Trial Outcomes & Findings for Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation (NCT NCT05282459)

Last Updated: 2026-04-17

Results Overview

Clinical response was assessed as the number of participants achieving a hematological improvement - erythroid (HI-E). Participants were characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows. * NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements * LTB = 0 units of RBC transfusions * HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

17 participants

Primary outcome timeframe

16 weeks

Results posted on

2026-04-17

Participant Flow

Participant milestones

Participant milestones
Measure	Enasidenib Mesylat 100mg Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Overall Study STARTED	3	14
Overall Study COMPLETED	2	8
Overall Study NOT COMPLETED	1	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Enasidenib Mesylat 100mg n=3 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=14 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.	Total n=17 Participants Total of all reporting groups
Age, Customized 18-29 years old	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Age, Customized 30-39 years old	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Age, Customized 40-49 years old	0 Participants n=130 Participants	1 Participants n=132 Participants	1 Participants n=130 Participants
Age, Customized 50-59 years old	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Age, Customized 60-69 years old	0 Participants n=130 Participants	1 Participants n=132 Participants	1 Participants n=130 Participants
Age, Customized 70-79 years old	3 Participants n=130 Participants	9 Participants n=132 Participants	12 Participants n=130 Participants
Age, Customized 80-89 years old	0 Participants n=130 Participants	3 Participants n=132 Participants	3 Participants n=130 Participants
Sex: Female, Male Female	0 Participants n=130 Participants	5 Participants n=132 Participants	5 Participants n=130 Participants
Sex: Female, Male Male	3 Participants n=130 Participants	9 Participants n=132 Participants	12 Participants n=130 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=130 Participants	14 Participants n=132 Participants	17 Participants n=130 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Race (NIH/OMB) Asian	0 Participants n=130 Participants	4 Participants n=132 Participants	4 Participants n=130 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Race (NIH/OMB) Black or African American	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Race (NIH/OMB) White	2 Participants n=130 Participants	9 Participants n=132 Participants	11 Participants n=130 Participants
Race (NIH/OMB) More than one race	0 Participants n=130 Participants	0 Participants n=132 Participants	0 Participants n=130 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=130 Participants	1 Participants n=132 Participants	2 Participants n=130 Participants
Region of Enrollment United States	3 participants n=130 Participants	14 participants n=132 Participants	17 participants n=130 Participants

PRIMARY outcome

Timeframe: 16 weeks

Outcome measures

Outcome measures
Measure	Enasidenib Mesylat 100mg n=2 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=8 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Clinical Response: Hematological Improvement - Erythroid (HI-E)	0 participants	0 participants

SECONDARY outcome

Timeframe: 12 months

Toxicity was assessed as the number of related non-serious adverse events and related serious adverse events (SAEs) reported by dose level (Cohort A or Cohort B) for the 12-cycle treatment period plus follow-up.

Outcome measures

Outcome measures
Measure	Enasidenib Mesylat 100mg n=3 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=14 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Related Adverse Events Related Serious Adverse Events	0 Adverse Events	0 Adverse Events
Related Adverse Events Related Non-Serious Adverse Events	20 Adverse Events	38 Adverse Events

SECONDARY outcome

Timeframe: 16 weeks

Population: No participants achieved Hematological Improvement - Erythroid (HI-E), and therefore no results could be calculated.

Time to hematological improvement - erythroid (HI-E) was assessed as the time from first dose of enasidenib to the first observed hemoglobin response. Participants will be characterized and stratified as non-transfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows. * NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements * LTB = 0 units of RBC transfusions * HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 16 weeks

Population: No participants achieved Hematological Improvement - Erythroid (HI-E), and therefore no results could be calculated.

Duration of Hematological Improvement - Erythroid (HI-E) will be assessed as the time from recorded response to loss of response. Participants will be characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows. NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements LTB = 0 units of RBC transfusions HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions The outcome will be reported as the number of participants that achieve the response, a number without dispersion.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 8 weeks

Clinical response for platelets was assessed as the number of participants achieving a hematological improvement - platelets (HI-P). Participants will be characterized and stratified as platelets \< or ≥ 20 x 10\^9/L, with response defined as follows. * \< 20 x 10\^9/L = increase in platelets from \< 20 x 10\^9/L to \> 20 x 10\^9/L AND by ≥ 100% * ≥ 20 x 10\^9/L = absolute increase in platelets of 30 x 10\^9/L The outcome will be reported as the number of participants that achieve the response, a number without dispersion.

Outcome measures

Outcome measures
Measure	Enasidenib Mesylat 100mg n=2 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=8 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Clinical Response: Hematological Improvement - Platelets (HI-P)	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 8 weeks

Clinical response for neutrophils was assessed as the number of participants achieving a hematological improvement - neutrophils (HI-N). Response was defined as an absolute increase in neutrophils \> 0.5 × 10\^9/L that was also an increase of ≥ 100%.

Outcome measures

Outcome measures
Measure	Enasidenib Mesylat 100mg n=2 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=8 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Clinical Response: Hematological Improvement - Neutrophils (HI-N)	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 12 months

Clinical response for red blood cells was assessed as the number of participants who were transfusion dependent that achieve red blood cell (RBC) transfusion independence (RBC TI) for for 8 weeks or longer.

Outcome measures

Outcome measures
Measure	Enasidenib Mesylat 100mg n=2 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=8 Participants Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Red Blood Cell (RBC) Transfusion Independence (RBC TI)	0 Participants	0 Participants

Adverse Events

Enasidenib Mesylat 100mg

Serious events: 3 serious events

Other events: 3 other events

Deaths: 1 deaths

Enasidenib Mesylat 200mg

Serious events: 7 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. Tian Yi Zhang

Stanford University

Phone: 650-497-6259

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Enasidenib Mesylat 100mg n=3 participants at risk Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily.	Enasidenib Mesylat 200mg n=14 participants at risk Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
Blood and lymphatic system disorders Anemia	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	14.3% 2/14 • Number of events 2 • Informed consent through 90 days after last dose, an average of 9 months
Blood and lymphatic system disorders Febrile Neutropenia	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Cardiac disorders Chest pain - cardiac	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months
Eye disorders Blurred vision	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Gastrointestinal disorders Duodenal Hemorrhage	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months
Gastrointestinal disorders Vomiting	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Gastrointestinal disorders Diarrhea	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Informed consent through 90 days after last dose, an average of 9 months
Gastrointestinal disorders Abdominal pain	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
General disorders Localized Edema	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
General disorders Fatigue	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
General disorders Volume overload	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Hepatobiliary disorders Ammonium level elevated	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Infections and infestations Sepsis	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months
Infections and infestations Lung Infection	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Infections and infestations Urinary tract infection	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Injury, poisoning and procedural complications Fall	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	14.3% 2/14 • Number of events 2 • Informed consent through 90 days after last dose, an average of 9 months
Injury, poisoning and procedural complications Hip Fracture	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months
Respiratory, thoracic and mediastinal disorders Pneumonitis	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 2 • Informed consent through 90 days after last dose, an average of 9 months
Skin and subcutaneous tissue disorders Skin Infection	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Nervous system disorders Syncope	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	7.1% 1/14 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months
Nervous system disorders Intracranial hemorrhage	33.3% 1/3 • Number of events 2 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months
Nervous system disorders Edema Cerebral	33.3% 1/3 • Number of events 2 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months
Musculoskeletal and connective tissue disorders Generalized Muscle Weakness	0.00% 0/3 • Informed consent through 90 days after last dose, an average of 9 months	14.3% 2/14 • Number of events 2 • Informed consent through 90 days after last dose, an average of 9 months
Vascular disorders Vasculitis	33.3% 1/3 • Number of events 1 • Informed consent through 90 days after last dose, an average of 9 months	0.00% 0/14 • Informed consent through 90 days after last dose, an average of 9 months