Acute Lymphoblastic LeukemiaPhase 1NCT00659425

CAT-8015 in Children, Adolescents and Young Adults With Acute Lymphoblastic Leukemia or Non-Hodgkin's Lymphoma

A Phase 1 study testing CAT-8015 (Moxetumomab Pasudotox) in people with Acute Lymphoblastic Leukemia and Non-Hodgkin's Lymphoma, run by MedImmune LLC at 5 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

A dose-escalation study to estimate maximum cummulative dose (MTCD) of CAT-8015 that can be safely administered to a participant. Read more about this study

Who is it for?: Ages 6 months to 25 years
Conditions the study is for: Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma
What is being tested?: CAT-8015 (Moxetumomab Pasudotox)
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 57 participants
Where: 5 sitesin 2 countries
Study dates: Sep 2008to Dec 2014
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 6 months to 25 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Histologically confirmed diagnosis of acute lymphoblastic leukemia (ALL) or non-Hodgkin's lymphoma (NHL) including lymphoblastic lymphoma, Burkitt's lymphoma, and large cell lymphoma; - Measurable or evaluable disease. - Evidence of CD22-positive malignancy by one of the following criteria: - greater than or equal to (>=) 30 % of malignant cells from a disease site cluster of differentiation 22+ (CD22+) by fluorescence-activated cell sorter (FACS) analysis or; ≥ 15 % of malignant cells from a disease site CD22+ by immunohistochemistry (IHC). Stage of disease: - Participants must have relapsed or refractory disease and have received at least one standard chemotherapy and one salvage regimen or allogeneic stem cell transplant; - Relapse after prior autologous or allogeneic HSCT is allowed. In the event of relapse after prior allogeneic HSCT, the participant must be at least 100 days post-transplant and have no evidence of ongoing active graft-vs-host disease; - Recovered from the acute toxic effects of all prior therapy before entry. Performance status: - Participants greater than or equal to (>=) 12 years of age: Eastern Cooperative Oncology Group (ECOG) score of 0, 1, 2, or 3; - Participants < 12 years of age: Lansky scale >= 50%; - Participants who are unable to walk because of paralysis, but who are up in a wheel chair will be considered ambulatory for the purpose of calculating the performance score. Participants with the following central nervous system (CNS) status, are eligible only in the absence of neurologic symptoms suggestive of CNS leukemia, such as cranial nerve palsy. - Female and male participants with childbearing potential and their sexual partners must agree to use an approved method of contraception during the study.

Who cannot join

Participants meeting any of the following criteria are not eligible for participation in the study: - Isolated testicular or CNS ALL; Hepatic function: - Inadequate liver function defined as total bilirubin > 2 × upper limit of normal (ULN) (except in the case of participants with documented Gilbert's disease > 5 × ULN) or transaminases (ALT and aspartate aminotransferase [AST]) > 5 × ULN based on age- and laboratory-specific normal ranges; Renal function: - With greater than age-adjusted normal serum creatinine (see Table below) and a creatinine clearance > 60 millilitre per minute mL/min/1.73 m2. - Age(Years)- Maximum Serum Creatinine (mg/dl)[≤5,0.8] [5 < age less than or equal to 10,1.0] [10 < age less than or equal to 15,1.2 [> 15, 1.5] Hematologic function: - For non-leukemic subjects only, absolute neutrophil count (ANC) < 1000/cmm, or platelet count < 50,000/cmm, if these cytopenias are not judged by the investigator to be due to underlying disease (ie potentially reversible with anti-neoplastic therapy); - Participants with CNS 3 disease; - Hyperleukocytosis (≥ 50,000 blasts/µL) or rapidly progressive disease (PD) that in the estimation of the investigator and sponsor would compromise ability to complete study therapy; - Prior treatment with CAT-3888 (BL22) or any pseudomonas-exotoxin-containing compound; - HIV positive serology (due to increased risk of severe infection and unknown interaction of CAT-8015 with antiretroviral drugs); - Active hepatitis B or C infection.

