Numerous clinical trials spanning Phase I through Phase III evaluate the efficacy, safety, and pharmacokinetics of ivosidenib (AG-120) across active, recruiting, pending, and completed statuses. Primary indications focus on IDH1-mutated hematologic malignancies and solid tumors—including acute myeloid leukemia, myeloproliferative neoplasms, chondrosarcoma, and glioma—as well as cohorts with hepatic impairment. Sponsors include commercial entities such as Servier and Agios Pharmaceuticals, alongside academic institutions like Memorial Sloan Kettering Cancer Center and the University of Chicago.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07745296 | NOT_YET_RECRUITING | Biliary Tract Cancer (BTC) |
UNICANCER |
2027-01-02 | PHASE3 |
| NCT03013998 | RECRUITING | Previously Untreated Relapsed Refractory Acute Myeloid Leukemia |
Beat AML, LLC |
2016-11 | PHASE2; PHASE3 |
| NCT07260175 | RECRUITING | Cholangiocarcinoma; IDH Mutation |
Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest |
2025-11-18 | PHASE2 |
| NCT07463768 | NOT_YET_RECRUITING | Acute Myeloid Leukemia |
French Innovative Leukemia Organisation |
2026-09 | PHASE2 |
| NCT05030441 | ACTIVE_NOT_RECRUITING | Clonal Cytopenia of Undetermined Significance |
Washington University School of Medicine |
2022-04-01 | PHASE2 |
| NCT06717958 | RECRUITING | AML; MDS |
Technische Universität Dresden |
2025-06-17 | PHASE2 |
| NCT06291987 | RECRUITING | Myeloproliferative Neoplasms |
University of Chicago |
2024-09-19 | PHASE1 |
| NCT07507760 | RECRUITING | Acute Myeloid Leukemia With Gene Mutations |
PETHEMA Foundation |
2026-06-12 | PHASE2 |
| NCT07075016 | RECRUITING | Acute Myeloid Leukemia |
Stichting Hemato-Oncologie voor Volwassenen Nederland |
2025-08-05 | PHASE3 |
| NCT07006688 | RECRUITING | IDH1-Mutated Malignancies |
Servier Bio-Innovation LLC |
2026-01-14 | PHASE1 |
| NCT04493164 | RECRUITING | Acute Myeloid Leukemia With Gene Mutations; Myelodysplastic Syndrome; Myeloproliferative Neoplasm; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2020-12-30 | PHASE2 |
| NCT07131059 | RECRUITING | AML, Adult |
Institute of Hematology & Blood Diseases Hospital, China |
2024-05-11 | |
| NCT06127407 | RECRUITING | Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen |
Servier Bio-Innovation LLC |
2024-07-09 | PHASE3 |
| NCT06265545 | RECRUITING | AML; Refractory; Relapsed |
Institute of Hematology & Blood Diseases Hospital, China |
2024-02-22 | |
| NCT06707493 | RECRUITING | IDH1 Mutation; Acute Myeloid Leukemia (AML); Hematopoietic Stem Cell Transplant (HSCT) |
Massachusetts General Hospital |
2026-01-16 | PHASE2 |
| NCT07392242 | RECRUITING | Acute Myeloid Leukemia |
Memorial Sloan Kettering Cancer Center |
2026-01-27 | PHASE2 |
| NCT06648473 | WITHDRAWN | Low Grade Glioma of Brain |
Duke University |
2026-01-01 | |
| NCT05876754 | ACTIVE_NOT_RECRUITING | Cholangiocarcinoma |
Servier Affaires Médicales |
2023-05-03 | PHASE3 |
| NCT04774393 | RECRUITING | Acute Myeloid Leukemia; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2021-05-24 | PHASE1; PHASE2 |
| NCT06465953 | RECRUITING | Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS); Myelodysplastic Syndromes (MDS) |
Institut de Recherches Internationales Servier |
2024-12-03 | PHASE3 |
| NCT05907057 | RECRUITING | Acute Myeloid Leukemia (AML) |
Servier Affaires Médicales |
2023-06-14 | PHASE3 |
| NCT03471260 | RECRUITING | Acute Myeloid Leukemia; Hematopoietic and Lymphoid System Neoplasm; Myelodysplastic Syndrome; Myeloproliferative Neoplasm; Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2018-03-19 | PHASE1; PHASE2 |
| NCT05401097 | RECRUITING | Acute Myeloid Leukemia |
Alice Mims |
2022-09-13 | PHASE2 |
| NCT05615818 | RECRUITING | Biliary Tract Neoplasms |
UNICANCER |
2024-07-18 | PHASE3 |
| NCT07548710 | RECRUITING | Acute Myeloid Leukemia |
Ruijin Hospital |
2026-05-01 | PHASE2 |
| NCT07607418 | RECRUITING | AML (Acute Myeloid Leukemia); MDS (Myelodysplastic Syndrome) |
Ruijin Hospital |
2026-05-22 | 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 |
| NCT03503409 | ACTIVE_NOT_RECRUITING | Myelodysplastic Syndromes; Acute Myeloid Leukemia |
Groupe Francophone des Myelodysplasies |
2019-05-14 | PHASE2 |
| NCT04278781 | ACTIVE_NOT_RECRUITING | Chondrosarcoma; Chondrosarcoma, Grade 2; Chondrosarcoma, Grade 3; IDH1 Gene Mutation |
Memorial Sloan Kettering Cancer Center |
2020-03-04 | PHASE2 |
| NCT05010772 | RECRUITING | Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2021-10-25 | PHASE1 |
| NCT02074839 | RECRUITING | Relapsed or Refractory Acute Myeloid Leukemia (AML); Untreated AML; Other IDH1-mutated Positive Hematologic Malignancies; Myelodysplastic Syndromes |
Institut de Recherches Internationales Servier |
2014-03-01 | PHASE1 |
| NCT06593548 | NOT_YET_RECRUITING | CRC; MSS; Metastatic Cancer |
Fudan University |
2024-11-01 | PHASE2 |
| NCT07282262 | RECRUITING | Biliary Tract Cancer |
Peking Union Medical College Hospital |
2024-05-01 | PHASE2 |
| NCT06611839 | RECRUITING | AML; IDH1 Mutation; Treatment |
Institute of Hematology & Blood Diseases Hospital, China |
2025-10-17 | PHASE1; PHASE2 |
| NCT06501625 | RECRUITING | Locally Advanced, Unresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation |
Institut de Recherches Internationales Servier |
2024-12-16 | PHASE1; PHASE2 |
| NCT05209074 | ACTIVE_NOT_RECRUITING | Pancreatic Ductal Adenocarcinoma; Resectable Pancreatic Cancer |
Case Comprehensive Cancer Center |
2022-09-08 | PHASE1 |
| NCT07007949 | RECRUITING | Acute Myeloid Leukemia; IDH1 Gene Mutation; Ivosidenib |
The First Affiliated Hospital of Soochow University |
2025-06-01 | PHASE2 |
| NCT03155620 | ACTIVE_NOT_RECRUITING | Advanced Malignant Solid Neoplasm; Ann Arbor Stage III Non-Hodgkin Lymphoma; Ann Arbor Stage IV Non-Hodgkin Lymphoma; Histiocytic Sarcoma; Juvenile Xanthogranuloma; Langerhans Cell Histiocytosis; Malignant Glioma; Recurrent Childhood Rhabdomyosarcoma; Recurrent Ependymoma; Recurrent Ewing Sarcoma; Recurrent Glioma; Recurrent Hepatoblastoma; Recurrent Langerhans Cell Histiocytosis; Recurrent Malignant Germ Cell Tumor; Recurrent Malignant Solid Neoplasm; Recurrent Medulloblastoma; Recurrent Neuroblastoma; Recurrent Non-Hodgkin Lymphoma; Recurrent Osteosarcoma; Recurrent Peripheral Primitive Neuroectodermal Tumor; Recurrent Primary Central Nervous System Neoplasm; Recurrent Rhabdoid Tumor; Recurrent Soft Tissue Sarcoma; Refractory Ewing Sarcoma; Refractory Glioma; Refractory Hepatoblastoma; Refractory Langerhans Cell Histiocytosis; Refractory Malignant Germ Cell Tumor; Refractory Malignant Solid Neoplasm; Refractory Medulloblastoma; Refractory Neuroblastoma; Refractory Non-Hodgkin Lymphoma; Refractory Osteosarcoma; Refractory Peripheral Primitive Neuroectodermal Tumor; Refractory Primary Central Nervous System Neoplasm; Refractory Rhabdoid Tumor; Refractory Rhabdomyosarcoma; Rhabdoid Tumor; Stage III Osteosarcoma AJCC v7; Stage III Soft Tissue Sarcoma AJCC v7; Stage IV Osteosarcoma AJCC v7; Stage IV Soft Tissue Sarcoma AJCC v7; Stage IVA Osteosarcoma AJCC v7; Stage IVB Osteosarcoma AJCC v7; Wilms Tumor |
National Cancer Institute (NCI) |
2017-07-31 | PHASE2 |
| NCT04195555 | ACTIVE_NOT_RECRUITING | Recurrent Ependymoma; Recurrent Ewing Sarcoma; Recurrent Hepatoblastoma; Recurrent Langerhans Cell Histiocytosis; Recurrent Malignant Germ Cell Tumor; Recurrent Malignant Glioma; Recurrent Malignant Solid Neoplasm; Recurrent Medulloblastoma; Recurrent Neuroblastoma; Recurrent Non-Hodgkin Lymphoma; Recurrent Osteosarcoma; Recurrent Peripheral Primitive Neuroectodermal Tumor; Recurrent Rhabdoid Tumor; Recurrent Rhabdomyosarcoma; Recurrent Soft Tissue Sarcoma; Recurrent WHO Grade 2 Glioma; Refractory Ependymoma; Refractory Ewing Sarcoma; Refractory Hepatoblastoma; Refractory Langerhans Cell Histiocytosis; Refractory Malignant Germ Cell Tumor; Refractory Malignant Glioma; Refractory Malignant Solid Neoplasm; Refractory Medulloblastoma; Refractory Neuroblastoma; Refractory Non-Hodgkin Lymphoma; Refractory Osteosarcoma; Refractory Peripheral Primitive Neuroectodermal Tumor; Refractory Rhabdoid Tumor; Refractory Rhabdomyosarcoma; Refractory Soft Tissue Sarcoma; Refractory WHO Grade 2 Glioma; Wilms Tumor |
National Cancer Institute (NCI) |
2020-07-20 | PHASE2 |
| NCT05921760 | TERMINATED | IDH1-mutant Cholangiocarcinoma |
Servier Bio-Innovation LLC |
2023-10-23 | PHASE1; PHASE2 |
| NCT06081829 | ACTIVE_NOT_RECRUITING | Cholangiocarcinoma Non-resectable; Cholangiocarcinoma Metastatic |
Servier |
2023-10-10 | PHASE2 |
| NCT06127407 | Not yet recruiting | Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation Untreated or Previously Treated With 1 Systemic Treatment Regimen |
Servier Bio-Innovation LLC|Institut de Recherches Internationales Servier|Servier |
2024-06-07 | Phase 3 |
| NCT06377579 | Not yet recruiting | AML Adult |
French Innovative Leukemia Organisation|Acute Leukemia French Association |
2024-05-15 | -- |
| NCT02073994 | COMPLETED | Cholangiocarcinoma; Chondrosarcoma; Glioma; Other Advanced Solid Tumors |
Institut de Recherches Internationales Servier |
2014-03-01 | PHASE1 |
| NCT04655391 | WITHDRAWN | Recurrent Acute Myeloid Leukemia |
City of Hope Medical Center |
2022-06-25 | PHASE1 |
| NCT04088188 | TERMINATED | Advanced Cholangiocarcinoma; Metastatic Cholangiocarcinoma; Unresectable Cholangiocarcinoma |
Academic and Community Cancer Research United |
2021-01-25 | PHASE1 |
| NCT06181734 | Recruiting | Acute Myeloid Leukemia (AML) |
iOMEDICO AG |
2023-12-20 | -- |
| NCT04056910 | COMPLETED | Advanced Solid Tumor; IDH1 Mutation; Glioma |
Jason J. Luke, MD |
2021-09-20 | PHASE2 |
| NCT04955938 | WITHDRAWN | IDH Mutation; IDH1 Mutation; IDH2 Gene Mutation; Blood Cancer; Myeloproliferative Neoplasm |
University of Chicago |
2021-10-29 | PHASE1 |
| NCT05907057 | Recruiting | Acute Myeloid Leukemia (AML) |
