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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02227108




Registration number
NCT02227108
Ethics application status
Date submitted
21/08/2014
Date registered
27/08/2014
Date last updated
6/04/2017

Titles & IDs
Public title
Study in Pediatrics With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma
Scientific title
A Phase 2, Multicenter, Single-arm Study of Moxetumomab Pasudotox in Pediatric Subjects With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin
Secondary ID [1] 0 0
CD-ON-CAT-8015-1036
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
B-Cell Pediatric ALL 0 0
Condition category
Condition code
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Moxetumomab Pasudotox

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


Treatment: Drugs: Moxetumomab Pasudotox
Participants received 6 doses of moxetumomab pasudotox 40 microgram per kilogram (mcg/kg) intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Composite Complete Response (CRc)
Timepoint [1] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [1] 0 0
Percentage of Participants With Minimal Residual Disease (MRD)-Negative CRc Rate
Timepoint [1] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [2] 0 0
Overall Response Rate (ORR)
Timepoint [2] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [3] 0 0
Time to Overall Response
Timepoint [3] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [4] 0 0
Best Overall Response (BOR)
Timepoint [4] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [5] 0 0
Bone Marrow Blast Percentage Change
Timepoint [5] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [6] 0 0
Percentage of Participants Who Became Eligible to Receive an Stem Cell Transplant (SCT) After Treatment With Moxetumomab Pasudotox
Timepoint [6] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [7] 0 0
Time to Transplant to Receive an Stem Cell Transplant (SCT) After Treatment With Moxetumomab Pasudotox
Timepoint [7] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [8] 0 0
Percentage of Participants Who Were Neutropenic at Study Entry and Who Experienced Hematologic Activity (HA)
Timepoint [8] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [9] 0 0
Duration of Complete Response (DOCR)
Timepoint [9] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [10] 0 0
Duration of Overall Response (DOR)
Timepoint [10] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [11] 0 0
Progression-Free Survival (PFS)
Timepoint [11] 0 0
Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
Secondary outcome [12] 0 0
Overall Survival (OS)
Timepoint [12] 0 0
Baseline to end of study or last contact date, up to 1 year
Secondary outcome [13] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
Timepoint [13] 0 0
Baseline up to 30 days after the last dose of study drug, up to 1 year
Secondary outcome [14] 0 0
Number of Participants With Abnormal Clinical Laboratory Parameters Reported as Treatment-Emergent Adverse Events (TEAE)
Timepoint [14] 0 0
Baseline up to 30 days after the last dose of study drug, up to 1 year
Secondary outcome [15] 0 0
Number of Participants With Clinically Significant Electrocardiogram (ECG) Abnormalities
Timepoint [15] 0 0
Baseline up to 30 days after the last dose of study drug, up to 1 year
Secondary outcome [16] 0 0
Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)
Timepoint [16] 0 0
Baseline up to 30 days after the last dose of study drug, up to 1 year
Secondary outcome [17] 0 0
Number of Participants With Positive Anti-drug Antibody (ADA) and Neutralizing Antibodies (NAb)
Timepoint [17] 0 0
Prior to the Start of Each Cycle for Cycles 1, 2, 3, and Subsequent Odd-Numbered Cycles, End of Treatment, and 30 Day Follow-up Visit, up to 1 year
Secondary outcome [18] 0 0
Area Under the Plasma Concentration Time Curve From Time 0 to Infinity (AUC0-inf) After the First Dose of Cycle 1
Timepoint [18] 0 0
Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion of Day 1 of Cycle 1
Secondary outcome [19] 0 0
Area Under the Concentration Versus Time Curve From Time Zero to Last Quantifiable Concentration [AUC0-last] After the First Dose of Cycle 1
Timepoint [19] 0 0
Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
Secondary outcome [20] 0 0
Maximum Observed Drug Concentration in Plasma (Cmax) After the First Dose of Cycle 1
Timepoint [20] 0 0
Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
Secondary outcome [21] 0 0
Time to Reach Maximum Drug Concentration in Plasma (Tmax) After the First Dose of Cycle 1
Timepoint [21] 0 0
Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
Secondary outcome [22] 0 0
Terminal Phase Elimination Half Life (t1/2) After the First Dose of Cycle 1
Timepoint [22] 0 0
Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1
Secondary outcome [23] 0 0
Systemic Clearance (CL) After the First Dose of Cycle 1
Timepoint [23] 0 0
Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1

