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NCT00866281 · OUTCOME

Maximum Tolerated Dose (MTD) of Midostaurin- Posterior Probability of DLT

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
MEAN
Unit
probability estimates
Interval / dispersion
97.5% Confidence Interval
Time frame
Baseline, End of dose escalation phase (6 months)

What was measured

MTD was defined as highest dose level for which no more than 1 participant in a dose cohort experienced dose limiting toxicity (DLT), based on a Bayesian logistic regression model (BLRM) employing the escalation with overdose control (EWOC) principle. A DLT was defined as a grade 3 or 4 non-hematological adverse event (AE) or abnormal laboratory value related to study drug. Mean and the 95% posterior probability estimates of having a DLT by age strata and dose is presented. Estimation of MTD and/or recommended dose for expansion (RDE) at the dose-escalation phase of the study was based upon the estimation of the probability of DLT for participants in the dose-determining set (DDS).

Analysis population: The analysis was performed in dose determining set (DDS) population. Here, 'n' signifies the number of evaluable participants for this measure.

Groups in this outcome

Cohort 1: Midostaurin (30 Milligrams/Meters^2)

Participants received bodyweight and BSA stratified dose of midostaurin 30 mg/m\^2 bid through oral route. The total daily dose in 30 mg/m\^2 bid cohort was 60 mg/m\^2.

Cohort 2: Midostaurin (60 mg/m^2)

Participants received bodyweight and BSA stratified dose of midostaurin 60 mg/m\^2 bid through oral route. The total daily dose in 60 mg/m\^2 bid cohort was 120 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

GroupSource count
Cohort 1: Midostaurin (30 Milligrams/Meters^2)6
Cohort 2: Midostaurin (60 mg/m^2)11

Reported measurements

Younger stratum (≥ 3months -≤ 2 years): (n=2, 5)
Values in probability estimates · Interval/dispersion: 97.5% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Midostaurin (30 Milligrams/Meters^2)0.03Not reported00.16Not reported
Cohort 2: Midostaurin (60 mg/m^2)0.10Not reported0.010.33Not reported
Older stratum (>2 years- <18years): (n=4,6)
Values in probability estimates · Interval/dispersion: 97.5% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Midostaurin (30 Milligrams/Meters^2)0.03Not reported00.13Not reported
Cohort 2: Midostaurin (60 mg/m^2)0.08Not reported0.010.26Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0
        upper Limit
        0.16
        value
        0.03
      2. group Id
        OG001
        lower Limit
        0.01
        upper Limit
        0.33
        value
        0.10
    title
    Younger stratum (≥ 3months -≤ 2 years): (n=2, 5)
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0
        upper Limit
        0.13
        value
        0.03
      2. group Id
        OG001
        lower Limit
        0.01
        upper Limit
        0.26
        value
        0.08
    title
    Older stratum (>2 years- <18years): (n=4,6)
denoms
  1. counts
    1. group Id
      OG000
      value
      6
    2. group Id
      OG001
      value
      11
    units
    Participants
description
MTD was defined as highest dose level for which no more than 1 participant in a dose cohort experienced dose limiting toxicity (DLT), based on a Bayesian logistic regression model (BLRM) employing the escalation with overdose control (EWOC) principle. A DLT was defined as a grade 3 or 4 non-hematological adverse event (AE) or abnormal laboratory value related to study drug. Mean and the 95% posterior probability estimates of having a DLT by age strata and dose is presented. Estimation of MTD and/or recommended dose for expansion (RDE) at the dose-escalation phase of the study was based upon the estimation of the probability of DLT for participants in the dose-determining set (DDS).
dispersion Type
97.5% Confidence Interval
groups
  1. description
    Participants received bodyweight and BSA stratified dose of midostaurin 30 mg/m\^2 bid through oral route. The total daily dose in 30 mg/m\^2 bid cohort was 60 mg/m\^2.
    id
    OG000
    title
    Cohort 1: Midostaurin (30 Milligrams/Meters^2)
  2. description
    Participants received bodyweight and BSA stratified dose of midostaurin 60 mg/m\^2 bid through oral route. The total daily dose in 60 mg/m\^2 bid cohort was 120 mg/m\^2.
    id
    OG001
    title
    Cohort 2: Midostaurin (60 mg/m^2)
param Type
MEAN
population Description
The analysis was performed in dose determining set (DDS) population. Here, 'n' signifies the number of evaluable participants for this measure.
reporting Status
POSTED
time Frame
Baseline, End of dose escalation phase (6 months)
title
Maximum Tolerated Dose (MTD) of Midostaurin- Posterior Probability of DLT
type
PRIMARY
unit Of Measure
probability estimates

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice