Clinical Trials
Several clinical trials are investigating the therapeutic potential of ONC206 across early-phase clinical evaluations. These Phase I and Phase II studies focus predominantly on central nervous system malignancies, including glioblastoma, diffuse midline glioma, and meningioma, alongside advanced pheochromocytoma and paraganglioma. Sponsored by academic institutions and pharmaceutical entities such as Chimerix, Jazz Pharmaceuticals, and the National Institutes of Health, these actively recruiting trials evaluate the safety, pharmacokinetics, and clinical efficacy of ONC206 in adult and pediatric populations.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07282587 | RECRUITING | Advanced Pheochromocytoma and Paraganglioma; PCPG |
Jazz Pharmaceuticals |
2026-01-02 | PHASE2 |
| NCT07533942 | RECRUITING | Meningioma |
Jazz Pharmaceuticals |
2026-07-16 | PHASE2 |
| NCT04732065 | RECRUITING | Diffuse Midline Glioma (DMG); Glioblastoma; Recurrent Ependymoma; Recurrent Malignant Central Nervous System Neoplasm; Spinal Cord Glioma; World Health Organization (WHO) Grade III Glioma; CNS Tumor; Central Nervous System Tumor |
Sabine Mueller, MD, PhD |
2021-08-23 | PHASE1 |
| NCT04541082 | RECRUITING | Central Nervous System Neoplasms; Glioblastoma; Gliosarcoma, Adult; Anaplastic Oligodendroglioma; Anaplastic Astrocytoma; Pilocytic Astrocytoma; Oligodendroglioma; Gliomatosis Cerebri; Pleomorphic Xanthoastrocytoma; Anaplastic Pleomorphic Xanthoastrocytoma; Diffuse Midline Glioma, H3 K27M-Mutant; Ependymoma; Ependymoma, Anaplastic; Medulloblastoma; Teratoid Rhabdoid Tumor; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors; Anaplastic Meningioma; Atypical Meningioma; Choroid Plexus Neoplasms; Pineal Tumor; Diffuse Astrocytoma; Glial Tumor |
Jazz Pharmaceuticals |
2020-10-26 | PHASE1 |
| NCT04541082 | Recruiting | Central Nervous System Neoplasms|Glioblastoma|Gliosarcoma Adult|Anaplastic Oligodendroglioma|Anaplastic Astrocytoma|Pilocytic Astrocytoma|Oligodendroglioma|Gliomatosis Cerebri|Pleomorphic Xanthoastrocytoma|Anaplastic Pleomorphic Xanthoastrocytoma|Diffuse Midline Glioma H3 K27M-Mutant|Ependymoma|Ependymoma Anaplastic|Medulloblastoma|Teratoid Rhabdoid Tumor|Neuroectodermal Tumors Primitive|Neuroectodermal Tumors|Anaplastic Meningioma|Atypical Meningioma|Choroid Plexus Neoplasms|Pineal Tumor|Diffuse Astrocytoma|Glial Tumor |
Chimerix|National Institutes of Health (NIH) |
2020-10-26 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2026-06-10)
Compliance for Clinical Use
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>).