1  8/80 (10.00%)  15
Hyperkalaemia  1  6/80 (7.50%)  8
Hypernatraemia  1  4/80 (5.00%)  5
Hypoalbuminaemia  1  16/80 (20.00%)  51
Hypocalcaemia  1  19/80 (23.75%)  39
Hypokalaemia  1  13/80 (16.25%)  22
Hypomagnesaemia  1  6/80 (7.50%)  8
Hyponatraemia  1  9/80 (11.25%)  17
Hypophosphataemia  1  19/80 (23.75%)  47
Musculoskeletal and connective tissue disorders   
Arthralgia  1  13/80 (16.25%)  22
Back pain  1  12/80 (15.00%)  12
Musculoskeletal chest pain  1  4/80 (5.00%)  4
Musculoskeletal pain  1  5/80 (6.25%)  7
Myalgia  1  11/80 (13.75%)  16
Pain in extremity  1  12/80 (15.00%)  14
Nervous system disorders   
Dizziness  1  8/80 (10.00%)  12
Dysgeusia  1  5/80 (6.25%)  5
Headache  1  26/80 (32.50%)  37
Paraesthesia  1  7/80 (8.75%)  8
Psychiatric disorders   
Anxiety  1  9/80 (11.25%)  9
Insomnia  1  8/80 (10.00%)  9
Renal and urinary disorders   
Haematuria  1  5/80 (6.25%)  5
Respiratory, thoracic and mediastinal disorders   
Cough  1  7/80 (8.75%)  8
Dyspnoea  1  8/80 (10.00%)  10
Nasal congestion  1  5/80 (6.25%)  5
Oropharyngeal pain  1  6/80 (7.50%)  8
Pleural effusion  1  5/80 (6.25%)  6
Skin and subcutaneous tissue disorders   
Pruritus  1  6/80 (7.50%)  12
Rash  1  4/80 (5.00%)  4
Rash maculo-papular  1  4/80 (5.00%)  5
Vascular disorders   
Flushing  1  4/80 (5.00%)  4
Hypertension  1  12/80 (15.00%)  38
Hypotension  1  6/80 (7.50%)  8
1
Term from vocabulary, MedDRA 19.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
MedImmune has 60 days to review results communications prior to public release and may delete information that compromises on-going studies or is considered proprietary. This restriction is not intended to compromise the objective scientific integrity of the manuscript, it being understood that results shall be published regardless of outcome.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Priti Patel
Organization: MedImmune, LLC
Phone: +1-650-264-9079
EMail: information.center@astrazeneca.com
Layout table for additonal information
Responsible Party: MedImmune LLC
ClinicalTrials.gov Identifier: NCT01829711    
Other Study ID Numbers: 130106
13-C-0106 ( Other Identifier: CTEP )
CD-ON-CAT-8015-1053 ( Other Identifier: MedImmune )
First Submitted: April 9, 2013
First Posted: April 11, 2013
Results First Submitted: May 24, 2018
Results First Posted: August 27, 2018
Last Update Posted: April 8, 2020