Clinical Trials

Numerous Phase I through Phase III clinical trials evaluate the therapeutic profile and pharmacokinetics of enasidenib across diverse oncology settings. Sponsored by academic institutions and pharmaceutical partners, these active, recruiting, and completed studies focus on IDH2-mutated hematologic malignancies—including acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome, and myeloproliferative neoplasms—as well as select solid tumors such as glioma, intrahepatic cholangiocarcinoma, and chondrosarcoma.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03013998 RECRUITING
Previously Untreated Relapsed Refractory Acute Myeloid Leukemia
Beat AML, LLC
 2016-11 PHASE2; PHASE3
NCT02813135 RECRUITING
Pediatric Cancer
Gustave Roussy, Cancer Campus, Grand Paris
 2016-08-03 PHASE1; PHASE2
NCT05564390 RECRUITING
Acute Myeloid Leukemia; Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm; Acute Myeloid Leukemia Post Cytotoxic Therapy; Acute Myeloid Leukemia, Myelodysplasia-Related; Myelodysplastic Syndrome
National Cancer Institute (NCI)
 2024-06-18 PHASE2
NCT06240754 RECRUITING
Clonal Cytopenia of Undetermined Significance; CCUS Clonal Cytopenia of Undetermined Significance
Washington University School of Medicine
 2024-10-10 PHASE2
NCT06176989 RECRUITING
Metastatic Chondrosarcoma; Locally Advanced Chondrosarcoma; Metastatic Sinonasal Adenocarcinoma; Locally Advanced Sinonasal Adenocarcinoma; Metastatic Large-cell Neuroendocrine Carcinoma; Locally Advanced Large-cell Neuroendocrine Carcinoma; Metastatic Olfactory Neuroblastoma; Locally Advanced Olfactory Neuroblastoma; Metastatic Sinonasal Undifferentiated Carcinoma; Locally Advanced Sinonasal Undifferentiated Carcinoma
National Cancer Institute (NCI)
 2024-03-04 PHASE2
NCT06756308 ACTIVE_NOT_RECRUITING
T-cell Lymphoma
Memorial Sloan Kettering Cancer Center
 2024-12-24 PHASE2
NCT04774393 RECRUITING
Acute Myeloid Leukemia; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
M.D. Anderson Cancer Center
 2021-05-24 PHASE1; PHASE2
NCT05401097 RECRUITING
Acute Myeloid Leukemia
Alice Mims
 2022-09-13 PHASE2
NCT03683433 RECRUITING
Acute Bilineal Leukemia; Acute Biphenotypic Leukemia; Chronic Myelomonocytic Leukemia; IDH2 Gene Mutation; Myelodysplastic Syndrome; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
M.D. Anderson Cancer Center
 2018-09-18 PHASE2
NCT05756777 RECRUITING
Acute Myeloid Leukemia (AML)
Memorial Sloan Kettering Cancer Center
 2023-10-25 PHASE1
NCT03839771 ACTIVE_NOT_RECRUITING
Acute Myeloid Leukemia; Myelodysplastic Syndrome With Excess Blasts-2
Stichting Hemato-Oncologie voor Volwassenen Nederland
 2019-03-01 PHASE3
NCT06672146 RECRUITING
Acute Myeloid Leukemia
National Cancer Institute (NCI)
 2025-05-16 PHASE2
NCT06577441 RECRUITING
Myelodysplastic Syndrome
National Cancer Institute (NCI)
 2025-06-12 PHASE2
NCT03383575 RECRUITING
Acute Myeloid Leukemia; Blasts 20-30 Percent of Bone Marrow Nucleated Cells; Chronic Myelomonocytic Leukemia; IDH2 Gene Mutation; Myelodysplastic Syndrome With Excess Blasts; Recurrent High Risk Myelodysplastic Syndrome; Refractory High Risk Myelodysplastic Syndrome
M.D. Anderson Cancer Center
 2018-01-17 PHASE2
NCT03728335 SUSPENDED
Acute Myeloid Leukemia
City of Hope Medical Center
 2019-12-23 PHASE2
NCT05010772 RECRUITING
Acute Myeloid Leukemia
M.D. Anderson Cancer Center
 2021-10-25 PHASE1
NCT05441514 COMPLETED
Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
City of Hope Medical Center
 2023-04-28 PHASE1
NCT05102370 COMPLETED
Clonal Cytopenia of Undetermined Significance; CCUS Clonal Cytopenia of Undetermined Significance
Memorial Sloan Kettering Cancer Center
 2021-10-06 PHASE1
NCT04203316 ACTIVE_NOT_RECRUITING
Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
Children's Oncology Group
 2023-08-14 PHASE2
NCT03732703 COMPLETED
Relapsed Refractory Multiple Myeloma
Multiple Myeloma Research Consortium
 2019-04-01 PHASE1; PHASE2
NCT05282459 COMPLETED
Leukemia; Leukemia, Myeloid; Monocytic Leukemia
Tian Yi Zhang
 2022-01-12 PHASE1; PHASE2
NCT03825796 ACTIVE_NOT_RECRUITING
Recurrent Acute Myeloid Leukemia
Jonsson Comprehensive Cancer Center
 2019-04-12 PHASE2
NCT04522895 COMPLETED
Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia; IDH2 Gene Mutation; IDH2 R172; IDH2 R140
Heinrich-Heine University, Duesseldorf
 2020-08-27 PHASE2
NCT04655391 WITHDRAWN
Recurrent Acute Myeloid Leukemia
City of Hope Medical Center
 2022-06-25 PHASE1
NCT04092179 TERMINATED
Acute Myeloid Leukemia; Relapsed Cancer; Refractory Cancer; IDH2 Gene Mutation
University Health Network, Toronto
 2020-11-05 PHASE1; PHASE2
NCT03720366 COMPLETED
Leukemia, Myeloid, Acute
Celgene
 2018-10-04 PHASE1
NCT04955938 WITHDRAWN
IDH Mutation; IDH1 Mutation; IDH2 Gene Mutation; Blood Cancer; Myeloproliferative Neoplasm
University of Chicago
 2021-10-29 PHASE1
NCT04281498 COMPLETED
Accelerated/Blast-phase Myeloproliferative Neoplasm; Chronic-phase Myelofibrosis; IDH2 Mutation
John Mascarenhas
 2021-01-14 PHASE2
NCT04573582 COMPLETED
Hepatic Impairment
Celgene
 2020-11-13 PHASE1
NCT03744390 ACTIVE_NOT_RECRUITING
Myelodysplastic Syndromes; Leukemia Acute Myeloid
Groupe Francophone des Myelodysplasies
 2019-04-02 PHASE2
NCT04075747 COMPLETED
Acute Myeloid Leukemia
Jazz Pharmaceuticals
 2019-12-02 PHASE1
NCT03515512 COMPLETED
Acute Myeloid Leukemia; Chronic Myelomonocytic Leukemia
Massachusetts General Hospital
 2018-07-17 PHASE1
NCT03881735 WITHDRAWN
Blasts Under 5 Percent of Peripheral Blood White Cells; Bone Marrow Blasts Decreased by 50 Percent or More Compared to Pretreatment Level; IDH2 Gene Mutation; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
Roswell Park Cancer Institute
 2019-12-02 PHASE2
NCT04955938 Recruiting
IDH Mutation|IDH1 Mutation|IDH2 Gene Mutation|Blood Cancer|Myeloproliferative Neoplasm
University of Chicago
 2021-10-29 Phase 1
NCT03878524 TERMINATED
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Anatomic Stage IV Breast Cancer AJCC v8; Anemia; Ann Arbor Stage III Hodgkin Lymphoma; Ann Arbor Stage III Non-Hodgkin Lymphoma; Ann Arbor Stage IV Hodgkin Lymphoma; Ann Arbor Stage IV Non-Hodgkin Lymphoma; Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Castration-Resistant Prostate Carcinoma; Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Hematopoietic and Lymphoid System Neoplasm; Locally Advanced Pancreatic Adenocarcinoma; Metastatic Breast Carcinoma; Metastatic Malignant Solid Neoplasm; Metastatic Pancreatic Adenocarcinoma; Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis; Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Primary Myelofibrosis; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Lymphocytic Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Recurrent Hematologic Malignancy; Recurrent Hodgkin Lymphoma; Recurrent Myelodysplastic Syndrome; Recurrent Myelodysplastic/Myeloproliferative Neoplasm; Recurrent Myeloproliferative Neoplasm; Recurrent Non-Hodgkin Lymphoma; Recurrent Plasma Cell Myeloma; Recurrent Small Lymphocytic Lymphoma; Refractory Acute Lymphoblastic Leukemia; Refractory Acute Myeloid Leukemia; Refractory Chronic Lymphocytic Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Chronic Myelomonocytic Leukemia; Refractory Hematologic Malignancy; Refractory Hodgkin Lymphoma; Refractory Malignant Solid Neoplasm; Refractory Myelodysplastic Syndrome; Refractory Myelodysplastic/Myeloproliferative Neoplasm; Refractory Non-Hodgkin Lymphoma; Refractory Plasma Cell Myeloma; Refractory Primary Myelofibrosis; Refractory Small Lymphocytic Lymphoma; Stage II Pancreatic Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8; Stage IV Prostate Cancer AJCC v8; Unresectable Pancreatic Adenocarcinoma
OHSU Knight Cancer Institute
 2020-04-01 PHASE1
NCT02577406 COMPLETED
Leukemia, Myeloid; Isocitrate Dehydrogenase
Celgene
 2015-12-30 PHASE3
NCT04310527 COMPLETED
Healthy Volunteers
Celgene
 2019-10-09 PHASE1
NCT01915498 COMPLETED
Hematologic Neoplasms
Celgene
 2013-08-27 PHASE1; PHASE2
NCT02632708 ACTIVE_NOT_RECRUITING
Newly Diagnosed Acute Myeloid Leukemia (AML); Untreated AML; AML Arising From Myelodysplastic Syndrome (MDS); AML Arising From Antecedent Hematologic Disorder (AHD); AML Arising After Exposure to Genotoxic Injury
Institut de Recherches Internationales Servier
 2015-12-31 PHASE1
NCT03290443 COMPLETED
Hepatic Impairment
Celgene
 2017-09-21 PHASE1
NCT02677922 ACTIVE_NOT_RECRUITING
Leukemia, Myeloid, Acute
Celgene
 2016-06-03 PHASE1; PHASE2
NCT03515512 Completed
Acute Myeloid Leukemia|Chronic Myelomonocytic Leukemia
Massachusetts General Hospital|Celgene
 2018-07-17 Phase 1
NCT02273739 COMPLETED
Solid Tumor; Glioma; Angioimmunoblastic T-cell Lymphoma; Intrahepatic Cholangiocarcinoma; Chondrosarcoma
Celgene
 2014-12-08 PHASE1; PHASE2
NCT02443168 COMPLETED
Healthy Volunteers
Celgene
 2015-05-14 PHASE1
NCT02387866 COMPLETED
Healthy
Celgene
 2015-03-02 PHASE1
NCT02273739 Completed
Solid Tumor|Glioma|Angioimmunoblastic T-cell Lymphoma|Intrahepatic Cholangiocarcinoma|Chondrosarcoma
Celgene|Celgene Corporation
 2014-12-08 Phase 1|Phase 2
NCT02218346 COMPLETED
Healthy Volunteers
Agios Pharmaceuticals, Inc.
 2014-08 PHASE1
NCT02218346 Completed
Healthy Volunteers
Agios Pharmaceuticals Inc.|Celgene Corporation
 2014-08 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-09-03)

Check the AG-221 (Enasidenib) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Enasidenib (AG-221) selectively binds to mutant isocitrate dehydrogenase-2 (IDH2) enzymes, blocking the pathological conversion of alpha-ketoglutarate into the oncometabolite D-2-hydroxyglutarate (2-HG). This reduction in intracellular 2-HG restores normal histone and DNA demethylation, driving the differentiation of malignant hematopoietic progenitor cells and suppressing leukemic progression in patients with acute myeloid leukemia and related myeloid neoplasms.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.