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NCT01915498 · OUTCOME

Phase 1 Dose Escalation: Investigator Assessed Overall Response Rate (ORR) 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 dose escalation phase was 5.0 months (range 0.4 to 34.2 months).

What was measured

ORR is defined as the percentage of participants achieving an overall response of CR, CRi, CRp, PR, marrow CR (mCR) (for MDS) and MLFS (for AML) based on the 2003 revised IWG criteria for AML and 2006 modified IWG criteria for MDS. 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) PR: • Meets hematologic criteria of CR • Decrease of BM blasts to 5-25% and decrease of pretreatment BM blast ≥ 50%. MLFS: • Bone marrow blasts \< 5% • Absence of blasts with Auer rods • Absence of extramedullary disease • No hematologic recovery required mCR: • Bone marrow myeloblasts ≤ 5% and decreased by ≥ 50%

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 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 mg33.3Not reported7.570.1Not reported
Phase 1 Dose Escalation: Enasidenib 60 mg42.9Not reported9.981.6Not reported
Phase 1 Dose Escalation: Enasidenib 75 mg14.3Not reported0.457.9Not reported
Phase 1 Dose Escalation: Enasidenib 100 mg44.8Not reported26.464.3Not reported
Phase 1 Dose Escalation: Enasidenib 150 mg46.2Not reported19.274.9Not reported
Phase 1 Dose Escalation: Enasidenib 200 mg50.0Not reported28.271.8Not reported
Phase 1 Dose Escalation: Enasidenib 300 mg35.7Not reported12.864.9Not reported
Phase 1 Dose Escalation: Enasidenib 450 mg60.0Not reported14.794.7Not reported
Phase 1 Dose Escalation: Enasidenib 650 mg71.4Not reported29.096.3Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        7.5
        upper Limit
        70.1
        value
        33.3
      2. group Id
        OG001
        lower Limit
        9.9
        upper Limit
        81.6
        value
        42.9
      3. group Id
        OG002
        lower Limit
        0.4
        upper Limit
        57.9
        value
        14.3
      4. group Id
        OG003
        lower Limit
        26.4
        upper Limit
        64.3
        value
        44.8
      5. group Id
        OG004
        lower Limit
        19.2
        upper Limit
        74.9
        value
        46.2
      6. group Id
        OG005
        lower Limit
        28.2
        upper Limit
        71.8
        value
        50.0
      7. group Id
        OG006
        lower Limit
        12.8
        upper Limit
        64.9
        value
        35.7
      8. group Id
        OG007
        lower Limit
        14.7
        upper Limit
        94.7
        value
        60.0
      9. group Id
        OG008
        lower Limit
        29.0
        upper Limit
        96.3
        value
        71.4
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
ORR is defined as the percentage of participants achieving an overall response of CR, CRi, CRp, PR, marrow CR (mCR) (for MDS) and MLFS (for AML) based on the 2003 revised IWG criteria for AML and 2006 modified IWG criteria for MDS. 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) PR: • Meets hematologic criteria of CR • Decrease of BM blasts to 5-25% and decrease of pretreatment BM blast ≥ 50%. MLFS: • Bone marrow blasts \< 5% • Absence of blasts with Auer rods • Absence of extramedullary disease • No hematologic recovery required mCR: • Bone marrow myeloblasts ≤ 5% and decreased by ≥ 50%
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received enasidenib 50 mg 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 dose escalation phase was 5.0 months (range 0.4 to 34.2 months).
title
Phase 1 Dose Escalation: Investigator Assessed Overall Response Rate (ORR) by Total Daily Dose
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice