NCT02610777 · OUTCOME

Percentage of Participants With CR by Cycle 4

An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML) · Source last updated 2022-09-19

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: Not reported
Time frame: From date of randomization until CR by Cycle 4 (cycle length is equal to [=] 28 days)

What was measured

Disease responses for HR MDS or CMML were based on modified IWG response criteria for MDS and for low-blast AML on revised IWG response criteria for AML. CR for HR MDS or CMML: \<=5% myeloblasts with normal maturation of all cell lines in bone marrow, and \>=11 g/dL Hb, \>=100\*10\^9/L plt, ANC \>=1.0\*10\^9/L and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, ANC of more than 1.0\*10\^9/L and plt of \>=100\*10\^9/L, transfusion independence, no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: some participants fulfill all criteria for CR except for residual neutropenia (\<1.0\*10\^9/L)/TTP (\<100\*10\^9/L). The percentages are rounded off to report the nearest whole numbers.

Analysis population: REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Overall number analyzed is the number of participants with data available for analyses.

Groups in this outcome

Azacitidine 75 mg/m^2

Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).

Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2

Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 and pevonedistat 20 mg/m\^2, infusion, intravenously, on Days 1, 3, and 5 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).

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
Azacitidine 75 mg/m^2	38
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	38

Reported measurements

Source class 1

Values in percentage of participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	13.2	Not reported	Not reported	Not reported	Not reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	26.3	Not reported	Not reported	Not reported	Not reported

Source statistical analyses

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

ci Lower Limit
-4.49
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
30.81
estimate Comment
Absolute difference \>0 represent better response achieved on pevonedistat combination compared to azacitidine treatment alone.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.152
p Value Comment
P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
13.16
statistical Method
Cochran-Mantel-Haenszel

Complete source fields

analyses

ci Lower Limit
-4.49
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
30.81
estimate Comment
Absolute difference \>0 represent better response achieved on pevonedistat combination compared to azacitidine treatment alone.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.152
p Value Comment
P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
13.16
statistical Method
Cochran-Mantel-Haenszel

classes

categories
measurements
group Id
OG000
value
13.2
group Id
OG001
value
26.3

denoms

counts
group Id
OG000
value
38
group Id
OG001
value
38
units
Participants

description

groups

description
Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).
id
OG000
title
Azacitidine 75 mg/m^2
description
Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 and pevonedistat 20 mg/m\^2, infusion, intravenously, on Days 1, 3, and 5 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).
id
OG001
title
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2

param Type

NUMBER

population Description

REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Overall number analyzed is the number of participants with data available for analyses.

reporting Status

POSTED

time Frame

From date of randomization until CR by Cycle 4 (cycle length is equal to [=] 28 days)

title

Percentage of Participants With CR by Cycle 4

type

SECONDARY

unit Of Measure

percentage of participants

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice