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NCT03173248 · OUTCOME

Event-Free Survival (EFS)

Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation · Source last updated 2026-06-29

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
MEDIAN
Unit
months
Interval / dispersion
95% Confidence Interval
Time frame
Up to Week 24

What was measured

EFS was defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure was defined as failure to achieve complete remission (CR) by Week 24. CR: Bone marrow blasts \<5% and no Auer rods; absence of extramedullary disease; Absolute neutrophil count (ANC) ≥1.0 × 10\^9 per litre (10\^9/L) (1000 per microlitre \[1000/μL\]); platelet count ≥100 × 10\^9/L (100,000/μL); independence of red blood cell transfusions. Participants who had an EFS event (relapse or death) after, 2 or more missing disease assessments were censored at the last adequate disease assessment documenting no relapse before the missing assessments. The reported data represents the Kaplan-Meier median value.

Analysis population: FAS included all participants who were randomized.

Groups in this outcome

AG-120 + Azacitidine

Participants received AG-120 500 mg orally, QD in combination with azacitidine 75 mg/m\^2/day SC or IV, on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation.

Placebo + Azacitidine

Participants received AG-120 matching placebo orally, QD in combination with azacitidine 75 mg/m\^2/day SC or IV, on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation.

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

Analysis denominator · Participants

GroupSource count
AG-120 + Azacitidine72
Placebo + Azacitidine74

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
AG-120 + Azacitidine0.03Not reported0.0311.01Not reported
Placebo + Azacitidine0.03Not reportedNANAThe upper and lower limit of 95% confidence interval (CI) were not estimable due to low number of participants with events.

Source statistical analyses

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

  1. ci Lower Limit
    0.16
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    0.69
    estimate Comment
    Hazard ratio is estimated using a Cox's proportional hazards model stratified by the randomization stratification factors (AML status and geographic region) with placebo + azacitidine as the denominator.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0011
    p Value Comment
    P-value is calculated from the one-sided log-rank test stratified by the randomization stratification factors (AML status and geographic region).
    param Type
    Hazard Ratio (HR)
    param Value
    0.33
    statistical Method
    Log Rank
Complete source fields
analyses
  1. ci Lower Limit
    0.16
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    0.69
    estimate Comment
    Hazard ratio is estimated using a Cox's proportional hazards model stratified by the randomization stratification factors (AML status and geographic region) with placebo + azacitidine as the denominator.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0011
    p Value Comment
    P-value is calculated from the one-sided log-rank test stratified by the randomization stratification factors (AML status and geographic region).
    param Type
    Hazard Ratio (HR)
    param Value
    0.33
    statistical Method
    Log Rank
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.03
        upper Limit
        11.01
        value
        0.03
      2. comment
        The upper and lower limit of 95% confidence interval (CI) were not estimable due to low number of participants with events.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        0.03
denoms
  1. counts
    1. group Id
      OG000
      value
      72
    2. group Id
      OG001
      value
      74
    units
    Participants
description
EFS was defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure was defined as failure to achieve complete remission (CR) by Week 24. CR: Bone marrow blasts \<5% and no Auer rods; absence of extramedullary disease; Absolute neutrophil count (ANC) ≥1.0 × 10\^9 per litre (10\^9/L) (1000 per microlitre \[1000/μL\]); platelet count ≥100 × 10\^9/L (100,000/μL); independence of red blood cell transfusions. Participants who had an EFS event (relapse or death) after, 2 or more missing disease assessments were censored at the last adequate disease assessment documenting no relapse before the missing assessments. The reported data represents the Kaplan-Meier median value.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received AG-120 500 mg orally, QD in combination with azacitidine 75 mg/m\^2/day SC or IV, on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation.
    id
    OG000
    title
    AG-120 + Azacitidine
  2. description
    Participants received AG-120 matching placebo orally, QD in combination with azacitidine 75 mg/m\^2/day SC or IV, on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation.
    id
    OG001
    title
    Placebo + Azacitidine
param Type
MEDIAN
population Description
FAS included all participants who were randomized.
reporting Status
POSTED
time Frame
Up to Week 24
title
Event-Free Survival (EFS)
type
PRIMARY
unit Of Measure
months

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

Preserved source evidence · Not medical advice