Eligibility
Key inclusion criteria
Inclusion Criteria -

1. Between the ages of greater or equal to (=) 6 months and less than (<) 18 years of age
2. Must have histologically proven B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma with marrow involvement
3. All participants (both ALL and participants with lymphoblastic lymphoma) must have M2 or M3 bone marrow classification
4. Disease status: a) Participants must have relapsed or refractory disease b) In the event of relapse after prior allogeneic hematopoietic stem cell transplant (HSCT), participants must be at least 3 months post-transplant and have no evidence of active graft-vs-host disease, and must have been off immunosuppression for at least 4 weeks, c) Must have resolution of the acute toxic effects to less than or equal to (=) Grade 2 from prior chemotherapy before entry, in the opinion of the investigator
5. Participants with the following central nervous system (CNS) 1 or 2 status are eligible only in the absence of neurologic symptoms
6. Female participants of childbearing potential and post-pubertal male participants must use an approved method of contraception for the study.
Minimum age
6 Months
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

1. Concurrent enrollment in another clinical study for cancer treatment, unless the subject is in the follow-up period from a previous study.
2. Isolated testicular or CNS ALL
3. Participants with mixed-lineage leukemia (MLL) gene rearrangement
4. Inadequate Hepatic function
5. Inadequate Renal function
6. Radiologically-detected CNS lymphoma
7. Participants with clear laboratory or clinical evidence of disseminated intravascular coagulation (DIC)
8. Hyperleukocytosis or rapidly progressive disease that would compromise ability to complete study therapy
9. QT interval corrected using Fridericia's formula (QTcF) greater than or equal to a Grade 2, confirmed by 2 additional seperate electrocardiographs (ECG's) within 28 days prior to starting study drug. The initial screening ECG need not be repeated for confirmation if the QTcF interval is <481 milliseconds.
10. Pregnant or breast-feeding females
11. Prior treatment with CAT-3888 (BL22), moxetumomab pasudotox, or any pseudomonas-exotoxin-containing compound
12. Prior treatment with any anticancer biologic therapy within 2 weeks prior to starting study drug, including but not limited to therapeutic monoclonal antibodies or antibody-drug conjugates
13. Systemic chemotherapy = 2 weeks (6 weeks for nitrosoureas) and radiation therapy = 3 weeks prior to starting study drug
14. Clinically significant ophthalmologic findings (evidence of retinal damage or injury) during the screening
15. Presence of a second invasive malignancy
16. Uncontrolled pulmonary infection, presence of pulmonary edema
17. Serum albumin < 2 gram per deciliter (g/dL). Albumin infusions for correction of hypoalbuminemia are allowed, but cannot have administered within 7 days prior to start of study drug
18. Radioimmunotherapy within 2 years prior to study start of study drug
19. Participants with prior history of thrombotic microangiopathy or hemolytic uremic syndrome (HUS)
20. T-cell ALL or T-cell lymphoblastic lymphoma
21. Participants currently receiving high-dose estrogen therapy defined as >0.625 milligram per day (mg/day) of an estrogen compound or within 2 weeks prior to starting study drug.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/08/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/11/2015
Sample size
Target
Accrual to date
Final
37
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Parkville
Recruitment hospital [2] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
France
State/province [12] 0 0
Lyon
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
France
State/province [14] 0 0
Vandoeuvre les Nancy Cedex
Country [15] 0 0
Italy
State/province [15] 0 0
Rome
Country [16] 0 0
Netherlands
State/province [16] 0 0
Rotterdam
Country [17] 0 0
Spain
State/province [17] 0 0
Barcelona
Country [18] 0 0
Spain
State/province [18] 0 0
Madrid
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Bristol

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
MedImmune LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medimmune Inc. Medimmune Inc.
Address 0 0
MedImmune LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02227108