NCT00702403 · STUDY IN CONTEXT

Nilotinib and Imatinib Mesylate After Donor Stem Cell Transplant in Treating Patients With ALL or CML

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. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. 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

Safety and tolerability of nilotinib therapy in patients with imatinib-sensitive leukemia graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events version 4.0. Treatment safety failure is defined for a patient with imatinib sensitive Ph+ leukemia as the inability to be able to deliver at least 400 milligrams per day of nilotinib in adults, and 230 milligrams/m2 per day in children, for at least 85% of the time interval between 81 and 365 days after transplant. The overall study will be considered successful if nilotinib is deliverable to more than 75% of the study participants at this minimum specified dose intensity.

Critical to note are two "intention-to-treat" populations: the 1st (N=57) at time of consent, evolved into the second ITT population (N=40), because 17 subjects lost eligibility to begin relapse prophylaxis at engraftment. A 2nd wave of discontinuations occurred at or after Day 81 when all patients were to be switched from imatinib to nilotinib.

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secondary · POSTED

To be considered a treatment efficacy success at 1 year posttransplant, the patient's bone marrow must demonstrate complete hematological remission, absence of Philadelphia chromosomes, and not satisfy any of the criteria for treatment failure (\>/= 1% aberrantly expressing marrow blasts by multiparameter flow cytometry, \>5% BCR/ABL in marrow by fluorescent in situ hybridization, or \>1 log rise in peripheral blood BCR/ABL by quantitative polymerase chain reaction (PCR) since day 80).

The analysis population was considered in two ways: first the number of treatment success in the entire cohort (by "intention to treat"), and second the number of treatment successes among only those patients who did not die before 1 year from non-relapse mortality.

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secondary · POSTED

The proportion of study participants alive at 1, 2 and 3 years

Overall Survival (complete follow-up is available out to three years for all patients)

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secondary · POSTED

The proportion of study participants alive and without hematologic, cytogenetic or molecular evidence of BCR/ABL-positive leukemia at 1 year

Proportion alive without relapse among the total number of patients with minimal residual disease follow-up data

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secondary · POSTED

The proportion of patients with hematologic, cytogenetic or molecular relapse of BCR/ABL-positive leukemia

Proportion of patients with hematologic, cytogenetic or molecular relapse of BCR/ABL-positive leukemia among those who did not die from non-relapse causes during the first year, and first 3-years after transplant.

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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-chronic-myeloid-leukemia-pediatric
nct id: NCT00702403
trial label: Nilotinib and Imatinib Mesylate After Donor Stem Cell Transplant in Treating Patients With ALL or CML
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. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
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: 5
harms Count: 1
qol Count: 0

outcomes

release id
clinical-indications-draft-1
disease state id
ds-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0000
source section id
NCT00702403-om-0000
source outcome index
0
endpoint
Number of Participants With Treatment Safety Failure
endpoint definition
Safety and tolerability of nilotinib therapy in patients with imatinib-sensitive leukemia graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events version 4.0. Treatment safety failure is defined for a patient with imatinib sensitive Ph+ leukemia as the inability to be able to deliver at least 400 milligrams per day of nilotinib in adults, and 230 milligrams/m2 per day in children, for at least 85% of the time interval between 81 and 365 days after transplant. The overall study will be considered successful if nilotinib is deliverable to more than 75% of the study participants at this minimum specified dose intensity.
time origin
Up to 365 days post-transplant
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
Critical to note are two "intention-to-treat" populations: the 1st (N=57) at time of consent, evolved into the second ITT population (N=40), because 17 subjects lost eligibility to begin relapse prophylaxis at engraftment. A 2nd wave of discontinuations occurred at or after Day 81 when all patients were to be switched from imatinib to nilotinib.
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-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0001
source section id
NCT00702403-om-0001
source outcome index
1
endpoint
The Proportion of Patients at 1 Year With Treatment Efficacy Success
endpoint definition
To be considered a treatment efficacy success at 1 year posttransplant, the patient's bone marrow must demonstrate complete hematological remission, absence of Philadelphia chromosomes, and not satisfy any of the criteria for treatment failure (\>/= 1% aberrantly expressing marrow blasts by multiparameter flow cytometry, \>5% BCR/ABL in marrow by fluorescent in situ hybridization, or \>1 log rise in peripheral blood BCR/ABL by quantitative polymerase chain reaction (PCR) since day 80).
time origin
Up to 1 year
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
The analysis population was considered in two ways: first the number of treatment success in the entire cohort (by "intention to treat"), and second the number of treatment successes among only those patients who did not die before 1 year from non-relapse mortality.
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-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0002
source section id
NCT00702403-om-0002
source outcome index
2
endpoint
Survival
endpoint definition
The proportion of study participants alive at 1, 2 and 3 years
time origin
Up to 3 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Overall Survival (complete follow-up is available out to three years for all 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-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0003
source section id
NCT00702403-om-0003
source outcome index
3
endpoint
Patients Alive With Out Relapse
endpoint definition
The proportion of study participants alive and without hematologic, cytogenetic or molecular evidence of BCR/ABL-positive leukemia at 1 year
time origin
Up to 1 year
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Proportion alive without relapse among the total number of patients with minimal residual disease follow-up data
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-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0004
source section id
NCT00702403-om-0004
source outcome index
4
endpoint
Relapse
endpoint definition
The proportion of patients with hematologic, cytogenetic or molecular relapse of BCR/ABL-positive leukemia
time origin
1 and 3 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Proportion of patients with hematologic, cytogenetic or molecular relapse of BCR/ABL-positive leukemia among those who did not die from non-relapse causes during the first year, and first 3-years after transplant.
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-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0000
arm key
OG000
arm label
Single Arm Nilotinib Relapse Prophylaxis
regimen
Beginning after engraftment and blood count recovery (21 to 28 days after allogeneic stem cell transplant), patients with imatinib-sensitive leukemia receive imatinib mesylate PO QD until day 80 and then nilotinib PO BID on days 81-445. Patients with imatinib-resistant leukemia receive nilotinib PO BID beginning after engraftment and blood count recovery until day 445. Treatment continues in the absence of disease progression or unacceptable toxicity.
analyzed count
40
analyzed unit
Participants
is comparator
Source null
source pointer
NCT00702403-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0001
arm key
OG000
arm label
Treatment (Prophylactic Inhibition of BCR-ABL Tyrosine Kinase)
regimen
Beginning after engraftment and blood count recovery (21 to 28 days after allogeneic stem cell transplant), patients with imatinib-sensitive leukemia receive imatinib mesylate PO once daily until day 80 and then nilotinib PO twice daily on days 81-445. Patients with imatinib-resistant leukemia receive nilotinib PO twice daily beginning after engraftment and blood count recovery until day 445. Treatment continues in the absence of disease progression or unacceptable toxicity.
analyzed count
40
analyzed unit
Participants
is comparator
Source null
source pointer
NCT00702403-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0002
arm key
OG000
arm label
Treatment (Prophylactic Inhibition of BCR-ABL Tyrosine Kinase)
regimen
Beginning after engraftment and blood count recovery (21 to 28 days after allogeneic stem cell transplant), patients with imatinib-sensitive leukemia receive imatinib mesylate PO once daily until day 80 and then nilotinib PO twice daily on days 81-445. Patients with imatinib-resistant leukemia receive nilotinib PO twice daily beginning after engraftment and blood count recovery until day 445. Treatment continues in the absence of disease progression or unacceptable toxicity. nilotinib: Given PO imatinib mesylate: Given PO pharmacological study: Correlative studies
analyzed count
40
analyzed unit
Participants
is comparator
Source null
source pointer
NCT00702403-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0003
arm key
OG000
arm label
Single Arm Nilotinib Relapse Prophylaxis
regimen
Beginning after engraftment and blood counts recover (21 to 28 days after allogeneic stem cell transplant), patients with imatinib-sensitive leukemia receive imatinib mesylate PO once daily until day 80 and then nilotinib PO twice daily on days 81-445. Patients with imatinib-resistant leukemia receive nilotinib PO twice daily beginning after engraftment and blood counts recover until day 445. Treatment continues in the absence of disease progression or unacceptable toxicity.
analyzed count
40
analyzed unit
Participants
is comparator
Source null
source pointer
NCT00702403-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
outcome key
om-0004
arm key
OG000
arm label
Treatment (Prophylactic Inhibition of BCR-ABL Tyrosine Kinase)
regimen
Beginning after engraftment and blood count recovery (21 to 28 days after allogeneic stem cell transplant), patients with imatinib-sensitive leukemia receive imatinib mesylate PO once daily until day 80 and then nilotinib PO twice daily on days 81-445. Patients with imatinib-resistant leukemia receive nilotinib PO twice daily beginning after engraftment and blood count recovery until day 445. Treatment continues in the absence of disease progression or unacceptable toxicity.
analyzed count
40
analyzed unit
Participants
is comparator
Source null
source pointer
NCT00702403-om-0004#groups/OG000

harms

release id
clinical-indications-draft-1
disease state id
ds-chronic-myeloid-leukemia-pediatric
nct id
NCT00702403
harm key
EG000
source section id
NCT00702403-ae
event group key
EG000
event group label
Single Arm Nilotinib Relapse Prophylaxis
serious affected
20
serious at risk
40
other affected
31
other at risk
40
frequency threshold
0
collection period
Adverse Events: Conditioning through Day 100; Severe Adverse Events Conditioning >Day 100
event definition
Source null
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