NCT01915498 · OUTCOME

Phase 1 Dose Expansion: Complete Response Rate

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 Phase 1 Dose Expansion was 5.2 months (range 0.5 to 32.8 months).

What was measured

Complete response rate is defined as the percentage of participants achieving a complete response (CR) based on the 2003 revised IWG criteria for AML or the 2006 modified IWG criteria for MDS as assessed by the investigator. CR for AML: - Absolute neutrophil count (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 CR for MDS: - Bone marrow: ≤ 5% myeloblasts with normal maturation of all cell lines - Peripheral blood: -- Hemoglobin ≥ 11 g/dL -- Platelets ≥ 100 × 10⁹/L -- Neutrophils ≥ 1.0 × 10⁹/L -- Blasts = 0%

Analysis population: All participants in Phase 1 Dose Expansion who received at least one dose of study treatment.

Groups in this outcome

Phase 1 Dose Expansion Arm 1: Enasidenib 100 mg QD

Participants ≥ 60 years old with relapsed, refractory AML or participants of any age if relapsed post-bone marrow transplant (BMT) received 100 mg enasidenib QD on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Expansion Arm 2: Enasidenib 100 mg QD

Participants \< 60 years old with R/R AML, excluding participants who relapsed post-BMT, received 100 mg enasidenib QD on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Expansion Arm 3: Enasidenib 100 mg QD

Untreated AML participants ≥ 60 years old who declined standard of care received 100 mg enasidenib QD on Day 1 to Day 28 of each 28-day treatment cycle until disease progression or development of unacceptable toxicity.

Phase 1 Dose Expansion Arm 4: Enasidenib 100 mg QD

Participants with advanced hematologic malignancies not eligible for Arms 1 to 3 received 100 mg enasidenib QD 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

Group	Source count
Phase 1 Dose Expansion Arm 1: Enasidenib 100 mg QD	49
Phase 1 Dose Expansion Arm 2: Enasidenib 100 mg QD	25
Phase 1 Dose Expansion Arm 3: Enasidenib 100 mg QD	25
Phase 1 Dose Expansion Arm 4: Enasidenib 100 mg QD	27

Reported measurements

Source class 1

Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Phase 1 Dose Expansion Arm 1: Enasidenib 100 mg QD	22.4	Not reported	11.8	36.6	Not reported
Phase 1 Dose Expansion Arm 2: Enasidenib 100 mg QD	0.0	Not reported	0.0	13.7	Not reported
Phase 1 Dose Expansion Arm 3: Enasidenib 100 mg QD	20.0	Not reported	6.8	40.7	Not reported
Phase 1 Dose Expansion Arm 4: Enasidenib 100 mg QD	22.2	Not reported	8.6	42.3	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
lower Limit
11.8
upper Limit
36.6
value
22.4
group Id
OG001
lower Limit
0.0
upper Limit
13.7
value
0.0
group Id
OG002
lower Limit
6.8
upper Limit
40.7
value
20.0
group Id
OG003
lower Limit
8.6
upper Limit
42.3
value
22.2

denoms

counts
group Id
OG000
value
49
group Id
OG001
value
25
group Id
OG002
value
25
group Id
OG003
value
27
units
Participants

description

dispersion Type

95% Confidence Interval

groups

description
Participants ≥ 60 years old with relapsed, refractory AML or participants of any age if relapsed post-bone marrow transplant (BMT) received 100 mg enasidenib 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 Expansion Arm 1: Enasidenib 100 mg QD
description
Participants \< 60 years old with R/R AML, excluding participants who relapsed post-BMT, received 100 mg enasidenib QD 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 Expansion Arm 2: Enasidenib 100 mg QD
description
Untreated AML participants ≥ 60 years old who declined standard of care received 100 mg enasidenib QD 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 Expansion Arm 3: Enasidenib 100 mg QD
description
Participants with advanced hematologic malignancies not eligible for Arms 1 to 3 received 100 mg enasidenib QD 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 Expansion Arm 4: Enasidenib 100 mg QD

param Type

NUMBER

population Description

All participants in Phase 1 Dose Expansion who received at least one dose of study treatment.

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 Phase 1 Dose Expansion was 5.2 months (range 0.5 to 32.8 months).

title

Phase 1 Dose Expansion: Complete Response Rate

type

SECONDARY

unit Of Measure

percentage of participants

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

Preserved source evidence · Not medical advice