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NCT02435849 · OUTCOME

Percentage of Participants With Overall Remission Rate (ORR) as Determined by Independent Review Committee (IRC) Assessment.

Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients · Source last updated 2024-02-13

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
95% Confidence Interval
Time frame
during the 3 months after tisagenlecleucel administration

What was measured

Evaluating the efficacy of tisagenlecleucel therapy from all manufacturing facilities as measured by overall remission rate (ORR) during the 3 months after tisagenlecleucel administration. ORR included complete response (CR) and CR with incomplete blood count recovery (CRi) as determined by an Independent Review Committee assessment. Per response criteria defined by National Comprehensive Cancer Network (NCCN), American Society of Hematology (ASH) and International Working Group (IWG) guidelines. CR is defined as: Bone marrow \<5% blasts, Peripheral blood: Neutrophils \>1.0 x 10\^9/L, and Platelets \>100 x 10\^9/L and Circulating blasts \<1% and No evidence of extramedullary disease, at least 7 days transfusion independency. CRi is defined as all the prior criteria being met, except that the following exists: Neutrophils ≤1.0 x 10\^9/L, and/or Platelets ≤100 x 10\^9/L, and/or Platelet and/or neutrophil transfusions ≤7 days before the date of the peripheral blood sample for disease assessment.

Analysis population: Full Analysis Set (FAS): The Full analysis set comprised all patients who received infusion of tisagenlecleucel in the Main Cohort.

Groups in this outcome

Main Cohort: Single Dose of CTL019

Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019).

Cohort 1: Single Dose of CTL019

Pediatric B-ALL patients who are very high risk at first relapse.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Main Cohort: Single Dose of CTL01979
Cohort 1: Single Dose of CTL0191

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Main Cohort: Single Dose of CTL01982.3Not reported72.190.0Not reported
Cohort 1: Single Dose of CTL019100.0Not reportedNANANA: Confidence Interval could not be calculated for only 1 participant.

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

  1. group Ids
    1. OG000
    non Inferiority Comment
    testing the null hypothesis that ORR within 3 months is less than or equal to 20%
    non Inferiority Type
    OTHER
    p Value
    <.0001
    statistical Method
    Clopper-Pearson
Complete source fields
analyses
  1. group Ids
    1. OG000
    non Inferiority Comment
    testing the null hypothesis that ORR within 3 months is less than or equal to 20%
    non Inferiority Type
    OTHER
    p Value
    <.0001
    statistical Method
    Clopper-Pearson
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        72.1
        upper Limit
        90.0
        value
        82.3
      2. comment
        NA: Confidence Interval could not be calculated for only 1 participant.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        100.0
denoms
  1. counts
    1. group Id
      OG000
      value
      79
    2. group Id
      OG001
      value
      1
    units
    Participants
description
Evaluating the efficacy of tisagenlecleucel therapy from all manufacturing facilities as measured by overall remission rate (ORR) during the 3 months after tisagenlecleucel administration. ORR included complete response (CR) and CR with incomplete blood count recovery (CRi) as determined by an Independent Review Committee assessment. Per response criteria defined by National Comprehensive Cancer Network (NCCN), American Society of Hematology (ASH) and International Working Group (IWG) guidelines. CR is defined as: Bone marrow \<5% blasts, Peripheral blood: Neutrophils \>1.0 x 10\^9/L, and Platelets \>100 x 10\^9/L and Circulating blasts \<1% and No evidence of extramedullary disease, at least 7 days transfusion independency. CRi is defined as all the prior criteria being met, except that the following exists: Neutrophils ≤1.0 x 10\^9/L, and/or Platelets ≤100 x 10\^9/L, and/or Platelet and/or neutrophil transfusions ≤7 days before the date of the peripheral blood sample for disease assessment.
dispersion Type
95% Confidence Interval
groups
  1. description
    Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019).
    id
    OG000
    title
    Main Cohort: Single Dose of CTL019
  2. description
    Pediatric B-ALL patients who are very high risk at first relapse.
    id
    OG001
    title
    Cohort 1: Single Dose of CTL019
param Type
NUMBER
population Description
Full Analysis Set (FAS): The Full analysis set comprised all patients who received infusion of tisagenlecleucel in the Main Cohort.
reporting Status
POSTED
time Frame
during the 3 months after tisagenlecleucel administration
title
Percentage of Participants With Overall Remission Rate (ORR) as Determined by Independent Review Committee (IRC) Assessment.
type
PRIMARY
unit Of Measure
Percentage of participants

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

Preserved source evidence · Not medical advice