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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01824693
Registration number
NCT01824693
Ethics application status
Date submitted
2/04/2013
Date registered
5/04/2013
Date last updated
5/12/2018
Titles & IDs
Public title
Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
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Scientific title
A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) for Children With Juvenile Myelomonocytic Leukemia (JMML)
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Secondary ID [1]
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NCI-2013-00738
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Secondary ID [2]
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ASCT1221
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Juvenile Myelomonocytic Leukemia
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Condition category
Condition code
Cancer
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Leukaemia - Acute leukaemia
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Cancer
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Leukaemia - Chronic leukaemia
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Cancer
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Children's - Leukaemia & Lymphoma
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Surgery - Allogeneic Hematopoietic Stem Cell Transplantation
Treatment: Drugs - Busulfan
Treatment: Drugs - Cyclophosphamide
Treatment: Drugs - Fludarabine Phosphate
Other interventions - Laboratory Biomarker Analysis
Treatment: Drugs - Melphalan
Treatment: Drugs - Mycophenolate Mofetil
Other interventions - Pharmacological Study
Treatment: Drugs - Tacrolimus
Experimental: Arm I (busulfan, cyclophosphamide, melphalan) - CONDITIONING REGIMEN: Patients receive busulfan IV QD, every 12 hours, or every 6 hours over 2-3 hours on days -8 to -5, cyclophosphamide IV QD over 60 minutes on days -4 and -3, and melphalan IV over 15-30 minutes on day -1.
TRANSPLANT: Patients undergo allogeneic HCT no sooner than 24 hours after the last dose of chemotherapy.
Patients receive tacrolimus IV or PO on days -1 to 98 (related donor) or 180 (unrelated donor) and mycophenolate mofetil IV over 2 hours or PO every 8 hours on days 1-30 (related donor) or 45 (unrelated donor).
Experimental: Arm II (busulfan, fludarabine phosphate) - CONDITIONING REGIMEN: Patients receive busulfan as in Arm I and fludarabine phosphate IV over 1 hour on days -5 to -2.
TRANSPLANT: Patients undergo allogeneic HCT as in Arm I.
Patients receive tacrolimus IV or PO on days -1 to 98 (related donor) or 180 (unrelated donor) and mycophenolate mofetil IV over 2 hours or PO every 8 hours on days 1-30 (related donor) or 45 (unrelated donor).
Treatment: Surgery: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo allogeneic HCT
Treatment: Drugs: Busulfan
Given IV
Treatment: Drugs: Cyclophosphamide
Given IV
Treatment: Drugs: Fludarabine Phosphate
Given IV
Other interventions: Laboratory Biomarker Analysis
Correlative studies
Treatment: Drugs: Melphalan
Given IV
Treatment: Drugs: Mycophenolate Mofetil
Given IV or PO
Other interventions: Pharmacological Study
Correlative studies
Treatment: Drugs: Tacrolimus
Given IV or PO
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Intervention code [1]
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Treatment: Surgery
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Intervention code [2]
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Treatment: Drugs
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percent Probability of Event-free Survival (EFS)
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Assessment method [1]
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Probability of Event-free Survival (EFS) for Patients after 18 months. An event is either treatment related mortality (TRM), primary or secondary graft failure, or relapse/non-response (as defined in protocol section 10). Time to event is time from transplant with patients who die between the start of the conditioning regimen and transplant given a time to event of zero.
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Timepoint [1]
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From transplant up to 18 months
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Primary outcome [2]
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Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100
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Assessment method [2]
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The number of patients who experience TRM on day 100. Treatment-Related Mortality (TRM) an event defined as a death prior to relapse or non-response. Time to TRM is defined as time from transplants to TRM. Patients who die between the start of the conditioning regimen and transplant will be considered a TRM with time to TRM of zero.
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Timepoint [2]
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From transplant up to 100 days
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Secondary outcome [1]
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Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
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Assessment method [1]
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Primary Graft failure is defined as the failure to achieve an ANC \>= 500/uL after 42 days, determined by 3 consecutive measurements on different days; OR \< 5% donor cells in blood or bone marrow by day +42 (as demonstrated by a chimerism assay), without evidence of Juvenile Myelomonocytic Leukemia (JMML).
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Timepoint [1]
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Day 0 - day 540 (18 months) following completion of stem cell transplant
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Secondary outcome [2]
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Percent Probability of 18 Months-relapse Event Between Arms
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Assessment method [2]
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Probability of patients relapsing at 18 months. A relapse event is defined in protocol section 10.2.3. Time to relapse/non-response is defined as time from transplant to when all criteria of section 10.2.3 are met.
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Timepoint [2]
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From transplant up to 18 months
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Eligibility
Key inclusion criteria
* Patients must have a strong clinical suspicion of JMML, based on a modified category 1 of the revised diagnostic criteria; specifically, eligible patients must have all of the following:
* Splenomegaly
* Absolute monocyte count (AMC) > 1000/uL
* Blasts in peripheral blood (PB)/bone marrow (BM) < 20%
* For the 7-10% of patients without splenomegaly, the diagnostic entry criteria must include all other features described above and at least 2 of the following criteria:
* Circulating myeloid precursors
* White blood cell (WBC) > 10,000/uL
* Increased fetal hemoglobin (HgbF) for age
* Sargramostim (GM-CSF) hypersensitivity OR, patients must have been previously diagnosed with JMML
* Patients must be previously untreated with HCT
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
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Minimum age
3
Months
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Maximum age
18
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Patients with a known germline mutation of PTPN11 (Noonan?s Syndrome) are not eligible
* Patients with a known history of NF1 (Neurofibromatosis Type 1) and either
* A history of a tumor of the central nervous system (astrocytoma or optic glioma), or
* A malignant peripheral nerve sheath tumor with a complete remission of < 1 year are not eligible
* Human immunodeficiency virus (HIV) positive patients are not eligible
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/06/2013
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
31/12/2017
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Sample size
Target
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Accrual to date
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Final
30
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Recruitment in Australia
Recruitment state(s)
WA
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Recruitment hospital [1]
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Princess Margaret Hospital for Children - Perth
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Recruitment postcode(s) [1]
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6008 - Perth
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Recruitment outside Australia
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United States of America
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Alabama
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New Zealand
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Auckland
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Funding & Sponsors
Primary sponsor type
Other
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Name
Children's Oncology Group
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Address
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Government body
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Name [1]
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National Cancer Institute (NCI)
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Ethics approval
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Summary
Brief summary
This randomized phase II trial studies how well giving busulfan, cyclophosphamide, and melphalan or busulfan and fludarabine phosphate before donor hematopoietic cell transplant works in treating younger patients with juvenile myelomonocytic leukemia. Giving chemotherapy before a donor hematopoietic transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether giving busulfan, cyclophosphamide, and melphalan or busulfan and fludarabine phosphate before a donor stem cell transplant is more effective in treating juvenile myelomonocytic leukemia.
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Trial website
https://clinicaltrials.gov/study/NCT01824693
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Christopher Dvorak
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Address
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Children's Oncology Group
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
Study Protocol and Statistical Analysis Plan
https://cdn.clinicaltrials.gov/large-docs/93/NCT01824693/Prot_SAP_000.pdf
Statistical analysis plan
Study Protocol and Statistical Analysis Plan
https://cdn.clinicaltrials.gov/large-docs/93/NCT01824693/Prot_SAP_000.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT01824693
Download to PDF