NCT02435849 · OUTCOME

Response as a Function of Baseline Tumor Burden (Tumor Load) in Main Cohort Only

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: 3 months

What was measured

Percentage of participants who achieved BOR of CR or CRi by flow cytometry as a function of baseline bone marrow tumor burden.

Analysis population: FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel with a baseline tumor burden assessment.

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

Group	Source count
Main Cohort: Single Dose of CTL019	79
Cohort 1: Single Dose of CTL019	1

Reported measurements

Baseline bone marrow tumor burden: Low (<50%)

Analysis denominator · Participants

Group	Source count
Main Cohort: Single Dose of CTL019	25
Cohort 1: Single Dose of CTL019	1

Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Main Cohort: Single Dose of CTL019	96.0	Not reported	79.6	99.9	Not reported
Cohort 1: Single Dose of CTL019	100.0	Not reported	NA	NA	NA: 95% CI could not be calculated as there was only 1 participant who had low baseline tumor burden

Baseline bone marrow tumor burden: High (>=50%)

Analysis denominator · Participants

Group	Source count
Main Cohort: Single Dose of CTL019	54
Cohort 1: Single Dose of CTL019	0

Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Main Cohort: Single Dose of CTL019	75.9	Not reported	62.4	86.5	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
lower Limit
79.6
upper Limit
99.9
value
96.0
comment
NA: 95% CI could not be calculated as there was only 1 participant who had low baseline tumor burden
group Id
OG001
lower Limit
NA
upper Limit
NA
value
100.0
denoms
counts
group Id
OG000
value
25
group Id
OG001
value
1
units
Participants
title
Baseline bone marrow tumor burden: Low (<50%)
categories
measurements
group Id
OG000
lower Limit
62.4
upper Limit
86.5
value
75.9
denoms
counts
group Id
OG000
value
54
group Id
OG001
value
0
units
Participants
title
Baseline bone marrow tumor burden: High (>=50%)

denoms

counts
group Id
OG000
value
79
group Id
OG001
value
1
units
Participants

description

Percentage of participants who achieved BOR of CR or CRi by flow cytometry as a function of baseline bone marrow tumor burden.

dispersion Type

95% Confidence Interval

groups

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
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

FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel with a baseline tumor burden assessment.

reporting Status

POSTED

time Frame

3 months

title

Response as a Function of Baseline Tumor Burden (Tumor Load) in Main Cohort Only

type

SECONDARY

unit Of Measure

Percentage of participants

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

Preserved source evidence · Not medical advice