A Phase I, Multicenter, Dose Escalation Study of CAT-8015 in Participants with Chronic Leukemia

Study identifier:CAT-8015-1002

ClinicalTrials.gov identifier:NCT00587457

EudraCT identifier:N/A

CTIS identifier:N/A

Terminated/Withdrawn

Official Title

A Phase I, Multicenter, Dose-Escalation Study of CAT-8015 in Patients with Relapsed or Refactory Chronic Lymphocytic Leukemia (CLL) Prolymphocytic Leukemia (PLL), or Small Lymphocytic Leukemia (SLL)

Medical condition

Leukemia, Lymphoma, Chronic Lymphocytic

Phase

Phase 1

Healthy volunteers

Study drug

CAT-8015 5 mcg/kg, CAT-8015 10 mcg/kg, CAT-8015 20 mcg/kg

Sex

All

Actual Enrollment

Study type

Interventional

Age

18 Years +

Date

Study Start Date: 07 Mar 2007

Primary Completion Date: 07 Apr 2008

Study Completion Date: 07 Apr 2008

Study design

Allocation: Non-randomized
Endpoint Classification: Safety
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Verification:

Verified 01 Jun 2017 by MedImmune, LLC

Collaborators

Cambridge Antibody Technology

Inclusion and exclusion criteria

Inclusion Criteria

- Confirmed diagnosis of B-Cell Leukemia [(chronic lymphocytic leukemia (CLL), prolymphocytic leukemia (PLL), or Small lymphocytic leukemia (SLL)]
- Measurable Disease
- Disease characteristics: Participants with CLL or SLL are eligible if they have failed 2 or more prior courses of standard chemo and/or biologic therapy (example, Rituxan) and PLL will be eligible if they have failed at least one prior standard chemotherapeutic regimen. Medical indications for treatment include progressive disease-related symptoms, progressive cytopenias due to marrow involvement, progressive or painful splenomegaly or adenopathy, rapidly increasing lymphocytosis, autoimmune hemolytic anemia or thrombocytopenia and increased frequency of infections.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Participants with other cancers who meet eligibility criteria and have had less than 5 years of disease-free survival will be considered on a case-by-case basis
- Life expectancy of greater than 6 months, as assessed by the principal investigator
- Must be able to understand and sign the informed consent
- Must be at least 18 years old
- Female and Male participants agree to use an approved method of contraception during the study

Exclusion Criteria

- History of allogeneic bone marrow transplant.
- Documented and ongoing central nervous system involvement with their malignant disease [history of central nervous system (CNS) involvement is not an exclusion criterion]
- Pregnant or breast-feeding females
- Participants who plasma contains either a significant level of antibody to CAT-8015 as measured by ELISA, or antibody that neutralizes the binding of CAT-8015 to CD22 as measured by a competition ELISA.
- HIV positive serology (due to increased risk of severe infection and unknown interaction of CAT-8015 with antiretroviral drugs)
- Hepatitis B surface antigen positive
- Uncontrolled, symptomatic, intercurrent illness including but not limited to: infections requiring systemic antibiotics, congestive heart failure, unstable angina pectoris, cardiac arrhythmia, psychiatric illness, or social situations that would limit compliance with study requirements.
- Hepatic function: Serum transaminases [either alanine aminotransferase (ALT) or aspartate aminotransferase (AST)] or direct bilirubin greater than or equal to Grade 2, unless bilirubin is due to Gilbert's disease
- Renal function: Serum creatinine clearance is less than or equal to 60 millilitre per minute (mL/min) as estimated by Cockcroft-Gault formula
Hematologic function:
- The ANC less than 1000/cubic millimeter (cmm), or platelet count less than 50,000/cmm, if these cytopenias are not judged by the investigator to be due to underlying disease (that is, potentially reversible with anti-neoplastic therapy).
- A participant will not be excluded because of pancytopenia greater than or equal to Grade 3, or erythropoietin dependence, if it is due to disease, based on the results of bone marrow studies.
- Baseline coagulopathy greater than or equal to grade 3 unless due to anticoagulant therapy
Pulmonary function:
- Participants with less than 50 percent (%) of predicted forced expiratory volume (FEV-1) or less than 50% of predicted diffusing capacity for carbon monoxide (DLCO) corrected for hemoglobin concentration and alveolar volume. Note: Participants with no prior history of pulmonary illness are not required to have pulmonary function test (PFTs). FEV1 will be assessed after bronchodilator therapy.
Recent prior therapy:
- Cytotoxic chemotherapy, corticosteroids (except stable doses of prednisone), whole body electron beam radiation therapy, hormonal, biologic or other standard or any investigational therapy of the malignancy for 3 weeks prior to entry into the trial.
- Less than or equal to 1 month prior monoclonal antibody therapy (that is, rituximab)
- Participants who are receiving or have received radiation therapy less than 3 weeks prior to study entry will not be excluded providing the volume of the bone marrow treated is less than 10% and also the participant has measurable disease outside the radiation report.
- Any history of prior pseudomonas - exotoxin immunotoxin administrator

Location

Research Site

Lodz, Poland

Location

Research Site

Indianapolis, IN, United States

Location

Research Site

Bethesda, MD, United States

Arms	Assigned Interventions
Experimental: CAT-8015 5 microgram per kilogram (mcg/kg) Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	Drug: CAT-8015 5 mcg/kg Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Experimental: CAT-8015 10 mcg/kg Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	Drug: CAT-8015 10 mcg/kg Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Experimental: CAT-8015 20 mcg/kg Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	Drug: CAT-8015 20 mcg/kg Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 11 participants were enrolled, of which all participants discontinued from the treatment.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Participant Flow: Overall Study

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
STARTED	3	3	5
COMPLETED	0	0	0
NOT COMPLETED	3	3	5
Adverse Event	1	2	1
Disease progression	2	0	2
Initiation of alternative therapy	0	1	0
Undefined	0	0	1
Development of neutralizing Abs	0	0	1

Baseline Characteristics

Analysis Population Description -- Explanation of how the number of participants for analysis was determined.

Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Baseline Measures

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)	Total
Number of Participants [units: Participants]	3	3	5	22
Age Continuous [units: Years] Mean ± Standard Deviation	58.7 ± 2.3	62.0 ± 4.4	61.6 ± 7.5	60.9 ± 5.4
Gender, Male/Female [units: Participants]
Female	1	1	1	3
Male	2	2	4	8

Outcome Measures

1. Primary Outcome Measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) [ Time Frame: From start of study drug administration until 30 days after the last dose of study drug ]

Measure Type	Primary
Measure Name	Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
Measure Description	An adverse event (AE) events present at baseline that worsened in intensity after administration of investigational product or events absent at baseline that emerged after administration of study drug, for the period extending to 30 days after the last dose of study drug. A serious adverse event (SAE) is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening situation (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a participant who received moxetumomab pasudotox. Treatment-emergent were events between administration of investigational product and Day 28 that were absent before treatment or that worsened relative to pretreatment state.
Time Frame	From start of study drug administration until 30 days after the last dose of study drug
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) [units: Participants]
TEAEs	3	3	5
TESAEs	1	2	3

No statistical analysis provided for Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

2. Primary Outcome Measure: Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: From start of study drug administration until 30 days after the last dose of study drug ]

Measure Type	Primary
Measure Name	Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Measure Description	The vital sign abnormalities which require an action or intervention by the investigator, or a finding judged by the investigator were reported as an adverse event. TEAEs were events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug, for the period extending to 30 days after the last dose of study drug.
Time Frame	From start of study drug administration until 30 days after the last dose of study drug
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [units: Participants]
Pyrexia	1	0	1
Sinus tachycardia	1	0	0

No statistical analysis provided for Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

3. Primary Outcome Measure: Number of Participants with Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs) [ Time Frame: From start of study drug administration until 30 days after the last dose of study drug ]

Measure Type	Primary
Measure Name	Number of Participants with Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)
Measure Description	AEs observed in participants with clinically significant ECG abnormalities were assessed.
Time Frame	From start of study drug administration until 30 days after the last dose of study drug
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Number of Participants with Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs) [units: Participants]	0	0	0

No statistical analysis provided for Number of Participants with Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)

4. Primary Outcome Measure: Number of Participants with Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: From start of study drug administration until 30 days after the last dose of study drug ]

Measure Type	Primary
Measure Name	Number of Participants with Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Measure 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 an adverse event. Treatment-emergent were events between first dose of study drug and 30 days after the last dose that were absent before treatment or that worsened relative to pretreatment state. Number of participants with grade 3 or higher treatment-emergent adverse events for laboratory abnormalities were reported as clinically relevant laboratory changes.
Time Frame	From start of study drug administration until 30 days after the last dose of study drug
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Number of Participants with Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) [units: Participants]
Hemoglobin decreased	3	0	1
Platelet count decreased	2	1	1
Neutrophil count decreased	2	0	0
Lymphocyte count decreased	1	0	0
White blood cell count decreased	1	0	0
Neutropenia	1	0	0
Febrile neutropenia	0	1	0
Blood albumin decreased	1	1	2
Blood magnesium decreased	1	2	0
Blood glucose increased	2	0	0
Blood magnesium increased	1	1	0
Blood phosphorus decreased	1	1	0
Aspartate aminotransferase increased	0	1	0
Blood alkaline phosphatase increased	0	1	0
Blood calcium increased	0	1	0
Blood glucose decreased	0	0	1
Blood potassium decreased	0	1	0
Blood sodium decreased	1	0	0
Blood sodium increased	0	1	0
Blood triglycerides increased	1	0	0

No statistical analysis provided for Number of Participants with Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

5. Primary Outcome Measure: Number of Participants With Objective Response Rate (ORR): Complete Response (CR) or Partial Response (PR) [ Time Frame: Up to 2 years of post-treatment follow-up ]

Measure Type	Primary
Measure Name	Number of Participants With Objective Response Rate (ORR): Complete Response (CR) or Partial Response (PR)
Measure Description	Objective response rate defined as the proportion of participants with confirmed complete response (CR) or confirmed partial response (PR) according to Response Evaluation Criteria.
Time Frame	Up to 2 years of post-treatment follow-up
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Number of Participants With Objective Response Rate (ORR): Complete Response (CR) or Partial Response (PR) [units: Participants]	0	0	0

No statistical analysis provided for Number of Participants With Objective Response Rate (ORR): Complete Response (CR) or Partial Response (PR)

6. Primary Outcome Measure: Best Overall Objective Tumor Response [ Time Frame: Up to 2 years of post-treatment follow-up ]

Measure Type	Primary
Measure Name	Best Overall Objective Tumor Response
Measure Description	Antitumor activity was assessed by best overall objective tumor response.
Time Frame	Up to 2 years of post-treatment follow-up
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Best Overall Objective Tumor Response [units: Participants]
Complete response (CR)	0	0	0
Partial Response (PR)	0	0	0
Stable Disease (SD)	2	3	2
Progressive Disease (PD)	1	0	3

No statistical analysis provided for Best Overall Objective Tumor Response

7. Primary Outcome Measure: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox [ Time Frame: Predose, 0.25 (During Infusion), 0.5 (End of Infusion), 1, 1.5, 2, 4, 8 and 12 hours postdose on Day 1 and 5; Predose and End of Infusion on Day 3 ]

