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NCT03814005 · OUTCOME

Part B: Percentage of HR MDS and AML Participants With Overall Response

A Study of Pevonedistat in People With Blood Cancers or Solid Tumors With Kidney or Liver Problems · Source last updated 2024-09-05

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
Cycle 2 Day 22, Cycles 5, 8, and 11 (between Days 15 and 28), and every 6 cycles (up to 2 years 9 months) (Cycle length= 28 days)

What was measured

Overall Response Rate is defined as CR+CRi+PR+HI. CR:≤5% myeloblasts with normal maturation of all bone marrow(BM)cell lines,≥11g/dL Hgb,≥100\*10\^9/L pl,≥1.0\*10\^9/L neutrophils,0% blasts in peripheral blood;PR:all CR criteria met except BM blasts ≥50% decrease over pretreatment but still \>5%;HI:Hgb increase(inc) ≥1.5g/dL if baseline(BL)\<11 g/dL;pl inc≥30\*10\^9/L if BL\>20\*10\^9/L or inc from \<20\*10\^9/L to \>20\*10\^9/L and by 100%;neutrophil inc by 100%;absolute inc of \>0.5\*10\^9/L if BL\<100\*10\^9/L.For AML-CR:morphologic leukemia-free state \>1.0\*10\^9 neutrophils,≥100\*10\^9/L pl,transfusion independence,no residual evidence of extramedullary leukemia;CR with incomplete blood count recovery:fulfill CR criteria except residual neutropenia (\<1000/μL) or thrombocytopenia (\<100,000/μL);PR:all CR hematological values but ≥50% decrease in BM aspirate.

Analysis population: Response-evaluable population included participants who received at least 1 dose of study drug, had a baseline disease assessment, and had at least 1 postbaseline disease assessment to analyze response. Percentages are rounded off to whole number at the nearest decimal.

Groups in this outcome

Part B: Control Arm

Pevonedistat 20 milligram per square meter (mg/m\^2), infusion, intravenously (IV), once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days. Following Part A participants received azacitidine 75 mg/m\^2, injection, subcutaneously (SC) in Cycle 1 or SC or IV in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8 and 9 in combination with pevonedistat 20 mg/m\^2, infusion, IV, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies in Part B until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.

Part B: Renal Arm

Pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days. Following Part A participants received azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8-9 in combination with a starting dose of pevonedistat 10 mg/m\^2 to a maximum dose of pevonedistat 15 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies in Part B until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.

Part B: Mild Hepatic Arm

Pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days. Following Part A participants received azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8 and 9 in combination with a starting dose of pevonedistat 10 mg/m\^2 to a maximum dose of pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.

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

Analysis denominator · Participants

GroupSource count
Part B: Control Arm8
Part B: Renal Arm1
Part B: Mild Hepatic Arm2

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Part B: Control Arm50Not reportedNot reportedNot reportedNot reported
Part B: Renal Arm0Not reportedNot reportedNot reportedNot reported
Part B: Mild Hepatic Arm0Not reportedNot reportedNot reportedNot reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        50
      2. group Id
        OG001
        value
        0
      3. group Id
        OG002
        value
        0
denoms
  1. counts
    1. group Id
      OG000
      value
      8
    2. group Id
      OG001
      value
      1
    3. group Id
      OG002
      value
      2
    units
    Participants
description
Overall Response Rate is defined as CR+CRi+PR+HI. CR:≤5% myeloblasts with normal maturation of all bone marrow(BM)cell lines,≥11g/dL Hgb,≥100\*10\^9/L pl,≥1.0\*10\^9/L neutrophils,0% blasts in peripheral blood;PR:all CR criteria met except BM blasts ≥50% decrease over pretreatment but still \>5%;HI:Hgb increase(inc) ≥1.5g/dL if baseline(BL)\<11 g/dL;pl inc≥30\*10\^9/L if BL\>20\*10\^9/L or inc from \<20\*10\^9/L to \>20\*10\^9/L and by 100%;neutrophil inc by 100%;absolute inc of \>0.5\*10\^9/L if BL\<100\*10\^9/L.For AML-CR:morphologic leukemia-free state \>1.0\*10\^9 neutrophils,≥100\*10\^9/L pl,transfusion independence,no residual evidence of extramedullary leukemia;CR with incomplete blood count recovery:fulfill CR criteria except residual neutropenia (\<1000/μL) or thrombocytopenia (\<100,000/μL);PR:all CR hematological values but ≥50% decrease in BM aspirate.
groups
  1. description
    Pevonedistat 20 milligram per square meter (mg/m\^2), infusion, intravenously (IV), once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days. Following Part A participants received azacitidine 75 mg/m\^2, injection, subcutaneously (SC) in Cycle 1 or SC or IV in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8 and 9 in combination with pevonedistat 20 mg/m\^2, infusion, IV, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies in Part B until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.
    id
    OG000
    title
    Part B: Control Arm
  2. description
    Pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days. Following Part A participants received azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8-9 in combination with a starting dose of pevonedistat 10 mg/m\^2 to a maximum dose of pevonedistat 15 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies in Part B until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.
    id
    OG001
    title
    Part B: Renal Arm
  3. description
    Pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Day 1 of Part A in participants with hematologic malignancies, followed by a washout period of approximately 4 to 7 days. Following Part A participants received azacitidine 75 mg/m\^2, injection, subcutaneously in Cycle 1 or subcutaneously or intravenously in Cycle 2 and subsequent cycles, once on Day 1 through Day 7 or Day 1 through Day 5, and on Days 8 and 9 in combination with a starting dose of pevonedistat 10 mg/m\^2 to a maximum dose of pevonedistat 20 mg/m\^2, infusion, intravenously, once, on Days 1, 3, and 5 in each 28-day treatment cycle in participants with hematologic malignancies until symptomatic deterioration or PD, discontinuation for any reason, study stopped by the sponsor, or up to 12 cycles.
    id
    OG002
    title
    Part B: Mild Hepatic Arm
param Type
NUMBER
population Description
Response-evaluable population included participants who received at least 1 dose of study drug, had a baseline disease assessment, and had at least 1 postbaseline disease assessment to analyze response. Percentages are rounded off to whole number at the nearest decimal.
reporting Status
POSTED
time Frame
Cycle 2 Day 22, Cycles 5, 8, and 11 (between Days 15 and 28), and every 6 cycles (up to 2 years 9 months) (Cycle length= 28 days)
title
Part B: Percentage of HR MDS and AML Participants With Overall Response
type
SECONDARY
unit Of Measure
percentage of participants

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

Preserved source evidence · Not medical advice