Trial locations and contacts

5 sites in 2 countries.

Country

City, postcode or hospital

5 sites in 2 countries

United States4 sites

Research Site
Bethesda, Maryland
Research Site
Boston, Massachusetts
Research Site
Los Angeles, California
Research Site
Memphis, Tennessee

Other trials in United States

Canada1 site

Research Site
Toronto, Ontario

Other trials in Canada

About this study

Official title: A Phase 1, Multicenter, Dose Escalation Study of CAT-8015 in Children, Adolescents and Young Adults With Refractory CD22+ Acute Lymphoblastic Leukemia (ALL) or Non-Hodgkin Lymphoma (NHL)

A dose-escalation study to estimate maximum cummulative dose (MTCD) of CAT-8015 that can be safely administered to a participant.

Read the detailed description

A Phase 1, Multicenter, Dose Escalation Study of CAT-8015 in Children, Adolescents and Young Adults with Refractory CD22+ Acute Lymphoblastic Leukemia (ALL) or Non-Hodgkin Lymphoma (NHL) to estimate the maximum tolerated cummulative dose (MTCD), defined as the highest dose and number of doses that can be safely administered to a participant, and to establish a safe dose, based on the MTCD, for subsequent clinical testing.

Conditions: Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma
Being tested: CAT-8015 (Moxetumomab Pasudotox)
Keywords: Moxetumomab pasudotox
Medical subject headings: Precursor Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Non-Hodgkin

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 40 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 32 mcg/kg moxetumomab pasudotox (CAT-8015) of process 3 material every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
CAT-8015 (Moxetumomab Pasudotox)Drug
Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) continuous every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle.

Study groups in NCT00659425
Study group	Role	What they receive
5 microgram per kilogram (mcg/kg)	Experimental	Participants received intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
10 microgram per kilogram (mcg/kg)	Experimental	Participants received intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
20 microgram per kilogram (mcg/kg): Schema A	Experimental	Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
20 microgram per kilogram (mcg/kg): Schema B	Experimental	Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
30 microgram per kilogram (mcg/kg): Schema A	Experimental	Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
30 microgram per kilogram (mcg/kg): Schema B	Experimental	Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
40 microgram per kilogram (mcg/kg): Schema B	Experimental	Participants received intravenous infusion of 40 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
32 microgram per kilogram (mcg/kg): Schema C	Experimental	Participants received intravenous infusion of 32 mcg/kg moxetumomab pasudotox (CAT-8015) of process 3 material every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
50 microgram per kilogram (mcg/kg): Schema B	Experimental	Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)
50 microgram per kilogram (mcg/kg): Schema C	Experimental	Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) continuous every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle. Receives: Drug: CAT-8015 (Moxetumomab Pasudotox)

