Numerous clinical trials evaluate TIC10 (ONC201) across central nervous system malignancies—including glioblastoma, refractory meningioma, and H3 K27M-mutant diffuse intrinsic pontine glioma—and systemic cancers such as triple-negative breast, endometrial, mantle cell, and colorectal carcinomas. Spanning Phase 1 through Phase 3 studies and expanded access programs, these trials encompass active, completed, terminated, withdrawn, and approved marketing protocols. The portfolio is supported by academic, government, and pharmaceutical sponsors, including the National Cancer Institute, M.D. Anderson Cancer Center, Chimerix, and Jazz Pharmaceuticals.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT05476939 | RECRUITING | Diffuse Intrinsic Pontine Glioma; Diffuse Midline Glioma, H3 K27M-Mutant; Diffuse Midline Glioma, H3K27-altered |
Gustave Roussy, Cancer Campus, Grand Paris |
2022-09-29 | PHASE3 |
| NCT05580562 | RECRUITING | H3 K27M; Glioma |
Jazz Pharmaceuticals |
2023-01-23 | PHASE3 |
| NCT05542407 | RECRUITING | Endometrial Cancer; Metastasis; Endometrioid Endometrial Cancer; Carcinosarcoma; Serous Adenocarcinoma of Endometrium (Diagnosis); Clear Cell Endometrial Carcinoma; Endometrial Endometrioid Adenocarcinoma; Endometrial Mixed Cell Adenocarcinoma |
UNC Lineberger Comprehensive Cancer Center |
2023-10-23 | PHASE1 |
| NCT05009992 | RECRUITING | Diffuse Intrinsic Pontine Glioma; Diffuse Midline Glioma, H3 K27M-Mutant; Recurrent Diffuse Intrinsic Pontine Glioma; Recurrent Diffuse Midline Glioma, H3 K27M-Mutant; Recurrent WHO Grade III Glioma; WHO Grade III Glioma |
University of California, San Francisco |
2021-10-20 | PHASE2 |
| NCT04055649 | RECRUITING | Malignant Ovarian Epithelial Tumor; Platinum-Resistant Fallopian Tube Carcinoma; Platinum-Resistant Ovarian Carcinoma; Platinum-Resistant Primary Peritoneal Carcinoma; Recurrent Fallopian Tube Carcinoma; Recurrent Ovarian Carcinoma; Recurrent Primary Peritoneal Carcinoma; Refractory Fallopian Tube Carcinoma; Refractory Ovarian Carcinoma; Refractory Primary Peritoneal Carcinoma |
Ira Winer |
2020-01-21 | PHASE2 |
| NCT05630794 | RECRUITING | Colorectal Adenomatous Polyp; Colorectal Carcinoma; Familial Adenomatous Polyposis; Multiple Adenomatous Polyps |
National Cancer Institute (NCI) |
2025-10-06 | PHASE1 |
| NCT06012929 | WITHDRAWN | Meningioma; Refractory Meningioma; Relapsed Meningioma |
University of Nebraska |
2026-12 | PHASE2 |
| NCT02392572 | TERMINATED | Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Recurrent Myelodysplastic Syndrome; Refractory Acute Lymphoblastic Leukemia; Refractory Acute Myeloid Leukemia; Refractory Myelodysplastic Syndrome |
M.D. Anderson Cancer Center |
2015-11-03 | PHASE1; PHASE2 |
| NCT04854044 | WITHDRAWN | Recurrent Glioblastoma; Recurrent Gliosarcoma; Recurrent Supratentorial Glioblastoma; Supratentorial Gliosarcoma |
Jonsson Comprehensive Cancer Center |
2021-05-01 | PHASE1 |
| NCT03932643 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndromes |
University of Nebraska |
2019-07-30 | PHASE1 |
| NCT04629209 | WITHDRAWN | Glioblastoma |
Masonic Cancer Center, University of Minnesota |
2024-06-28 | PHASE2 |
| NCT04629209 | Withdrawn | Glioblastoma |
Masonic Cancer Center University of Minnesota|University of Minnesota |
2024-06-28 | Phase 2 |
| NCT06012929 | Not yet recruiting | Meningioma|Refractory Meningioma|Relapsed Meningioma |
University of Nebraska|Chimerix |
2024-04 | Phase 1 |
| NCT03034200 | COMPLETED | Recurrent Neuroendocrine Tumor; Metastatic Neuroendocrine Tumor |
Peter Anderson |
2017-08-02 | PHASE2 |
| NCT02525692 | TERMINATED | Glioblastoma; Diffuse Midline Glioma; H3 K27M Glioma; Thalamic Glioma; Infratentorial Glioma; Basal Ganglia Glioma |
Jazz Pharmaceuticals |
2016-01 | PHASE2 |
| NCT03485729 | TERMINATED | Endometrial Cancer Recurrent |
Jazz Pharmaceuticals |
2018-03-21 | PHASE2 |
| NCT03295396 | TERMINATED | Glioma |
Jazz Pharmaceuticals |
2017-10-30 | PHASE2 |
| NCT03416530 | TERMINATED | Diffuse Intrinsic Pontine Glioma; Glioma, Malignant |
Jazz Pharmaceuticals |
2018-01-25 | PHASE1 |
| NCT05476939 | Recruiting | Diffuse Intrinsic Pontine Glioma|Diffuse Midline Glioma H3 K27M-Mutant |
Gustave Roussy Cancer Campus Grand Paris|Chimerix|Innovative Therapies For Children with Cancer Consortium |
2022-09-29 | Phase 3 |
| NCT03791398 | TERMINATED | Metastatic Colorectal Cancer |
Brown University |
2019-11-15 | PHASE1; PHASE2 |
| NCT03394027 | COMPLETED | Triple Negative Breast Cancer; Endometrial Cancer; Hormone Receptor Positive, HER2 Negative Breast Cancer |
National Cancer Institute (NCI) |
2018-01-17 | PHASE2 |
| NCT03733119 | TERMINATED | Triple Negative Breast Cancer |
University of Wisconsin, Madison |
2018-11-13 | PHASE2 |
| NCT02420795 | TERMINATED | Central Nervous System Lymphoma; Gastric Mantle Cell Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Non-Hodgkin Lymphoma; Refractory Mantle Cell Lymphoma; Refractory Non-Hodgkin Lymphoma; Splenic Mantle Cell Lymphoma |
M.D. Anderson Cancer Center |
2015-11-03 | PHASE1; PHASE2 |
| NCT03099499 | TERMINATED | Endometrial Cancer |
Fox Chase Cancer Center |
2017-06-08 | PHASE2 |
| NCT02609230 | COMPLETED | Advanced Solid Tumors; Multiple Myeloma |
Fox Chase Cancer Center |
2015-11-05 | PHASE1 |
| NCT03492138 | TERMINATED | Multiple Myeloma |
Ajai Chari |
2018-03-26 | PHASE1; PHASE2 |
| NCT02863991 | TERMINATED | Multiple Myeloma |
Jazz Pharmaceuticals |
2017-04-19 | PHASE1; PHASE2 |
| NCT02324621 | COMPLETED | Unspecified Adult Solid Tumor |
Rutgers, The State University of New Jersey |
2015-02-20 | PHASE1 |
| NCT02250781 | COMPLETED | Solid Tumors |
Rutgers, The State University of New Jersey |
2015-01-12 | PHASE1 |
| NCT02038699 | WITHDRAWN | Advanced Glioblastoma; Advanced Colorectal Cancer; Advanced Triple-negative Breast Cancer; Advanced Non-small Cell Lung Cancer |
Jazz Pharmaceuticals |
2014-01 | PHASE1; PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-08-13)
Check the
TIC10 (ONC201)
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.