NCT03245151 · OUTCOME

Phase 2: Clinical Benefit Rate (CBR)

Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including Central Nervous System Tumors · Source last updated 2023-08-30

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: From first dose of study drug until PD or death, whichever occurred first (up to 6.5 months)

What was measured

CBR was defined as percentage of participants who had BOR of CR, PR, or durable SD (SD duration \>=23 weeks since the first dose of study treatment) divided by number of participants in analysis set. CBR was assessed by an investigator based on RECIST v1.1 for non-HGG cohorts or RANO for HGG cohort. CR: 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 \<10 mm. PR: at least a 30% decrease in the SOD of target lesions, taking as reference the baseline sum diameters. SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest SOD. PD: at least 20% increase (including an absolute increase of at least 5 mm) in the SOD of target lesions, taking as reference the smallest sum and/or unequivocal progression of existing non-target lesions and/or appearance of 1 or more new lesions.

Analysis population: Evaluable analysis set included all evaluable participants who have measurable disease present at baseline, and at least one postbaseline efficacy assessment, unless they have discontinued prior to the first efficacy assessment due to PD.

Groups in this outcome

Phase 2: Cohort 1, Ewing Sarcoma

Participants with recurrent or refractory ewing sarcoma received lenvatinib 11 mg/m\^2, capsules, orally, once daily in combination with everolimus 3 mg/m\^2, tablets, orally, once daily for up to 7 cycles (each cycle was 28 days) unless participant discontinued early from the study.

Phase 2: Cohort 2, Rhabdomyosarcoma

Participants with recurrent or refractory rhabdomyosarcoma received lenvatinib 11 mg/m\^2, capsules, orally, once daily in combination with everolimus 3 mg/m\^2, tablets, orally, once daily for up to 7 cycles (each cycle was 28 days) unless participant discontinued early from the study.

Phase 2: Cohort 3, HGG

Participants with recurrent or refractory HGG received lenvatinib 11 mg/m\^2, capsules, orally, once daily in combination with everolimus 3 mg/m\^2, tablets, orally, once daily for up to 7 cycles (each cycle was 28 days) unless participant discontinued early from the study.

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
Phase 2: Cohort 1, Ewing Sarcoma	10
Phase 2: Cohort 2, Rhabdomyosarcoma	20
Phase 2: Cohort 3, HGG	10

Reported measurements

Source class 1

Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Phase 2: Cohort 1, Ewing Sarcoma	20.0	2.5	2.5	55.6	Not reported
Phase 2: Cohort 2, Rhabdomyosarcoma	10.0	1.2	1.2	31.7	Not reported
Phase 2: Cohort 3, HGG	0.0	0.0	0.0	30.8	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
lower Limit
2.5
spread
2.5
upper Limit
55.6
value
20.0
group Id
OG001
lower Limit
1.2
spread
1.2
upper Limit
31.7
value
10.0
group Id
OG002
lower Limit
0.0
spread
0.0
upper Limit
30.8
value
0.0

denoms

counts
group Id
OG000
value
10
group Id
OG001
value
20
group Id
OG002
value
10
units
Participants

description

dispersion Type

95% Confidence Interval

groups

description
Participants with recurrent or refractory ewing sarcoma received lenvatinib 11 mg/m\^2, capsules, orally, once daily in combination with everolimus 3 mg/m\^2, tablets, orally, once daily for up to 7 cycles (each cycle was 28 days) unless participant discontinued early from the study.
id
OG000
title
Phase 2: Cohort 1, Ewing Sarcoma
description
Participants with recurrent or refractory rhabdomyosarcoma received lenvatinib 11 mg/m\^2, capsules, orally, once daily in combination with everolimus 3 mg/m\^2, tablets, orally, once daily for up to 7 cycles (each cycle was 28 days) unless participant discontinued early from the study.
id
OG001
title
Phase 2: Cohort 2, Rhabdomyosarcoma
description
Participants with recurrent or refractory HGG received lenvatinib 11 mg/m\^2, capsules, orally, once daily in combination with everolimus 3 mg/m\^2, tablets, orally, once daily for up to 7 cycles (each cycle was 28 days) unless participant discontinued early from the study.
id
OG002
title
Phase 2: Cohort 3, HGG

param Type

NUMBER

population Description

Evaluable analysis set included all evaluable participants who have measurable disease present at baseline, and at least one postbaseline efficacy assessment, unless they have discontinued prior to the first efficacy assessment due to PD.

reporting Status

POSTED

time Frame

From first dose of study drug until PD or death, whichever occurred first (up to 6.5 months)

title

Phase 2: Clinical Benefit Rate (CBR)

type

SECONDARY

unit Of Measure

percentage of participants

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Preserved source evidence · Not medical advice