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NCT04154189 · OUTCOME

Objective Response Rate at Month 4 (ORR-4m) by IIR Assessment

A Study to Compare the Efficacy and Safety of Ifosfamide and Etoposide With or Without Lenvatinib in Children, Adolescents and Young Adults With Relapsed and Refractory Osteosarcoma · Source last updated 2024-07-22

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
95% Confidence Interval
Time frame
Month 4

What was measured

ORR-4m was defined as the percentage of participants with best overall response of complete response (CR) or partial response (PR) as determined by IIR using RECIST v1.1 within the first 4 months. CR: defined as the disappearance of all target and non-target lesions (non-lymph nodes). All pathological lymph nodes (whether target or non-target) must have a reduction in their short axis to less than (\<) 10 mm. PR: defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. 95% confidence interval (CI) of ORR was calculated using the method of Clopper and Pearson.

Analysis population: FAS included all randomized participants regardless of the treatment actually received.

Groups in this outcome

Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide

Participants received lenvatinib 14 mg/m\^2, capsules, orally, once daily, plus ifosfamide 3000 mg/m\^2/day, intravenously and etoposide 100 mg/m\^2/day, intravenously. Ifosfamide and etoposide were administered on Days 1 to 3 of each 21-day cycle for up to 5 cycles. Lenvatinib was administered in continuous 21-day cycles. Treatment continued until PD, development of unacceptable toxicity, participant choice, withdrawal of consent or discontinuation of study by the sponsor, whichever occurred first.

Treatment Arm B: Ifosfamide + Etoposide

Participants received ifosfamide 3000 mg/m\^2/day, intravenously and etoposide 100 mg/m\^2/day, intravenously on Days 1 to 3 of each 21-day cycle for up to 5 cycles. Participants who had PD per RECIST v1.1 were eligible for an optional lenvatinib treatment (14 mg/m\^2, capsules, orally, once daily in continuous 21-day cycles until next PD, development of unacceptable toxicity, participant choice, withdrawal of consent or discontinuation of study by the sponsor, whichever occurred first) plus any remaining cycles of chemotherapy if 5 cycles were not completed prior to PD.

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

Analysis denominator · Participants

GroupSource count
Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide40
Treatment Arm B: Ifosfamide + Etoposide41

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide15.0Not reported5.729.8Not reported
Treatment Arm B: Ifosfamide + Etoposide7.3Not reported1.519.9Not reported

Source statistical analyses

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

  1. ci Lower Limit
    -6.4
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    22.3
    group Ids
    1. OG000
    2. OG001
    non Inferiority Comment
    Difference calculated as Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide minus Treatment Arm B: Ifosfamide + Etoposide. 2-sided 95% CI: Miettinen-Nurminen (Score) confidence limits, stratified by randomization stratification factor age (\<18 and \>=18 years).
    non Inferiority Type
    OTHER
    param Type
    Difference in Percentage
    param Value
    7.7
Complete source fields
analyses
  1. ci Lower Limit
    -6.4
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    22.3
    group Ids
    1. OG000
    2. OG001
    non Inferiority Comment
    Difference calculated as Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide minus Treatment Arm B: Ifosfamide + Etoposide. 2-sided 95% CI: Miettinen-Nurminen (Score) confidence limits, stratified by randomization stratification factor age (\<18 and \>=18 years).
    non Inferiority Type
    OTHER
    param Type
    Difference in Percentage
    param Value
    7.7
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        5.7
        upper Limit
        29.8
        value
        15.0
      2. group Id
        OG001
        lower Limit
        1.5
        upper Limit
        19.9
        value
        7.3
denoms
  1. counts
    1. group Id
      OG000
      value
      40
    2. group Id
      OG001
      value
      41
    units
    Participants
description
ORR-4m was defined as the percentage of participants with best overall response of complete response (CR) or partial response (PR) as determined by IIR using RECIST v1.1 within the first 4 months. CR: defined as the disappearance of all target and non-target lesions (non-lymph nodes). All pathological lymph nodes (whether target or non-target) must have a reduction in their short axis to less than (\<) 10 mm. PR: defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. 95% confidence interval (CI) of ORR was calculated using the method of Clopper and Pearson.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received lenvatinib 14 mg/m\^2, capsules, orally, once daily, plus ifosfamide 3000 mg/m\^2/day, intravenously and etoposide 100 mg/m\^2/day, intravenously. Ifosfamide and etoposide were administered on Days 1 to 3 of each 21-day cycle for up to 5 cycles. Lenvatinib was administered in continuous 21-day cycles. Treatment continued until PD, development of unacceptable toxicity, participant choice, withdrawal of consent or discontinuation of study by the sponsor, whichever occurred first.
    id
    OG000
    title
    Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide
  2. description
    Participants received ifosfamide 3000 mg/m\^2/day, intravenously and etoposide 100 mg/m\^2/day, intravenously on Days 1 to 3 of each 21-day cycle for up to 5 cycles. Participants who had PD per RECIST v1.1 were eligible for an optional lenvatinib treatment (14 mg/m\^2, capsules, orally, once daily in continuous 21-day cycles until next PD, development of unacceptable toxicity, participant choice, withdrawal of consent or discontinuation of study by the sponsor, whichever occurred first) plus any remaining cycles of chemotherapy if 5 cycles were not completed prior to PD.
    id
    OG001
    title
    Treatment Arm B: Ifosfamide + Etoposide
param Type
NUMBER
population Description
FAS included all randomized participants regardless of the treatment actually received.
reporting Status
POSTED
time Frame
Month 4
title
Objective Response Rate at Month 4 (ORR-4m) by IIR Assessment
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice