Table 2.
Clinical Trials Involving Imipridonesa
| Clinical trials involving ONC201 | |||||
|---|---|---|---|---|---|
| Trial name | Phase, Status | Start – Est. completion date | Patient criteria | Publication of results (doi) | |
| CNS | Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma (NCT02525692) | 2, R | 1/2016-12/2020 | GBM, DMG | 10.1007/s11060-019-03271-3 |
| Young adult, adult (16+ yrs) | |||||
| Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline High Grade Gliomas (NCT03134131) | TNA | 04/2017 - | H3K27M-mutant glioma, grade III/IV midline glioma, DIPG | ||
| Child, adult, older adult (3+ yrs) | |||||
| ONC201 in Pediatric H3 K27M Gliomas (NCT03416530) | 1, R | 1/2018-1/2023 | DIPG, malignant glioma | ||
| Pediatric (2–18 years) | |||||
| ONC201 in Adults With Recurrent H3 K27M-mutant Glioma (NCT03295396) | 2, R | 10/2017-12/2022 | HGG, H3K27M-mutant | ||
| Adult (18+ yrs) | |||||
| A Phase II, Open Label Study of ONC201 in Adults with EGFR-low Glioblastoma (NCT04629209) | 2, NYR | 12/2020-12/2023 | EGFR-low glioblastoma | ||
| Adult (18+ yrs) | |||||
| A First-in-human Phase I Single-agent Dose-escalation, Food Effect and Dose Expansion Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms (NCT04541082) | 1, R | 9/2020-02/2025 | CNS neoplasms | ||
| Adult (18+ yrs) | |||||
| Solid | Oral ONC201 in Treating Patients With Advanced Solid Tumors (NCT02250781) | 1, C | 1/2015-10/2018 | Solid tumors | 10.1186/s40425-019-0599-8 |
| Adult (18+ yrs) | |||||
| Continuation of Oral ONC201 in Treating Patients With Advanced Solid Tumors (NCT02324621) | 1, C | 2/2015-10/2018 | Solid tumors | 10.1186/s40425-019-0599-8 | |
| Adult (18+ yrs) | |||||
| ONC201 in Recurrent or Metastatic Type II Endometrial Cancer Endometrial Cancer (NCT03485729) | 2, R | 3/2018-3/2021 | Recurrent endometrial cancer (female), Adult (18+ yrs) | ||
| Single Agent ONC201 in Recurrent or Metastatic Endometrial Cancer (NCT03099499) | 2, R | 6/2017-10/2022 | Endometrial cancer (female) Adult (18+ yrs) | ||
| BrUOG 379 Phase Ib/II Trial ONC201 + Nivolumab in MSS mCRC (379) (NCT03791398) | 1/2, R | 11/2019-12/2021 | Metastatic colorectal cancer Adult (18+ yrs) | ||
| Phase 2 Study of ONC201 in Neuroendocrine Tumors (NCT03034200) | 2, R | 8/2017–9/2020 | Recurrent or metastatic neuroendocrine tumor | ||
| Adult (18+ yrs) | |||||
| ONC201 in Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma (NCT03394027) | 2, R | 1/2018-12/2027 | Hormone receptor positive breast cancer (Cohort 1), TNBC (Cohort 2), Endometrial cancer (Cohort 3) | ||
| Adult (18+ yrs) | |||||
| ONC201 and Paclitaxel in Treating Patients with Platinum-Resistant Refractory or Recurrent Epithial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer (NCT04055649) | 2, NYR | 9/2019-6/2020 | Epithelial ovarian, fallopian tube, or primary peritoneal cancer, refractory disease; female; Adult (18+ yrs) | ||
| ONC201 With and Without Methionine-Restricted Diet in Patients With Metastatic Triple Negative Breast Cancer (NCT03733119) | 2, R | 11/2018-10/2021 | TNBC | ||
| Female, adult (17–64 yrs) | |||||
| Mixed | A Dose-Escalation Study of Onc201 Administered Every One or Three Weeks in Advanced Solid Tumors and Multiple Myeloma (NCT02609230) | 1, R | 11/2015-1/2022 | Advanced solid tumors, multiple myeloma | |
| Adult (18+ yrs) | |||||
| Hematologic | Akt/ERK Inhibitor ONC201 in Treating Patients With Relapsed or Refractory Non-Hodgkin’s Lymphoma (NCT02420795) | 1/2, R | 11/2015-11/2021 | Lymphoma | |
| Adult (18+ yrs) | |||||
| ONC 201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After Stem Cell Transplant (NCT03932643) | 1, R | 7/2019-7/2024 | AML, myelodysplastic syndromes; | ||
| Adult (19+ yrs) | |||||
| ONC201 in Relapsed/Refractory Acute Leukemias and High-Risk Myelodysplastic Syndromes (HR-MDS) (NCT02392572) | 1/2, ANR | 11/2018-11/2023 | Leukemia | ||
| Adult (18+ yrs) | |||||
| Oral ONC201 in Relapsed/Refractory Multiple Myeloma (NCT02863991) | 1/2, ANR | 1/2016-12/2021 | Multiple myeloma, relapsed/refractory | ||
| Adult (18+ yrs) | |||||
Abbreviations: DMG, midline glioma; DIPG, diffuse intrinsic pontine glioma; EGFR, epidermal growth factor receptor; TNBC, triple-negative breast cancer; AML, acute myeloid leukemia; HGG, high grade glioma; GBM, glioblastoma multiforme; CNS, central nervous system.
aThe table summarizes the current state of clinical trials involving the lead imipridones ONC201 (n = 19 clinical trials) and ONC206 (n = 1). Included are trials that are recruiting (R, n = 13), temporarily not available (TNA) (n = 1), completed (C, n = 2), not yet recruiting (NYR, n = 2), or active and not recruiting (ANR, n = 2), as registered on Clinicaltrials.gov. Trials are arranged by cancer type, and the trial phase, status, study start/estimated completion dates, and patient criteria are listed.