Skip to content
Triangle logo TriangleGet started
← Full study record

NCT02610777 · OUTCOME

Percentage of Participants With Overall Response 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, PR or HI, by Cycle 4 (cycle length=28 days)

What was measured

Disease responses (HR MDS/CMML): modified IWG criteria for MDS; low-blast (LB) AML: revised IWG criteria for AML. Overall response(HR MDS/CMML)=CR,PR/HI,LB AML=CR+ CR with incomplete blood count recovery(Cri)+PR.HR MDS/CMML-CR:\<=5%myeloblasts with normal maturation of bone marrow (BM) cell lines,\>=11g/dL Hb,\>=100\*10\^9/L plt,\>=1.0\*10\^9/L ANC,0% blasts in peripheral blood; PR:CR criteria met except BM blasts\>=50%less over pretreatment but still\>5%; HI:hb increase (inc)\>=1.5g/dL if baseline\<11g/dL;plt inc\>=30\*10\^9/L if baseline\>20\*10\^9/L/Inc. from \<20\*10\^9/L-\>20\*10\^9/L, ANC inc. by 100%;absolute inc. of\>0.5\*10\^9/L if baseline\<100\*10\^9/L. LB AML-CR:morphologic leukemia-freestate\>1.0\*10\^9ANC,\>=100\*10\^9/L plt, transfusion independence, no residual evidence of extramedullary leukemia;CRi:fulfill CR criteria except residual neutropenia\<1.0\*10\^9/L/TTP\<100\*10\^9/L;PR:all CR hematological values but\>=50%less in BM aspirate. 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

GroupSource count
Azacitidine 75 mg/m^238
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^238

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^244.7Not reportedNot reportedNot reportedNot reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^257.9Not reportedNot reportedNot reportedNot reported

Source statistical analyses

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

  1. ci Lower Limit
    -9.12
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    35.44
    estimate Comment
    Absolute difference \>0 represent better response achieved on pevonedistat combination compared to azacitidine treatment alone.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.254
    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
  1. ci Lower Limit
    -9.12
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    35.44
    estimate Comment
    Absolute difference \>0 represent better response achieved on pevonedistat combination compared to azacitidine treatment alone.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.254
    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
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        44.7
      2. group Id
        OG001
        value
        57.9
denoms
  1. counts
    1. group Id
      OG000
      value
      38
    2. group Id
      OG001
      value
      38
    units
    Participants
description
Disease responses (HR MDS/CMML): modified IWG criteria for MDS; low-blast (LB) AML: revised IWG criteria for AML. Overall response(HR MDS/CMML)=CR,PR/HI,LB AML=CR+ CR with incomplete blood count recovery(Cri)+PR.HR MDS/CMML-CR:\<=5%myeloblasts with normal maturation of bone marrow (BM) cell lines,\>=11g/dL Hb,\>=100\*10\^9/L plt,\>=1.0\*10\^9/L ANC,0% blasts in peripheral blood; PR:CR criteria met except BM blasts\>=50%less over pretreatment but still\>5%; HI:hb increase (inc)\>=1.5g/dL if baseline\<11g/dL;plt inc\>=30\*10\^9/L if baseline\>20\*10\^9/L/Inc. from \<20\*10\^9/L-\>20\*10\^9/L, ANC inc. by 100%;absolute inc. of\>0.5\*10\^9/L if baseline\<100\*10\^9/L. LB AML-CR:morphologic leukemia-freestate\>1.0\*10\^9ANC,\>=100\*10\^9/L plt, transfusion independence, no residual evidence of extramedullary leukemia;CRi:fulfill CR criteria except residual neutropenia\<1.0\*10\^9/L/TTP\<100\*10\^9/L;PR:all CR hematological values but\>=50%less in BM aspirate. The percentages are rounded off to report the nearest whole numbers.
groups
  1. 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
  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, PR or HI, by Cycle 4 (cycle length=28 days)
title
Percentage of Participants With Overall Response 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