Servier Affaires Médicales|Servier |
2023-06-14 | Phase 3 |
| NCT03498521 | COMPLETED | Cancer of Unknown Primary Site |
Hoffmann-La Roche |
2018-07-10 | PHASE2 |
| NCT03564821 | COMPLETED | IDH1 Mutation Myeloid Neoplasms |
Massachusetts General Hospital |
2019-01-16 | PHASE1 |
| NCT04250051 | TERMINATED | Recurrent Acute Myeloid Leukemia; Recurrent Myelodysplastic Syndrome; Recurrent Myeloproliferative Neoplasm; Refractory Acute Myeloid Leukemia; Refractory Myelodysplastic Syndrome; Newly Diagnosed Acute Myeloid Leukemia (AML); IDH1 Mutation Myeloid Neoplasms; Acute Myeloid Leukemia (AML) |
Northwestern University |
2020-12-21 | PHASE1 |
| NCT04955938 | Recruiting | IDH Mutation|IDH1 Mutation|IDH2 Gene Mutation|Blood Cancer|Myeloproliferative Neoplasm |
University of Chicago |
2021-10-29 | Phase 1 |
| NCT04044209 | WITHDRAWN | Myelodysplastic Syndromes; Acute Myeloid Leukemia |
Yale University |
2020-02-12 | PHASE2 |
| NCT03173248 | ACTIVE_NOT_RECRUITING | Newly Diagnosed Acute Myeloid Leukemia (AML); Untreated AML; AML Arising From Myelodysplastic Syndrome (MDS); Leukemia, Myeloid, Acute |
Institut de Recherches Internationales Servier |
2017-06-26 | PHASE3 |
| NCT04176393 | COMPLETED | Relapsed or Refractory Acute Myeloid Leukemia |
CStone Pharmaceuticals |
2019-11-12 | PHASE1 |
| NCT03343197 | COMPLETED | Glioma |
Institut de Recherches Internationales Servier |
2018-03-20 | PHASE1 |
| NCT02989857 | COMPLETED | Advanced Cholangiocarcinoma; Metastatic Cholangiocarcinoma |
Institut de Recherches Internationales Servier |
2017-02-20 | PHASE3 |
| 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 |
| NCT02677922 | ACTIVE_NOT_RECRUITING | Leukemia, Myeloid, Acute |
Celgene |
2016-06-03 | PHASE1; PHASE2 |
| NCT03282513 | COMPLETED | Hepatic Impairment |
Agios Pharmaceuticals, Inc. |
2017-09-26 | PHASE1 |
| NCT03071770 | COMPLETED | Healthy |
Agios Pharmaceuticals, Inc. |
2017-03-31 | PHASE1 |
| NCT02831972 | COMPLETED | Healthy |
Agios Pharmaceuticals, Inc. |
2016-06 | PHASE1 |
| NCT02579707 | COMPLETED | Healthy |
Agios Pharmaceuticals, Inc. |
2015-10 | PHASE1 |
| NCT02489513 | COMPLETED | Healthy Volunteers |
Agios Pharmaceuticals, Inc. |
2015-06 | PHASE1 |
(data from https://clinicaltrials.gov, updated on 2026-08-04)
Check the
AG-120 (Ivosidenib)
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>).
- AG-120 (Ivosidenib) Mechanism of Action: IDH1 Inhibition in Leukemic Blast Cells Research
- AG-120 (Ivosidenib): Selectivity & Specificity
- AG-120 (Ivosidenib) Working Concentration | Cell Culture | Treatment
- AG-120 (Ivosidenib) Solubility in DMSO
- AG-120 (Ivosidenib) Stock Solution
- AG-120 (Ivosidenib) Storage
- AG-120 (Ivosidenib) Stability
- AG-120 (Ivosidenib) Combination Database
- AG-120 (Ivosidenib) Molecular Weight
- AG-120 (Ivosidenib) SMILES
- AG-120 (Ivosidenib) CAS Number
- AG-120 (Ivosidenib) Chemical Structure (2D and 3D)
- AG-120 (Ivosidenib) IC50 for Cell-based and Cell-free Assays
- AG-120 (Ivosidenib) Sigma (AMBH303C4A12)? Try a better option.
- AG-120 (Ivosidenib) APExBIO (B7805)? Try a better option.
- AG-120 (Ivosidenib) MedChemExpress (HY-18767)? Try a better option.
- AG-120 (Ivosidenib) Cayman (19894)? Try a better option.
- AG-120 (Ivosidenib) SDS｜MSDS
Note: Technical data last updated: Sep 1, 2026.