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NCT01915498 · OUTCOME

Phase 1 Dose Escalation: Rate of Complete Response and Complete Response With Incomplete Hematological Recovery (CR/CRi/CRp) by Total Daily Dose

Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation · Source last updated 2024-10-22

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
Response assessments were performed every 28 days through Month 12 and every 56 days thereafter until the end of treatment; median duration of treatment exposure in the Phase 1 Dose Escalation was 5.0 months (range 0.4 to 34.2 months).

What was measured

The percentage of participants achieving a complete response (CR), CR with incomplete neutrophil recovery (CRi), or a CR with incomplete platelet recovery (CRp), based on the 2003 revised IWG criteria for AML and 2006 modified IWG criteria for MDS per investigator review. CR: - ANC \> 1.0 x10⁹/L - Platelet count \> 100 x10⁹/L - Bone marrow (BM) blasts \< 5% - Absence of blasts with Auer rods - Independence of red cell transfusions CRi: - All CR criteria except for residual neutropenia (ANC \< 1.0 x 10⁹/L CRp: - All CR criteria except for residual thrombocytopenia (platelet counts \< 100 x 10⁹/L)

Analysis population: All participants who received at least one dose of study treatment in Phase 1 Dose Escalation. Participants were grouped according to assigned total daily dose.

Groups in this outcome

Phase 1 Dose Escalation: Enasidenib 50 mg

Participants received enasidenib 50 mg tablets QD on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 60 mg

Participants who received a total daily dose of enasidenib 60 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 75 mg

Participants who received a total daily dose of enasidenib 75 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 100 mg

Participants who received a total daily dose of enasidenib 100 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 150 mg

Participants who received a total daily dose of enasidenib 150 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 200 mg

Participants who received a total daily dose of enasidenib 200 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 300 mg

Participants who received a total daily dose of enasidenib 300 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 450 mg

Participants who received a total daily dose of enasidenib 450 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Escalation: Enasidenib 650 mg

Participants who received a total daily dose of enasidenib 650 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

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

Analysis denominator · Participants

GroupSource count
Phase 1 Dose Escalation: Enasidenib 50 mg9
Phase 1 Dose Escalation: Enasidenib 60 mg7
Phase 1 Dose Escalation: Enasidenib 75 mg7
Phase 1 Dose Escalation: Enasidenib 100 mg29
Phase 1 Dose Escalation: Enasidenib 150 mg13
Phase 1 Dose Escalation: Enasidenib 200 mg22
Phase 1 Dose Escalation: Enasidenib 300 mg14
Phase 1 Dose Escalation: Enasidenib 450 mg5
Phase 1 Dose Escalation: Enasidenib 650 mg7

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase 1 Dose Escalation: Enasidenib 50 mg22.2Not reported2.860.0Not reported
Phase 1 Dose Escalation: Enasidenib 60 mg14.3Not reported0.457.9Not reported
Phase 1 Dose Escalation: Enasidenib 75 mg0.0Not reported0.041.0Not reported
Phase 1 Dose Escalation: Enasidenib 100 mg31.0Not reported15.350.8Not reported
Phase 1 Dose Escalation: Enasidenib 150 mg15.4Not reported1.945.4Not reported
Phase 1 Dose Escalation: Enasidenib 200 mg18.2Not reported5.240.3Not reported
Phase 1 Dose Escalation: Enasidenib 300 mg28.6Not reported8.458.1Not reported
Phase 1 Dose Escalation: Enasidenib 450 mg40.0Not reported5.385.3Not reported
Phase 1 Dose Escalation: Enasidenib 650 mg57.1Not reported18.490.1Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        2.8
        upper Limit
        60.0
        value
        22.2
      2. group Id
        OG001
        lower Limit
        0.4
        upper Limit
        57.9
        value
        14.3
      3. group Id
        OG002
        lower Limit
        0.0
        upper Limit
        41.0
        value
        0.0
      4. group Id
        OG003
        lower Limit
        15.3
        upper Limit
        50.8
        value
        31.0
      5. group Id
        OG004
        lower Limit
        1.9
        upper Limit
        45.4
        value
        15.4
      6. group Id
        OG005
        lower Limit
        5.2
        upper Limit
        40.3
        value
        18.2
      7. group Id
        OG006
        lower Limit
        8.4
        upper Limit
        58.1
        value
        28.6
      8. group Id
        OG007
        lower Limit
        5.3
        upper Limit
        85.3
        value
        40.0
      9. group Id
        OG008
        lower Limit
        18.4
        upper Limit
        90.1
        value
        57.1
denoms
  1. counts
    1. group Id
      OG000
      value
      9
    2. group Id
      OG001
      value
      7
    3. group Id
      OG002
      value
      7
    4. group Id
      OG003
      value
      29
    5. group Id
      OG004
      value
      13
    6. group Id
      OG005
      value
      22
    7. group Id
      OG006
      value
      14
    8. group Id
      OG007
      value
      5
    9. group Id
      OG008
      value
      7
    units
    Participants
description
The percentage of participants achieving a complete response (CR), CR with incomplete neutrophil recovery (CRi), or a CR with incomplete platelet recovery (CRp), based on the 2003 revised IWG criteria for AML and 2006 modified IWG criteria for MDS per investigator review. CR: - ANC \> 1.0 x10⁹/L - Platelet count \> 100 x10⁹/L - Bone marrow (BM) blasts \< 5% - Absence of blasts with Auer rods - Independence of red cell transfusions CRi: - All CR criteria except for residual neutropenia (ANC \< 1.0 x 10⁹/L CRp: - All CR criteria except for residual thrombocytopenia (platelet counts \< 100 x 10⁹/L)
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received enasidenib 50 mg tablets QD on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG000
    title
    Phase 1 Dose Escalation: Enasidenib 50 mg
  2. description
    Participants who received a total daily dose of enasidenib 60 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG001
    title
    Phase 1 Dose Escalation: Enasidenib 60 mg
  3. description
    Participants who received a total daily dose of enasidenib 75 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG002
    title
    Phase 1 Dose Escalation: Enasidenib 75 mg
  4. description
    Participants who received a total daily dose of enasidenib 100 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG003
    title
    Phase 1 Dose Escalation: Enasidenib 100 mg
  5. description
    Participants who received a total daily dose of enasidenib 150 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG004
    title
    Phase 1 Dose Escalation: Enasidenib 150 mg
  6. description
    Participants who received a total daily dose of enasidenib 200 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG005
    title
    Phase 1 Dose Escalation: Enasidenib 200 mg
  7. description
    Participants who received a total daily dose of enasidenib 300 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG006
    title
    Phase 1 Dose Escalation: Enasidenib 300 mg
  8. description
    Participants who received a total daily dose of enasidenib 450 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG007
    title
    Phase 1 Dose Escalation: Enasidenib 450 mg
  9. description
    Participants who received a total daily dose of enasidenib 650 mg on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.
    id
    OG008
    title
    Phase 1 Dose Escalation: Enasidenib 650 mg
param Type
NUMBER
population Description
All participants who received at least one dose of study treatment in Phase 1 Dose Escalation. Participants were grouped according to assigned total daily dose.
reporting Status
POSTED
time Frame
Response assessments were performed every 28 days through Month 12 and every 56 days thereafter until the end of treatment; median duration of treatment exposure in the Phase 1 Dose Escalation was 5.0 months (range 0.4 to 34.2 months).
title
Phase 1 Dose Escalation: Rate of Complete Response and Complete Response With Incomplete Hematological Recovery (CR/CRi/CRp) by Total Daily Dose
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice