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
Group
Value
95% CI
Enasidenib Mesylat 100mg
0
Enasidenib Mesylat 200mg
0
Related Adverse EventsSecondary· 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.
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.
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%.
Group
Value
95% CI
Enasidenib Mesylat 100 mg
0
Enasidenib Mesylat 200 mg
1
Red Blood Cell (RBC) Transfusion Independence (RBC TI)Secondary· 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.
Group
Value
95% CI
Enasidenib Mesylat 100 mg
0
Enasidenib Mesylat 200 mg
0
Adverse events — posted to ClinicalTrials.gov
Time frame: Informed consent through 90 days after last dose, an average of 9 months.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This is a phase 1b/2, open-label, single arm study to evaluate if enasidenib is safe and effective in improving anemia and decreasing transfusion needs in subjects diagnosed with lower risk myelodysplastic syndrome (MDS) or nonproliferative chronic myelomonocytic leukemia (CMML) without a mutation in isocitrate dehydrogenase type 2 (IDH2 wildtype). Other objectives include assessment of improvements in platelet production and characterization of the mechanism of action of enasidenib in enhancing endogenous erythropoiesis.
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07405424 — Spiritual Care Intervention in Adult Patients Diagnosed With Acute Leukemia
· NA
· recruiting
NCT07249346 — Dose-Expansion Study of Low Dose Post-Transplant Cyclophosphamide/Tacrolimus/Ruxolitinib for Graft-versus-Host Disease (
· Phase 2
· recruiting
NCT07566377 — Cord Blood Transplantation in Children and Young Adults With Blood Cancer
· Phase 2
· recruiting
NCT06856226 — Natural History Study to Determine Drug Metabolism Phenotype and Appropriate Germline Source DNA in Patients Undergoing
· recruiting
NCT07148180 — A Multi-Site Break Through Cancer Trial: Targeting Measurable Residual Disease in Patients With Acute Myeloid Leukemia:
· Phase 1, PHASE2
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Tian Yi Zhang
Last refreshed: 17 April 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05282459.