NCT00866281 · OUTCOME
Percentage of Participants With Best Overall Response by Indication
A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia · Source last updated 2015-12-22
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
- Measure
- NUMBER
- Unit
- Percentage of Participants
- Interval / dispersion
- Not reported
- Time frame
- Baseline, Day 15 (Day 1 of Cycle 2), Day 22 (Day 8 of Cycle 2), Day 29(Day 1 of Cycle 9), End of treatment (up to 24 months after last dose or until death whichever occurred first)
What was measured
The best overall clinical response was determined as per the clinical assessment done by the investigator. Responders were defined as all participants with a best clinical response of leukemia free state, morphological complete remission, incomplete morphological complete remission, partial remission, bone marrow blast response, bone marrow minor blast response, peripheral blood blast response, minor peripheral blood blast response. Participants with stable disease, progressive disease and with missing tumour assessment or who discontinued the study or who died before having their first assessment were considered as non-responders. Stable disease was defined as failure to achieve any of the above response. Progressive disease was defined as doubling of the bone marrow blast percentage from baseline in participants with \<40% bone marrow blasts at baseline, or a 50% increase in bone marrow blast percentage from baseline in participants with \>40% bone marrow blasts at baseline,
Analysis population: The analysis was performed in full analysis set (FAS) population, defined as all participants to whom study treatment was assigned.
Groups in this outcome
AML Subjects
Subjects with acute myeloid leukemia (AML) and received body-weight stratified dosage midostaurin 30 or 60 mg/m\^2.
MLLr-ALL Subjects
Subjects with mixed lineage leukemia gene- rearranged acute lymphoblastic leukemia (MLLr-ALL) and received body-weight stratified dosage midostaurin 30 or 60 mg/m\^2.
Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.
Analysis denominator · Participants
| Group | Source count |
|---|
| AML Subjects | 9 |
|---|
| MLLr-ALL Subjects | 13 |
|---|
Reported measurements
Leukemia free state
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Morphological complete remission
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Incomplete morphological complete remission
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 11.1 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Partial remission
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Bone marrow blast response
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 22.2 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Bone marrow minor blast response
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Peripheral blood blast response
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 11.1 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 23.1 | Not reported | Not reported | Not reported | Not reported |
|---|
Minor peripheral blood blast response
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 11.1 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
Stable disease
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 44.4 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 7.7 | Not reported | Not reported | Not reported | Not reported |
|---|
Progressive disease
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 61.5 | Not reported | Not reported | Not reported | Not reported |
|---|
Not Done
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 0 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 7.7 | Not reported | Not reported | Not reported | Not reported |
|---|
Participants with response
Values in Percentage of Participants · Interval/dispersion: Not reported| Group | Value | Spread | Lower | Upper | Comment |
|---|
| AML Subjects | 55.6 | Not reported | Not reported | Not reported | Not reported |
|---|
| MLLr-ALL Subjects | 23.1 | Not reported | Not reported | Not reported | Not reported |
|---|
Complete source fields
- classes
- categories
- measurements
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- title
- Leukemia free state
- categories
- measurements
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- title
- Morphological complete remission
- categories
- measurements
- group Id
- OG000
- value
- 11.1
- group Id
- OG001
- value
- 0
- title
- Incomplete morphological complete remission
- categories
- measurements
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- title
- Partial remission
- categories
- measurements
- group Id
- OG000
- value
- 22.2
- group Id
- OG001
- value
- 0
- title
- Bone marrow blast response
- categories
- measurements
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- title
- Bone marrow minor blast response
- categories
- measurements
- group Id
- OG000
- value
- 11.1
- group Id
- OG001
- value
- 23.1
- title
- Peripheral blood blast response
- categories
- measurements
- group Id
- OG000
- value
- 11.1
- group Id
- OG001
- value
- 0
- title
- Minor peripheral blood blast response
- categories
- measurements
- group Id
- OG000
- value
- 44.4
- group Id
- OG001
- value
- 7.7
- title
- Stable disease
- categories
- measurements
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 61.5
- title
- Progressive disease
- categories
- measurements
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 7.7
- title
- Not Done
- categories
- measurements
- group Id
- OG000
- value
- 55.6
- group Id
- OG001
- value
- 23.1
- title
- Participants with response
- denoms
- counts
- group Id
- OG000
- value
- 9
- group Id
- OG001
- value
- 13
- units
- Participants
- description
- The best overall clinical response was determined as per the clinical assessment done by the investigator. Responders were defined as all participants with a best clinical response of leukemia free state, morphological complete remission, incomplete morphological complete remission, partial remission, bone marrow blast response, bone marrow minor blast response, peripheral blood blast response, minor peripheral blood blast response. Participants with stable disease, progressive disease and with missing tumour assessment or who discontinued the study or who died before having their first assessment were considered as non-responders. Stable disease was defined as failure to achieve any of the above response. Progressive disease was defined as doubling of the bone marrow blast percentage from baseline in participants with \<40% bone marrow blasts at baseline, or a 50% increase in bone marrow blast percentage from baseline in participants with \>40% bone marrow blasts at baseline,
- groups
- description
- Subjects with acute myeloid leukemia (AML) and received body-weight stratified dosage midostaurin 30 or 60 mg/m\^2.
- id
- OG000
- title
- AML Subjects
- description
- Subjects with mixed lineage leukemia gene- rearranged acute lymphoblastic leukemia (MLLr-ALL) and received body-weight stratified dosage midostaurin 30 or 60 mg/m\^2.
- id
- OG001
- title
- MLLr-ALL Subjects
- param Type
- NUMBER
- population Description
- The analysis was performed in full analysis set (FAS) population, defined as all participants to whom study treatment was assigned.
- reporting Status
- POSTED
- time Frame
- Baseline, Day 15 (Day 1 of Cycle 2), Day 22 (Day 8 of Cycle 2), Day 29(Day 1 of Cycle 9), End of treatment (up to 24 months after last dose or until death whichever occurred first)
- title
- Percentage of Participants With Best Overall Response by Indication
- type
- SECONDARY
- unit Of Measure
- Percentage of Participants
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