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Dose Limiting Toxicities (DLTs)
Adverse events that were suspected of a relationship to moxetumomab pasudotox and were greater than or equal to (>=) Grade 3 in severity were considered DLTs with the following additional criteria or exceptions: Participants with hematologic abnormalities of any grade, Grade 2 allergic reactions of bronchospasm or urticaria, or any Grade ≥ 3 allergic reaction, in the presence of premedication.
Measured: Day 1 up to 21 days of Cycle 1 (each cycle duration was of 21 days)
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was any untoward medical occurrence attributed to study drug in a participant who received investigational product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent were events between administration of investigational product and 30 days after the last dose of study drug that were absent before treatment or that worsened relative to pre-treatment state.
Measured: From start of study drug administration until 30 days after the last dose of study drug
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Vital signs included parameters as heart rate, blood pressure, temperature, weight, pulse oximetry and respiratory rate. 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.
Measured: From start of study drug administration until 30 days after the last dose of study drug
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
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 (5% cut off) for laboratory abnormalities were reported as clinically relevant laboratory changes.
Measured: From start of study drug administration up to 30 days after the last dose of study drug
Treatment-Emergent Adverse Events (TEAEs) Related to Chemistry Abnormalities Occurring in Greater Than (>) 5 Percent of Participants
An abnormal chemistry 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.
Measured: From start of study drug administration up to 30 days after the last dose of study drug
Number of Participants With Abnormalities in Ophthalmologic Examination at End of Treatment That Were Not Present at Baseline
Ophthalmologic examination included evaluation of retinal, corneal and lens abnormalities at baseline and end of treatment that were not present at screening. Participants who experienced abnormalitities during ophthalmologic examination recorded and reported.
Measured: Baseline and end of treatment (up to 1 year after the last participants begins study drug treatment)
Number of Participants With Change From Baseline in Normal Sinus Rhythm Findings in ECG
Number of participants with abnormal ECG changes (compared with baseline) as assessed by study cardiologist
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Change From Baseline to End of Treatment in Clinical Findings in Electrocardiogram (ECG) QT, QTC Interval and Ventricular Rate
The 12-lead ECG data were summarized and evaluated for the following parameters: ,QT, QTC intervals and ventricular rate. Change from baseline in these parameters were reported.
Measured: Baseline and end of treatment (up to 1 year after the last participants begins study drug treatment)
Best Overall Tumor Response
Antitumor activity was assessed by best overall tumor response.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Objective Response Rate (ORR)
Objective response based on assessment of confirmed composite complete response (CRc) or partial response (PR) according to disease specific criteria [modified criteria for response in acute lymphoblastic leukemia (ALL)].
Measured: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
Percentage of Participants With Relapse of Disease
Relapse is defined as progressive disease (PD) following complete response (CR). Rate of relapse was only calculated for the subgroup of participants with complete response.
Measured: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
Time to Disease Response
Time to disease response was measured from the start of moxetumomab pasudotox administration to the first documentation of response (CR or PR) and was assessed in participants who achieved objective response.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Duration of Response (DR)
Duration of response was defined as the duration from the first documentation of objective response to the first documented disease progression.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment
Time to Disease Progression (TDP)
Time to disease progression was measured from the start of treatment with moxetumomab pasudotox until the documentation of disease progression. Number of progressions were reported here.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment
Progression-Free Survival (PFS)
Progression-free survival was measured from the start of treatment with moxetumomab pasudotox until the documentation of disease progression or death due to any cause, whichever occurs first. Number of progressions/deaths were reported here.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Overall Survival (OS)
Overall survival was determined as the time from the start of treatment with Moxetumomab Pasudotox until death. Number of deaths were reported here.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox
The Cmax is the maximum observed plasma concentration of Moxetumomab Pasudotox.
Measured: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
Area Under the Serum Concentration Time Curve From Time Zero to Infinity (AUC [0 to Infinity]) for Moxetumomab Pasudotox
The AUC (0 to infinity) is the area under the plasma concentration-time curve from time zero to infinity hours.
Measured: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
Systemic Clearance (CL) for Moxetumomab Pasudotox
The CL is a quantitative measure of the rate at which a drug substance is removed from the body. The total systemic clearance after intravenous dose was estimated by dividing the total administered dose by the plasma area under the plasma concentration-time curve from time zone to infinite time (AUC[0-infinity]).
Measured: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
Terminal Phase Elimination Half Life (t1/2) for Moxetumomab Pasudotox
Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.
Measured: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6

Secondary outcomes (3)

Number of Participants With Positive Anti-Drug Antibody (ADA) and Neutralizing Antibody
Participants tested for immunogenicity to CAT-8015 (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.
Measured: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
CD22 Expression Cells in Peripheral Blood by Best Response
Participants malignant cells (peripheral blood) was tested for cluster of differentiation 22 (CD22) expression by fluorescence-activated cell sorter (FACS) analysis.
Measured: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
Number of Participants With Potential Biomarkers of Predicting Capillary Leak Syndrome (CLS)
Potential biomarkers include orthostatic blood pressure, albumin levels, weight change, edema and hypoxia were evaluated.
Measured: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2008Dec 2014

First posted16 Apr 2008 (estimated)
Study startSep 2008
Primary completionDec 2014
Study completionDec 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 57 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00659425
Sponsor's protocol ID: CAT-8015-1004
Status last verified: Aug 2017
First submitted: 10 Apr 2008
Last updated: 2 Oct 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Wayne AS, Shah NN, Bhojwani D, Silverman LB, Whitlock JA, Stetler-Stevenson M, et al. Pediatric phase 1 trial of moxetumomab pasudotox: activity in chemotherapy refractory acute lymphoblastic leukemia (ALL) . http://cancerres.aacrjournals.org/content/74/19_Supplement/CT230.short
Wayne AS, Shah NN, Bhojwani D, Silverman LB, Whitlock JA, Stetler-Stevenson M, Sun W, Liang M, Yang J, Kreitman RJ, Lanasa MC, Pastan I. Phase 1 study of the anti-CD22 immunotoxin moxetumomab pasudotox for childhood acute lymphoblastic leukemia. Blood. 2017 Oct 5;130(14):1620-1627. doi: 10.1182/blood-2017-02-749101. Epub 2017 Aug 9.
PubMed 28983018

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 2 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1, Multicenter, Dose Escalation Study of CAT-8015 in Children, Adolescents and Young Adults With Refractory CD22+ Acute Lymphoblastic Leukemia (ALL) or Non-Hodgkin Lymphoma (NHL)
ClinicalTrials.gov ID: NCT00659425
Acronym / sponsor ID: CAT-8015-1004
Conditions: Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma
Keywords: Moxetumomab pasudotox
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 57 participants (actual)
Lead sponsor: MedImmune LLCIndustry (pharma, biotech, devices)
Collaborators: National Cancer Institute (NCI)
Study start: Sep 2008
Primary completion: Dec 2014 (actual)
Study completion: Dec 2014 (actual)
First posted: 16 Apr 2008 (estimated)
Results: Posted 2 Oct 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

A dose-escalation study to estimate maximum cummulative dose (MTCD) of CAT-8015 that can be safely administered to a participant.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 10

Arm	Type	Receives
5 microgram per kilogram (mcg/kg)	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
10 microgram per kilogram (mcg/kg)	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
20 microgram per kilogram (mcg/kg): Schema A	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
20 microgram per kilogram (mcg/kg): Schema B	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
30 microgram per kilogram (mcg/kg): Schema A	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
30 microgram per kilogram (mcg/kg): Schema B	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
40 microgram per kilogram (mcg/kg): Schema B	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 40 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
32 microgram per kilogram (mcg/kg): Schema C	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 32 mcg/kg moxetumomab pasudotox (CAT-8015) of process 3 material every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle.
50 microgram per kilogram (mcg/kg): Schema B	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.
50 microgram per kilogram (mcg/kg): Schema C	Experimental	Drug: CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) continuous every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle.

Who may qualify

Ages: 6 months to 25 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (1 listed)

Histologically confirmed diagnosis of acute lymphoblastic leukemia (ALL) or non-Hodgkin's lymphoma (NHL) including lymphoblastic lymphoma, Burkitt's lymphoma, and large cell lymphoma; - Measurable or evaluable disease. - Evidence of CD22-positive malignancy by one of the following criteria: - greater than or equal to (>=) 30 % of malignant cells from a disease site cluster of differentiation 22+ (CD22+) by fluorescence-activated cell sorter (FACS) analysis or; ≥ 15 % of malignant cells from a disease site CD22+ by immunohistochemistry (IHC). Stage of disease: - Participants must have relapsed or refractory disease and have received at least one standard chemotherapy and one salvage regimen or allogeneic stem cell transplant; - Relapse after prior autologous or allogeneic HSCT is allowed. In the event of relapse after prior allogeneic HSCT, the participant must be at least 100 days post-transplant and have no evidence of ongoing active graft-vs-host disease; - Recovered from the acute toxic effects of all prior therapy before entry. Performance status: - Participants greater than or equal to (>=) 12 years of age: Eastern Cooperative Oncology Group (ECOG) score of 0, 1, 2, or 3; - Participants < 12 years of age: Lansky scale >= 50%; - Participants who are unable to walk because of paralysis, but who are up in a wheel chair will be considered ambulatory for the purpose of calculating the performance score. Participants with the following central nervous system (CNS) status, are eligible only in the absence of neurologic symptoms suggestive of CNS leukemia, such as cranial nerve palsy. - Female and male participants with childbearing potential and their sexual partners must agree to use an approved method of contraception during the study.

Key exclusion criteria (1 listed)

Participants meeting any of the following criteria are not eligible for participation in the study: - Isolated testicular or CNS ALL; Hepatic function: - Inadequate liver function defined as total bilirubin > 2 × upper limit of normal (ULN) (except in the case of participants with documented Gilbert's disease > 5 × ULN) or transaminases (ALT and aspartate aminotransferase [AST]) > 5 × ULN based on age- and laboratory-specific normal ranges; Renal function: - With greater than age-adjusted normal serum creatinine (see Table below) and a creatinine clearance > 60 millilitre per minute mL/min/1.73 m2. - Age(Years)- Maximum Serum Creatinine (mg/dl)[≤5,0.8] [5 < age less than or equal to 10,1.0] [10 < age less than or equal to 15,1.2 [> 15, 1.5] Hematologic function: - For non-leukemic subjects only, absolute neutrophil count (ANC) < 1000/cmm, or platelet count < 50,000/cmm, if these cytopenias are not judged by the investigator to be due to underlying disease (ie potentially reversible with anti-neoplastic therapy); - Participants with CNS 3 disease; - Hyperleukocytosis (≥ 50,000 blasts/µL) or rapidly progressive disease (PD) that in the estimation of the investigator and sponsor would compromise ability to complete study therapy; - Prior treatment with CAT-3888 (BL22) or any pseudomonas-exotoxin-containing compound; - HIV positive serology (due to increased risk of severe infection and unknown interaction of CAT-8015 with antiretroviral drugs); - Active hepatitis B or C infection.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	5 microgram per kilogram (mcg/kg)
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	10 microgram per kilogram (mcg/kg)
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	20 microgram per kilogram (mcg/kg): Schema A
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	20 microgram per kilogram (mcg/kg): Schema B
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	30 microgram per kilogram (mcg/kg): Schema A
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 30 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	30 microgram per kilogram (mcg/kg): Schema B
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 40 mcg/kg moxetumomab pasudotox (CAT-8015) with concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	40 microgram per kilogram (mcg/kg): Schema B
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 32 mcg/kg moxetumomab pasudotox (CAT-8015) of process 3 material every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle.	Drug	32 microgram per kilogram (mcg/kg): Schema C
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) without concomitant corticosteroid administration every other day (QoD) in a 21-day cycle for a total of 6 doses per cycle.	Drug	50 microgram per kilogram (mcg/kg): Schema B
CAT-8015 (Moxetumomab Pasudotox)Participants received intravenous infusion of 50 mcg/kg moxetumomab pasudotox (CAT-8015) continuous every other day (QoD) in a 21-day cycle for a total of 10 doses per cycle.	Drug	50 microgram per kilogram (mcg/kg): Schema C

Primary outcome

Number of Participants With Dose Limiting Toxicities (DLTs)
Time frame: Day 1 up to 21 days of Cycle 1 (each cycle duration was of 21 days)
Adverse events that were suspected of a relationship to moxetumomab pasudotox and were greater than or equal to (>=) Grade 3 in severity were considered DLTs with the following additional criteria or exceptions: Participants with hematologic abnormalities of any grade, Grade 2 allergic reactions of bronchospasm or urticaria, or any Grade ≥ 3 allergic reaction, in the presence of premedication.
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
An adverse event (AE) was any untoward medical occurrence attributed to study drug in a participant who received investigational product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent were events between administration of investigational product and 30 days after the last dose of study drug that were absent before treatment or that worsened relative to pre-treatment state.
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
Vital signs included parameters as heart rate, blood pressure, temperature, weight, pulse oximetry and respiratory rate. 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.
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Time frame: From start of study drug administration up to 30 days after the last dose of study drug
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 (5% cut off) for laboratory abnormalities were reported as clinically relevant laboratory changes.
Treatment-Emergent Adverse Events (TEAEs) Related to Chemistry Abnormalities Occurring in Greater Than (>) 5 Percent of Participants
Time frame: From start of study drug administration up to 30 days after the last dose of study drug
An abnormal chemistry 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 Abnormalities in Ophthalmologic Examination at End of Treatment That Were Not Present at Baseline
Time frame: Baseline and end of treatment (up to 1 year after the last participants begins study drug treatment)
Ophthalmologic examination included evaluation of retinal, corneal and lens abnormalities at baseline and end of treatment that were not present at screening. Participants who experienced abnormalitities during ophthalmologic examination recorded and reported.
Number of Participants With Change From Baseline in Normal Sinus Rhythm Findings in ECG
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Number of participants with abnormal ECG changes (compared with baseline) as assessed by study cardiologist
Change From Baseline to End of Treatment in Clinical Findings in Electrocardiogram (ECG) QT, QTC Interval and Ventricular Rate
Time frame: Baseline and end of treatment (up to 1 year after the last participants begins study drug treatment)
The 12-lead ECG data were summarized and evaluated for the following parameters: ,QT, QTC intervals and ventricular rate. Change from baseline in these parameters were reported.
Best Overall Tumor Response
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Antitumor activity was assessed by best overall tumor response.
Objective Response Rate (ORR)
Time frame: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
Objective response based on assessment of confirmed composite complete response (CRc) or partial response (PR) according to disease specific criteria [modified criteria for response in acute lymphoblastic leukemia (ALL)].
Percentage of Participants With Relapse of Disease
Time frame: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
Relapse is defined as progressive disease (PD) following complete response (CR). Rate of relapse was only calculated for the subgroup of participants with complete response.
Time to Disease Response
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Time to disease response was measured from the start of moxetumomab pasudotox administration to the first documentation of response (CR or PR) and was assessed in participants who achieved objective response.
Duration of Response (DR)
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment
Duration of response was defined as the duration from the first documentation of objective response to the first documented disease progression.
Time to Disease Progression (TDP)
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment
Time to disease progression was measured from the start of treatment with moxetumomab pasudotox until the documentation of disease progression. Number of progressions were reported here.
Progression-Free Survival (PFS)
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Progression-free survival was measured from the start of treatment with moxetumomab pasudotox until the documentation of disease progression or death due to any cause, whichever occurs first. Number of progressions/deaths were reported here.
Overall Survival (OS)
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)
Overall survival was determined as the time from the start of treatment with Moxetumomab Pasudotox until death. Number of deaths were reported here.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox
Time frame: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
The Cmax is the maximum observed plasma concentration of Moxetumomab Pasudotox.
Area Under the Serum Concentration Time Curve From Time Zero to Infinity (AUC [0 to Infinity]) for Moxetumomab Pasudotox
Time frame: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
The AUC (0 to infinity) is the area under the plasma concentration-time curve from time zero to infinity hours.
Systemic Clearance (CL) for Moxetumomab Pasudotox
Time frame: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
The CL is a quantitative measure of the rate at which a drug substance is removed from the body. The total systemic clearance after intravenous dose was estimated by dividing the total administered dose by the plasma area under the plasma concentration-time curve from time zone to infinite time (AUC[0-infinity]).
Terminal Phase Elimination Half Life (t1/2) for Moxetumomab Pasudotox
Time frame: Cycles 1, 2 and every 4th cycle: pre-dose, end of infusion (EOI), 1, 1.5, 2.5, 4 and 8 hours post-dose of Dose 1; pre-dose and end of infusion after Dose 6
Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.

3 secondary outcomes

Number of Participants With Positive Anti-Drug Antibody (ADA) and Neutralizing Antibody
Time frame: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
CD22 Expression Cells in Peripheral Blood by Best Response
Time frame: Baseline until end of treatment (up to 1 year after the last participant begins study drug treatment)
Number of Participants With Potential Biomarkers of Predicting Capillary Leak Syndrome (CLS)
Time frame: Baseline and end of treatment (up to 1 year after the last participant begins study drug treatment)

Results and safety

Results posted to the registry on 2 Oct 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
5 Microgram Per Kilogram (mcg/kg)	1	0
10 Microgram Per Kilogram (mcg/kg)	1	0
20 Microgram Per Kilogram (mcg/kg): Schema A	1	0
20 Microgram Per Kilogram (mcg/kg): Schema B	4	0
30 Microgram Per Kilogram (mcg/kg): Schema A	4	0
30 Microgram Per Kilogram (mcg/kg): Schema B	5	1
40 Microgram Per Kilogram (mcg/kg): Schema B	8	0
32 Microgram Per Kilogram (mcg/kg): Schema C	11	0
50 Microgram Per Kilogram (mcg/kg): Schema B	6	0
50 Microgram Per Kilogram (mcg/kg): Schema C	14	1

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

Time frame: Day 1 up to 21 days of Cycle 1 (each cycle duration was of 21 days). Number. Unit: participants

Measure	10 Microgram Per Kilogram (mcg/kg) n = 1	20 Microgram Per Kilogram (mcg/kg): Schema A n = 1	20 Microgram Per Kilogram (mcg/kg): Schema B n = 3	30 Microgram Per Kilogram (mcg/kg): Schema A n = 4	30 Microgram Per Kilogram (mcg/kg): Schema B n = 3	40 Microgram Per Kilogram (mcg/kg): Schema B n = 7	32 Microgram Per Kilogram (mcg/kg): Schema C n = 7	50 Microgram Per Kilogram (mcg/kg): Schema B n = 3	50 Microgram Per Kilogram (mcg/kg): Schema C n = 11
Value	0	0	0	2	0	1	1	0	1

Population analysed: Evaluable Population for DLT included all participants who received any treatment of moxetumomab pasudotox (CAT-8015) and completed the DLT period without a DLT, or did not complete the DLT period due to a DLT. Here, "N" is number of participants evaluated for this outcome measure.

Primary outcome: 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. Number. Unit: Participants

Measure	5 Microgram Per Kilogram (mcg/kg) n = 1	10 Microgram Per Kilogram (mcg/kg) n = 1	20 Microgram Per Kilogram (mcg/kg): Schema A n = 1	20 Microgram Per Kilogram (mcg/kg): Schema B n = 4	30 Microgram Per Kilogram (mcg/kg): Schema A n = 4	30 Microgram Per Kilogram (mcg/kg): Schema B n = 5	40 Microgram Per Kilogram (mcg/kg): Schema B n = 8	32 Microgram Per Kilogram (mcg/kg): Schema C n = 11	50 Microgram Per Kilogram (mcg/kg): Schema B n = 6	50 Microgram Per Kilogram (mcg/kg): Schema C n = 14
TEAEs	1	1	1	4	4	5	8	11	6	14
TESAEs	1	1	1	4	3	2	5	7	4	9

Population analysed: Safety Population included all participants who received any treatment of study drug.

Primary outcome: 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. Number. Unit: Participants

Measure	5 Microgram Per Kilogram (mcg/kg) n = 1	20 Microgram Per Kilogram (mcg/kg): Schema B n = 4	30 Microgram Per Kilogram (mcg/kg): Schema A n = 4	30 Microgram Per Kilogram (mcg/kg): Schema B n = 5	40 Microgram Per Kilogram (mcg/kg): Schema B n = 8	32 Microgram Per Kilogram (mcg/kg): Schema C n = 11	50 Microgram Per Kilogram (mcg/kg): Schema B n = 6	50 Microgram Per Kilogram (mcg/kg): Schema C n = 14
Tachycardia	0	2	3	3	2	2	0	3
Arrhythmia	0	0	0	0	1	0	0	0
Bradycardia	0	0	1	1	0	1	0	1
Sinus bradycardia	0	0	0	0	0	1	0	0
Sinus tachycardia	1	0	1	0	0	0	0	1
Pyrexia	1	2	4	2	2	2	2	7
Dyspnoea	0	0	1	0	0	1	0	4

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

Time frame: From start of study drug administration up to 30 days after the last dose of study drug. Number. Unit: Participants

Measure	10 Microgram Per Kilogram (mcg/kg) n = 1	20 Microgram Per Kilogram (mcg/kg): Schema A n = 1	20 Microgram Per Kilogram (mcg/kg): Schema B n = 4	30 Microgram Per Kilogram (mcg/kg): Schema A n = 4	30 Microgram Per Kilogram (mcg/kg): Schema B n = 5	40 Microgram Per Kilogram (mcg/kg): Schema B n = 8	32 Microgram Per Kilogram (mcg/kg): Schema C n = 11	50 Microgram Per Kilogram (mcg/kg): Schema C n = 14
Febrile neutropenia	1	1	1	2	1	3	5	1
Anemia	1	1	0	0	0	0	0	3
Leukopenia	0	0	0	0	1	3	0	1
Lymphopenia	1	1	0	0	1	3	0	1
Neutropenia	1	0	0	0	1	0	0	1
Thrombocytopenia	1	1	0	0	1	3	1	1

Population analysed: Safety population included all participants who received any treatment of study drug.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: From start of study drug administration up to 30 days after the last dose of study drug. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
5 UG/KG SCHEMA A	1 of 1 (100%)	1 of 1 (100%)	Not reported
10 UG/KG SCHEMA A	1 of 1 (100%)	1 of 1 (100%)	Not reported
20 UG/KG SCHEMA A	1 of 1 (100%)	1 of 1 (100%)	Not reported
20 UG/KG SCHEMA B	4 of 4 (100%)	4 of 4 (100%)	Not reported
30 UG/KG SCHEMA A	3 of 4 (75%)	4 of 4 (100%)	Not reported
30 UG/KG SCHEMA B	2 of 5 (40%)	5 of 5 (100%)	Not reported
40 UG/KG SCHEMA B	5 of 8 (62.5%)	8 of 8 (100%)	Not reported
32 UG/KG SCHEMA C	7 of 11 (63.6%)	11 of 11 (100%)	Not reported
50 UG/KG SCHEMA B	4 of 6 (66.7%)	6 of 6 (100%)	Not reported
50 UG/KG SCHEMA C	9 of 14 (64.3%)	14 of 14 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publicationWayne AS, Shah NN, Bhojwani D, Silverman LB, Whitlock JA, Stetler-Stevenson M, et al. Pediatric phase 1 trial of moxetumomab pasudotox: activity in chemotherapy refractory acute lymphoblastic leukemia (ALL) . http://cancerres.aacrjournals.org/content/74/19_Supplement/CT230.short
Derived from this study: PubMed 28983018Wayne AS, Shah NN, Bhojwani D, Silverman LB, Whitlock JA, Stetler-Stevenson M, Sun W, Liang M, Yang J, Kreitman RJ, Lanasa MC, Pastan I. Phase 1 study of the anti-CD22 immunotoxin moxetumomab pasudotox for childhood acute lymphoblastic leukemia. Blood. 2017 Oct 5;130(14):1620-1627. doi: 10.1182/blood-2017-02-749101. Epub 2017 Aug 9.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Medimmune LLC Medimmune LLC study director

MedImmune LLC

5 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CAT-8015 (Moxetumomab Pasudotox) in people with Acute Lymphoblastic Leukemia and Non-Hodgkin's Lymphoma, run by MedImmune LLC at 5 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 2 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Dose Limiting Toxicities (DLTs) (time frame: Day 1 up to 21 days of Cycle 1 (each cycle duration was of 21 days)). The study lists 20 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00659425, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2017.