NCT01824693 · STUDY IN CONTEXT

Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

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.

All eligible randomized patients

→

primary · POSTED

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.

All eligible randomized patients

→

secondary · POSTED

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).

All Eligible randomized patients

→

secondary · POSTED

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.

All eligible randomized patients

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-juvenile-myelomonocytic-leukemia
nct id: NCT01824693
trial label: Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic
applicability: narrower
applicability note: Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: registry-condition-search
discovery note: Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
outcome Count: 4
harms Count: 3
qol Count: 0

outcomes

release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0000
source section id
NCT01824693-om-0000
source outcome index
0
endpoint
Percent Probability of Event-free Survival (EFS)
endpoint definition
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.
time origin
From transplant up to 18 months
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
All eligible randomized patients
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0001
source section id
NCT01824693-om-0001
source outcome index
1
endpoint
Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100
endpoint definition
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.
time origin
From transplant up to 100 days
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
All eligible randomized patients
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0002
source section id
NCT01824693-om-0002
source outcome index
2
endpoint
Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
endpoint definition
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).
time origin
Day 0 - day 540 (18 months) following completion of stem cell transplant
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
All Eligible randomized patients
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0003
source section id
NCT01824693-om-0003
source outcome index
3
endpoint
Percent Probability of 18 Months-relapse Event Between Arms
endpoint definition
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.
time origin
From transplant up to 18 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
All eligible randomized patients
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0000
arm key
OG000
arm label
Arm I (Busulfan, Cyclophosphamide, Melphalan)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Cyclophosphamide: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
6
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0000
arm key
OG001
arm label
Arm II (Busulfan, Fludarabine Phosphate)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
9
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0000#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0001
arm key
OG000
arm label
Arm I (Busulfan, Cyclophosphamide, Melphalan)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Cyclophosphamide: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
6
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0001
arm key
OG001
arm label
Arm II (Busulfan, Fludarabine Phosphate)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
9
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0001#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0002
arm key
OG000
arm label
Arm I (Busulfan, Cyclophosphamide, Melphalan)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Cyclophosphamide: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
6
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0002
arm key
OG001
arm label
Arm II (Busulfan, Fludarabine Phosphate)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
9
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0002#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0003
arm key
OG000
arm label
Arm I (Busulfan, Cyclophosphamide, Melphalan)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Cyclophosphamide: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
6
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
outcome key
om-0003
arm key
OG001
arm label
Arm II (Busulfan, Fludarabine Phosphate)
regimen
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). Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic HCT Busulfan: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Pharmacological Study: Correlative studies Tacrolimus: Given IV or PO
analyzed count
9
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01824693-om-0003#groups/OG001

harms

release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
harm key
EG000
source section id
NCT01824693-ae
event group key
EG000
event group label
Arm I (Busulfan, Cyclophosphamide, Melphalan)
serious affected
1
serious at risk
6
other affected
5
other at risk
6
frequency threshold
0
collection period
Up to 1 year after completion of transplant.
event definition
Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. 3 patients from ARM III (Non-Randomized) were excluded due to ineligibility.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
harm key
EG001
source section id
NCT01824693-ae
event group key
EG001
event group label
Arm II (Busulfan, Fludarabine Phosphate)
serious affected
1
serious at risk
9
other affected
7
other at risk
9
frequency threshold
0
collection period
Up to 1 year after completion of transplant.
event definition
Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. 3 patients from ARM III (Non-Randomized) were excluded due to ineligibility.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-juvenile-myelomonocytic-leukemia
nct id
NCT01824693
harm key
EG002
source section id
NCT01824693-ae
event group key
EG002
event group label
ARM III (Non-Randomized)
serious affected
0
serious at risk
12
other affected
0
other at risk
12
frequency threshold
0
collection period
Up to 1 year after completion of transplant.
event definition
Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. 3 patients from ARM III (Non-Randomized) were excluded due to ineligibility.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

setting source links

Preserved source evidence · Not medical advice