Multiple clinical trials spanning Phase 1 through Phase 4 are evaluating olutasidenib across IDH1-mutated hematologic malignancies and solid tumors, including acute myeloid leukemia, myelodysplastic syndromes, and high-grade gliomas. Sponsored by pharmaceutical entities, academic medical centers, and federal research institutions, these studies encompass combination therapies, pediatric protocols, and drug interaction evaluations. Protocols across this active clinical landscape currently range from recruiting and active to completed or withdrawn.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07604064 | RECRUITING | Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia |
Kissei Pharmaceutical Co., Ltd. |
2026-06 | PHASE2 |
| NCT07153497 | NOT_YET_RECRUITING | Acute Myeloid Leukemia; Myelodysplastic Syndrome |
National Cancer Institute (NCI) |
2026-05-27 | PHASE2 |
| NCT06161974 | RECRUITING | High Grade Glioma; Astrocytoma; Astrocytoma, Grade III; Astrocytoma, Grade IV; Diffuse Intrinsic Pontine Glioma; WHO Grade III Glioma; WHO Grade IV Glioma; Metastatic Brain Tumor; Diffuse Midline Glioma, H3 K27M-Mutant; Thalamus Tumor; Spinal Tumor; IDH1 Mutation; IDH1 R132; IDH1 R132C; IDH1 R132H; IDH1 R132S; IDH1 R132G; IDH1 R132L; Oligodendroglioma |
Rigel Pharmaceuticals |
2025-02-01 | 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 |
| NCT06782542 | RECRUITING | Acute Myeloid Leukemia; IDH1 Mutation |
Justin Watts, MD |
2026-03-31 | PHASE2 |
| NCT07130695 | RECRUITING | Acute Myeloid Leukemia |
Virginia Commonwealth University |
2026-02-02 | PHASE1 |
| NCT07471841 | RECRUITING | IDH1 Mutation; Relapsed / Refractory AML |
Timothy Pardee |
2026-09 | PHASE2 |
| NCT06668584 | RECRUITING | Myeloid Malignancies |
M.D. Anderson Cancer Center |
2024-12-31 | PHASE2 |
| NCT07304011 | RECRUITING | Acute Myeloid Leukemia |
University of California, Davis |
2025-12-17 | PHASE2 |
| NCT06566742 | RECRUITING | Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia; Clonal Cytopenia of Undetermined Significance |
M.D. Anderson Cancer Center |
2024-12-10 | PHASE2 |
| NCT06445959 | RECRUITING | Mutant IDH1 Inhibitor Olutasidenib |
M.D. Anderson Cancer Center |
2024-08-29 | PHASE1; PHASE2 |
| NCT07032727 | RECRUITING | Targeted Therapy; IDH1-Mutated Malignancies; Mutations |
M.D. Anderson Cancer Center |
2025-09-12 | PHASE2 |
| NCT07486713 | RECRUITING | AML (Acute Myeloid Leukemia); Glioma; Cholangiocarcinoma; Solid Tumor Malignancies |
Rigel Pharmaceuticals |
2026-02-23 | PHASE4 |
| NCT06543381 | RECRUITING | Acute Myeloid Leukemia; Chronic Myelomonocytic Leukemia; Myelodysplastic Syndrome |
City of Hope Medical Center |
2025-01-17 | PHASE1 |
| NCT07411586 | ACTIVE_NOT_RECRUITING | Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2026-05-28 | PHASE1 |
| NCT06597734 | WITHDRAWN | Chronic Myelomonocytic Leukemia; Advanced Myeloproliferative Neoplasms; IDH1-mutated Higher-Risk Myelodysplastic Syndromes |
M.D. Anderson Cancer Center |
2025-01-28 | PHASE2 |
| NCT06161974 | Not yet recruiting | High Grade Glioma|Astrocytoma|Astrocytoma Grade III|Astrocytoma Grade IV|Diffuse Intrinsic Pontine Glioma|WHO Grade III Glioma|WHO Grade IV Glioma|Metastatic Brain Tumor|Diffuse Midline Glioma H3 K27M-Mutant|Thalamus Tumor|Spinal Tumor|IDH1 Mutation|IDH1 R132|IDH1 R132C|IDH1 R132H|IDH1 R132S|IDH1 R132G|IDH1 R132L|Oligodendroglioma |
Rigel Pharmaceuticals|Nationwide Children''s Hospital |
2024-06 | Phase 2 |
| NCT02719574 | COMPLETED | Acute Myeloid Leukemia; Acute Myelogenous Leukemia; Myelodysplastic Syndrome |
Forma Therapeutics, Inc. |
2016-04-30 | PHASE1; PHASE2 |
| NCT03684811 | COMPLETED | Cohort 1a and 1b: Glioma (Advanced Gliomas and Glioblastoma Multiforme); Cohort 2a and 2b: Hepatobiliary Tumors (Hepatocellular Carcinoma, Bile Duct Carcinoma, Intrahepatic Cholangiocarcinoma, Other Hepatobiliary Carcinomas); Cohort 3a and 3b: Chondrosarcoma; Cohort 4a and 4b: Intrahepatic Cholangiocarcinoma; Cohort 5a: Other Non-Central Nervous System Solid Tumors With IDH1 Mutations |
Forma Therapeutics, Inc. |
2018-11-01 | PHASE1; PHASE2 |
| NCT04013880 | WITHDRAWN | Acute Myeloid Leukemia; Myelodysplastic Syndrome; Recurrent Acute Myeloid Leukemia; Recurrent Myelodysplastic Syndrome; Refractory Acute Myeloid Leukemia; Refractory Myelodysplastic Syndrome |
Vanderbilt-Ingram Cancer Center |
2019-08-27 | PHASE1; PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-05-27)
Check the
Olutasidenib
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
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>).
Note: Technical data last updated: Sep 1, 2026.