NCT04154189 · OUTCOME

Percentage of Participants With PFS at Month 4 (PFS-4m Rate) 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

PFS rate at 4 months as assessed by IIR was defined as the percentage of participants who were alive and without PD at 4 months from the randomization date using RECIST v1.1. PD was defined as at least a 20% increase or 5 mm increase in the sum of diameters of target lesions (taking as reference the smallest sum on study) recorded since the treatment started or the appearance of 1 or more new lesions. The PFS-4m was estimated using the Kaplan-Meier method.

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

Group	Source count
Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide	40
Treatment Arm B: Ifosfamide + Etoposide	41

Reported measurements

Source class 1

Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Treatment Arm A: Lenvatinib + Ifosfamide + Etoposide	76.3	Not reported	59.3	86.9	Not reported
Treatment Arm B: Ifosfamide + Etoposide	66.0	Not reported	47.7	79.2	Not reported

Source statistical analyses

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

ci Lower Limit
-10.6
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
31.1
group Ids
OG000
OG001
non Inferiority Comment
Difference in PFS rates, and 2-sided 95% confidence intervals (CIs): CI and p-value constructed using the difference of the 2 Kaplan-Meier PFS rates (4 months) and the 2 corresponding Greenwood standard errors.
non Inferiority Type
OTHER
p Value
0.1683
p Value Comment
One-sided P value
param Type
Difference in Percentage
param Value
10.2
statistical Method
Kaplan-Meier Method

Complete source fields

analyses

ci Lower Limit
-10.6
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
31.1
group Ids
OG000
OG001
non Inferiority Comment
Difference in PFS rates, and 2-sided 95% confidence intervals (CIs): CI and p-value constructed using the difference of the 2 Kaplan-Meier PFS rates (4 months) and the 2 corresponding Greenwood standard errors.
non Inferiority Type
OTHER
p Value
0.1683
p Value Comment
One-sided P value
param Type
Difference in Percentage
param Value
10.2
statistical Method
Kaplan-Meier Method

classes

categories
measurements
group Id
OG000
lower Limit
59.3
upper Limit
86.9
value
76.3
group Id
OG001
lower Limit
47.7
upper Limit
79.2
value
66.0

denoms

counts
group Id
OG000
value
40
group Id
OG001
value
41
units
Participants

description

dispersion Type

95% Confidence Interval

groups

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
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

Percentage of Participants With PFS at Month 4 (PFS-4m Rate) by IIR Assessment

type

SECONDARY

unit Of Measure

percentage of participants

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

Preserved source evidence · Not medical advice