Measure Type	Primary
Measure Name	Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox
Measure Description	The Tmax is the time to reach maximum observed plasma concentration of Moxetumomab Pasudotox.
Time Frame	Predose, 0.25 (During Infusion), 0.5 (End of Infusion), 1, 1.5, 2, 4, 8 and 12 hours postdose on Day 1 and 5; Predose and End of Infusion on Day 3
Safety Issue?	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox. Here ‘n’ signifies participants evaluable for specified categories, for each arm, respectively.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox [units: hour] Median (Full Range)
Cycle 1, Day 1 (n=3,3,5)	0.500 (0.500 to 0.500)	0.50 (0.500 to 0.500)	0.500 (0.00 to 0.500)
Cycle 1, Day 5 (n=3,3,5)	0.500 (0.500 to 0.500)	0.500 (0.300 to 0.500)	0.500 (0.300 to 1.00)
Cycle 2, Day 1 (n=2,2,2)	4.25 (0.500 to 8.00)	0.375 (0.250 to 0.500)	0.500 (0.500 to 0.500)
Cycle 2, Day 5 (n=2,2,2)	0.500 (NA to NA) ^[1]	0.800 (0.500 to 1.00)	0.500 (0.500 to 0.500)
Cycle 3, Day 1 (n=1,1,2)	0.500 (NA to NA) ^[2]	0.500 (NA to NA) ^[3]	0.500 (0.500 to 0.500)
Cycle 3, Day 5 (n=1,1,2)	0.500 (NA to NA) ^[4]	2.00 (NA to NA) ^[5]	0.500 (0.500 to 0.500)
Cycle 4, Day 1 (n=1,NA,NA)	0.500 (NA to NA) ^[6]	NA (NA to NA) ^[7]	NA (NA to NA) ^[8]
Cycle 4, Day 5 (n=1,NA,NA)	0.500 (NA to NA) ^[9]	NA (NA to NA) ^[10]	NA (NA to NA) ^[11]
Cycle 5, Day 1 (n=1,NA,NA)	0.500 (NA to NA) ^[12]	NA (NA to NA) ^[13]	NA (NA to NA) ^[14]
Cycle 5, Day 5 (n=1,NA,NA)	0.500 (NA to NA) ^[15]	NA (NA to NA) ^[16]	NA (NA to NA) ^[17]
Cycle 6, Day 1 (n=1,NA,NA)	0.500 (NA to NA) ^[18]	NA (NA to NA) ^[19]	NA (NA to NA) ^[20]
Cycle 6, Day 5 (n=1,NA,NA)	0.500 (NA to NA) ^[21]	NA (NA to NA) ^[22]	NA (NA to NA) ^[23]

[1]	Participant had no measurable Tmax concentration.
[2]	Median range was not evaluable since only 1 participant was evaluated.
[3]	Median range was not evaluable since only 1 participant was evaluated.
[4]	Median range was not evaluable since only 1 participant was evaluated.
[5]	Median range was not evaluable since only 1 participant was evaluated.
[6]	Median range was not evaluable since only 1 participant was evaluated.
[7]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[8]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[9]	Median range was not evaluable since only 1 participant was evaluated.
[10]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[11]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[12]	Median range was not evaluable since only 1 participant was evaluated.
[13]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[14]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[15]	Median range was not evaluable since only 1 participant was evaluated.
[16]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[17]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[18]	Median range was not evaluable since only 1 participant was evaluated.
[19]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[20]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[21]	Median range was not evaluable since only 1 participant was evaluated.
[22]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[23]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.

No statistical analysis provided for Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox

8. Primary Outcome Measure: Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox [ Time Frame: Predose, 0.25 (During Infusion), 0.5 (End of Infusion), 1, 1.5, 2, 4, 8 and 12 hours postdose on Day 1 and 5; Predose and End of Infusion on Day 3 ]

Measure Type	Primary
Measure Name	Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox
Measure Description	The Cmax is the maximum observed plasma concentration of Moxetumomab Pasudotox.
Time Frame	Predose, 0.25 (During Infusion), 0.5 (End of Infusion), 1, 1.5, 2, 4, 8 and 12 hours postdose on Day 1 and 5; Predose and End of Infusion on Day 3
Safety Issue?	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox. Here ‘n’ signifies participants evaluable for specified categories, for each arm, respectively.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox [units: nanogram per milliliter] Median (Full Range)
Cycle 1, Day 1 (n=3,3,5)	97 (59.1 to 107)	57.7 (46.5 to 145)	180 (63.7 to 351)
Cycle 1, Day 5 (n=3,3,5)	80.9 (56.4 to 122)	117 (44.7 to 166)	206 (108 to 353)
Cycle 2, Day 1 (n=2,2,2)	94.0 (66.0 to 122)	723 (146 to 1300)	156 (134 to 178)
Cycle 2, Day 5 (n=2,2,2)	50.5 (0.00 to 101)	80.8 (72.3 to 89.2)	211 (171 to 250)
Cycle 3, Day 1 (n=1,1,2)	107 (NA to NA) ^[1]	154 (NA to NA) ^[2]	160 (63.9 to 257)
Cycle 3, Day 5 (n=1,1,2)	93.4 (NA to NA) ^[3]	200 (NA to NA) ^[4]	266 (156 to 376)
Cycle 4, Day 1 (n=1,NA,NA)	84.9 (NA to NA) ^[5]	NA (NA to NA) ^[6]	NA (NA to NA) ^[7]
Cycle 4, Day 5 (n=1,NA,NA)	86.2 (NA to NA) ^[8]	NA (NA to NA) ^[9]	NA (NA to NA) ^[10]
Cycle 5, Day 1 (n=1,NA,NA)	95.0 (NA to NA) ^[11]	NA (NA to NA) ^[12]	NA (NA to NA) ^[13]
Cycle 5, Day 5 (n=1,NA,NA)	86.9 (NA to NA) ^[14]	NA (NA to NA) ^[15]	NA (NA to NA) ^[16]
Cycle 6, Day 1 (n=1,NA,NA)	93.2 (NA to NA) ^[17]	NA (NA to NA) ^[18]	NA (NA to NA) ^[19]
Cycle 6, Day 5 (n=1,NA,NA)	67.0 (NA to NA) ^[20]	NA (NA to NA) ^[21]	NA (NA to NA) ^[22]

[1]	Median range was not evaluable since only 1 participant was evaluated.
[2]	Median range was not evaluable since only 1 participant was evaluated.
[3]	Median range was not evaluable since only 1 participant was evaluated.
[4]	Median range was not evaluable since only 1 participant was evaluated.
[5]	Median range was not evaluable since only 1 participant was evaluated.
[6]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[7]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[8]	Median range was not evaluable since only 1 participant was evaluated.
[9]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[10]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[11]	Median range was not evaluable since only 1 participant was evaluated.
[12]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[13]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[14]	Median range was not evaluable since only 1 participant was evaluated.
[15]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[16]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[17]	Median range was not evaluable since only 1 participant was evaluated.
[18]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[19]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[20]	Median range was not evaluable since only 1 participant was evaluated.
[21]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
[22]	Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.

No statistical analysis provided for Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox

9. Secondary Outcome Measure: Number of Participants With Positive Neutralizing Antibodies [ Time Frame: Up to end of treatment (4-6 weeks after the last dose) ]

Measure Type	Secondary
Measure Name	Number of Participants With Positive Neutralizing Antibodies
Measure Description	Participants tested for immunogenicity to moxetumomab pasudotox prior to enrollment, before each cycle and at end of study. The neutralization assay measures the capacity of participant's plasma (antibodies) to inhibit the binding of moxetumomab pasudotox to its target, cluster of differentiation 22 (CD22), coated onto enzyme linked immunosorbent assay (ELISA) plates. It was used as a direct surrogate for biological activity based on the mechanism of action of this drug. Significant level of neutralizing antibody activity defined as the capacity of test plasma to inhibit greater than (>)50 percentage (%) of the binding of CAT-8015 to CD22 using an ELISA-based method.
Time Frame	Up to end of treatment (4-6 weeks after the last dose)
Safety Issue?	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Number of Participants With Positive Neutralizing Antibodies [units: Participants]	0	0	1

No statistical analysis provided for Number of Participants With Positive Neutralizing Antibodies

10. Secondary Outcome Measure: Percentage Change From Baseline in Cluster of Differentiation 22 (CD22) Expression [ Time Frame: End of treatment (4-6 weeks after the last dose) ]

Measure Type	Secondary
Measure Name	Percentage Change From Baseline in Cluster of Differentiation 22 (CD22) Expression
Measure Description	Participants demonstrated CD22 expression on malignant cells at Screening and CD22 is a regulatory molecule that prevents the over activation of the immune system and the development of autoimmune diseases.
Time Frame	End of treatment (4-6 weeks after the last dose)
Safety Issue?	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population included all participants who received any treatment of moxetumomab pasudotox.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Measured Values

	CAT-8015 5 microgram per kilogram (mcg/kg)	CAT-8015 10 microgram per kilogram (mcg/kg)	CAT-8015 20 microgram per kilogram (mcg/kg)
Number of Participants Analyzed [units:participants]	3	3	5
Percentage Change From Baseline in Cluster of Differentiation 22 (CD22) Expression [units: Percentage] Mean (Standard Deviation)
CD22-PE [OF CD5+/CD19+]	-33.65 (27.79)	-10.25 (16.05)	-8.85 (16.19)
(MESF CD22-PE [of CD5+/CD19+])	1139 (1514.6)	-3076 (5982.1)	-342.8 (1816.8)

No statistical analysis provided for Percentage Change From Baseline in Cluster of Differentiation 22 (CD22) Expression

Serious Adverse Events

Time Frame	From start of study drug administration until 30 days after the last dose of study drug
Additional Description	No text entered.

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Serious Adverse Events

CAT-8015 5 microgram per kilogram (mcg/kg)

CAT-8015 10 microgram per kilogram (mcg/kg)

CAT-8015 20 microgram per kilogram (mcg/kg)

Total, serious adverse events

# participants affected / at risk

1/3 (33.33%)

2/3 (66.67%)

3/5 (60.00%)

Blood and lymphatic system disorders

Febrile neutropenia¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Eye disorders

Optic ischaemic neuropathy¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Infections and infestations

Acute pulmonary histoplasmosis¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Infections and infestations

Infection¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

Infections and infestations

Neutropenic infection¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Infections and infestations

Pneumonia¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

1/5 (20.00%)

# events

Investigations

Blood calcium increased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Metabolism and nutrition disorders

Tumour lysis syndrome¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

Musculoskeletal and connective tissue disorders

Back pain¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

Respiratory, thoracic and mediastinal disorders

Obstructive airways disorder¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

†	Events were collected by systematic assessment
1	Term from vocabulary, MedDRA 13.1

Other Adverse Events

Time Frame	From start of study drug administration until 30 days after the last dose of study drug
Additional Description	No text entered.

Frequency Threshold

Threshold above which other adverse events are reported	1%

Reporting Groups

	Description
CAT-8015 5 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 10 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
CAT-8015 20 microgram per kilogram (mcg/kg)	Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.

Other Adverse Events

CAT-8015 5 microgram per kilogram (mcg/kg)

CAT-8015 10 microgram per kilogram (mcg/kg)

CAT-8015 20 microgram per kilogram (mcg/kg)

Total, other (not including serious) adverse events

# participants affected / at risk

3/3 (100.00%)

5/5 (100.00%)

Blood and lymphatic system disorders

Neutropenia¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Cardiac disorders

Sinus tachycardia¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Endocrine disorders

Hypothyroidism¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Eye disorders

Vision blurred¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Abdominal distension¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Abdominal pain¹,^†

# participants affected / at risk

2/3 (66.67%)

0/3 (0.00%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Diarrhoea¹,^†

# participants affected / at risk

1/3 (33.33%)

1/5 (20.00%)

# events

Gastrointestinal disorders

Dysphagia¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Gingival pain¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Nausea¹,^†

# participants affected / at risk

1/3 (33.33%)

2/3 (66.67%)

1/5 (20.00%)

# events

Gastrointestinal disorders

Oral pain¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Stomatitis¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Gastrointestinal disorders

Vomiting¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

General disorders and administration site conditions

Chills¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

General disorders and administration site conditions

Fatigue¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

1/5 (20.00%)

# events

General disorders and administration site conditions

Localised oedema¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

General disorders and administration site conditions

Oedema¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

1/5 (20.00%)

# events

General disorders and administration site conditions

Oedema peripheral¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

General disorders and administration site conditions

Pain¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

General disorders and administration site conditions

Pyrexia¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

1/5 (20.00%)

# events

Infections and infestations

Cystitis¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Infections and infestations

Histoplasmosis¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Infections and infestations

Rhinitis¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Injury, poisoning and procedural complications

Contusion¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Aspartate aminotransferase increased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Blood albumin decreased¹,^†

# participants affected / at risk

1/3 (33.33%)

2/5 (40.00%)

# events

Investigations

Blood alkaline phosphatase increased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Blood glucose decreased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

Investigations

Blood glucose increased¹,^†

# participants affected / at risk

2/3 (66.67%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Blood magnesium decreased¹,^†

# participants affected / at risk

1/3 (33.33%)

2/3 (66.67%)

0/5 (0.00%)

# events

Investigations

Blood magnesium increased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Blood phosphorus decreased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Blood potassium decreased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Blood sodium decreased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Blood sodium increased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Blood triglycerides increased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Blood uric acid increased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Haemoglobin decreased¹,^†

# participants affected / at risk

3/3 (100.00%)

0/3 (0.00%)

1/5 (20.00%)

# events

Investigations

Lymphocyte count decreased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Neutrophil count decreased¹,^†

# participants affected / at risk

2/3 (66.67%)

0/3 (0.00%)

0/5 (0.00%)

# events

Investigations

Platelet count decreased¹,^†

# participants affected / at risk

2/3 (66.67%)

1/3 (33.33%)

1/5 (20.00%)

# events

Investigations

Protein urine present¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

Urine output decreased¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Investigations

White blood cell count decreased¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Metabolism and nutrition disorders

Decreased appetite¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

Metabolism and nutrition disorders

Hyperuricaemia¹,^†

# participants affected / at risk

0/3 (0.00%)

2/5 (40.00%)

# events

Musculoskeletal and connective tissue disorders

Arthralgia¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Musculoskeletal and connective tissue disorders

Arthritis¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Musculoskeletal and connective tissue disorders

Musculoskeletal chest pain¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Musculoskeletal and connective tissue disorders

Musculoskeletal pain¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Musculoskeletal and connective tissue disorders

Pain in extremity¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

1/5 (20.00%)

# events

Nervous system disorders

Headache¹,^†

# participants affected / at risk

1/3 (33.33%)

0/5 (0.00%)

# events

Psychiatric disorders

Anxiety¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

Psychiatric disorders

Confusional state¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Psychiatric disorders

Depression¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Renal and urinary disorders

Haemorrhage urinary tract¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Renal and urinary disorders

Urinary incontinence¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Renal and urinary disorders

Urinary retention¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Respiratory, thoracic and mediastinal disorders

Bronchospasm¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Respiratory, thoracic and mediastinal disorders

Cough¹,^†

# participants affected / at risk

1/3 (33.33%)

1/5 (20.00%)

# events

Respiratory, thoracic and mediastinal disorders

Dyspnoea¹,^†

# participants affected / at risk

0/3 (0.00%)

2/3 (66.67%)

1/5 (20.00%)

# events

Respiratory, thoracic and mediastinal disorders

Lung infiltration¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Respiratory, thoracic and mediastinal disorders

Oropharyngeal pain¹,^†

# participants affected / at risk

0/3 (0.00%)

1/5 (20.00%)

# events

Respiratory, thoracic and mediastinal disorders

Pleural effusion¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Respiratory, thoracic and mediastinal disorders

Pulmonary congestion¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Skin and subcutaneous tissue disorders

Angioedema¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Skin and subcutaneous tissue disorders

Hyperhidrosis¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Skin and subcutaneous tissue disorders

Petechiae¹,^†

# participants affected / at risk

0/3 (0.00%)

1/3 (33.33%)

0/5 (0.00%)

# events

Skin and subcutaneous tissue disorders

Rash¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

Vascular disorders

Capillary leak syndrome¹,^†

# participants affected / at risk

1/3 (33.33%)

0/3 (0.00%)

0/5 (0.00%)

# events

†	Events were collected by systematic assessment
1	Term from vocabulary, MedDRA 13.1

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.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days . The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: MedImmune has 60 days to review results communications prior to public release and may delete information that compromises ongoing 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.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The study was terminated early due to unavailability of investigational product.

Results Point of Contact

Name/Title:	Mark C Lanasa
Organization:	MedImmune, LLC.
Phone	301-398-0000
E-mail:	[email protected]

Documents available

Trial Results Summaries
Available here

Contacts

Contact

AstraZeneca Clinical Study Information Center

1-877-240-9479

information.center@astrazeneca.com

Investigators

Principal Investigator

Mark C Lanasa

MedImmune Inc.

Sponsors

Collaborators

Inclusion Criteria

Exclusion Criteria

Research Site

Research Site

Research Site

Trial Results Summaries