ONC201 Clinical Trials

We are actively recruiting patients for several clinical trials that are evaluating the activity of ONC201 to treat different types of cancers For more information about ONC201, how it works, and its efficacy that has been demonstrated to date, please refer to the Our Science section of this website.

BRAIN TUMORS WITH A FOCUS ON H3 K27M MUTATIONS

Massachusetts General Hospital / Dana Farber Cancer Institute / Miami Cancer Institute:

Phase II – Adults with recurrent Glioblastoma (GBM) (NCT02525692)

This Phase II clinical trial is investigating the use of ONC201 in patients with relapsed or refractory glioblastoma multiforme (GBM) as well as a specific cohort of patients with H3 K27M mutant gliomas. Isabel Arrillaga-Romany, MD, PhD, Associate Clinical Director of Neuro-Oncology at Massachusetts General Hospital, Patrick Wen, MD, Director of the Center for Neuro-Oncology at the Dana Farber Cancer Institute, and Yazmin Odia, MD at the Miami Cancer Institute are the Principal Investigators for the trial. The interim results from this Phase II study, including the first H3 K27M patient, can be found here.

New York University School of Medicine:

Phase II – Adults with Recurrent H3 K27M High-grade Glioma (NCT03295396)

The primary objective of this phase II trial is to determine the efficacy and safety of ONC201 in adult patients with recurrent high-grade glioma that have the H3 K27M mutation. Andrew S. Chi, MD PhD, Chief of Neuro-Oncology and the Codirector of the Brain Tumor Center at NYU Langone, is the Principal Investigator for the trial.

New York University Stephen D. Hassenfeld Children Center for Cancer and Blood Disorders:

Phase I – Pediatric Patients with H3 K27M High-Grade Gliomas Including Diffuse Intrinsic Pontine Gliomas (DIPG) (NCT: NCT03416530)

The primary objective of this phase I trial is to determine the efficacy and safety of ONC201 in pediatric patients with newly diagnosed or recurrent high-grade gliomas that exhibit the H3 K27M mutation.  Sharon Gardner, MD, pediatric hematologist/oncologist at the Stephen D. Hassenfeld Children Center for Cancer and Blood Disorders, is the Principal Investigator for this trial.

Expanded Access

Recurrent Histone H3 Mutant Glioma (NCT03134131)

The objective of this expanded access program is to provide ONC201 on a single patient compassionate use basis to eligible patients with recurrent gliomas that exhibit a missense histone H3 mutation and are unable to participate in the clinical trials listed above. The expanded access program is open to both adult and pediatric patients.

NEUROENDOCRINE TUMORS

Cleveland Clinic

Phase II – Adults with Neuroendocrine Tumors (NCT03034200)

This clinical trial is evaluating ONC201 in patients with recurrent neuroendocrine tumors. Many neuroendocrine tumors are known to overexpress DRD2, the target that ONC201 utilizes to drive its anti-cancer effects. Dr. Peter Anderson, MD, hematologist/oncologist at the Cleveland Clinic, is the Principal Investigator for the trial.

ENDOMETRIAL AND BREAST CANCER

Rutgers Cancer Institute of New Jersey

Phase II Study – Adults with Recurrent or Metastatic Type II Endometrial Cancer (NCT03485729)

This clinical trial is evaluating ONC201 in patients with metastatic or recurrent Type II endometrial cancer who failed at least 1 prior chemotherapy regimen. Dr. Lorna Rodriguez, MD, PhD at the Rutgers Cancer Institute of New Jersey is the Principal Investigator for the trial.

Fox Chase Cancer Center

Phase II – Adults with Endometrial Cancers (NCT03099499)

This clinical trial is evaluating ONC201 in patients with recurrent or metastatic endometrial cancer. Dr. Gina Mantia-Smaldone is the Principal Investigator for the trial.

National Cancer Institute (NCI)

Phase II – Adults with Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma (NCT03394027)

The Phase II trial is investigating the use of ONC201 in patients with metastatic breast cancer (hormone-positive or triple-negative) or metastatic endometrial cancers. Alexandra Zimmer, MD at the National Cancer Institute (NCI) is the Principal Investigator for this trial.

MULTIPLE MYELOMA

Icahn School of Medicine at Mount Sinai

Phase I/II Study – Adults with Relapsed and/or Refractory Multiple Myeloma (NCT03492138)

This clinical trial is evaluating ONC201 in patients with relapsed/refractory multiple myeloma in combination with Ixazomib and Dexamethasone. Dr. Ajai Chari, MD at the Icahn School of Medicine at Mount Sinai is the Principal Investigator of the trial.

Fox Chase Cancer Center and Mount Sinai Medical Center

Phase II – Adults with Multiple Myeloma (NCT02863991)

This clinical trial is evaluating ONC201 in patients with relapsed/refractory multiple myeloma. Carlyn Tan, MD at the Fox Chase Cancer Center, is the Principal Investigator for the trial. Dr. Ajai Chari is the Lead Investigator at the Mount Sinai Medical Center.

OTHER SOLID TUMORS

Fox Chase Cancer Center

Phase I – Adults with Solid Tumors (NCT02609230)

This clinical trial is evaluating the safety and pharmacodynamics of different ONC201 administration schedules in patients with advanced solid tumors. Anthony Olszanski, MD, Director of Clinical Drug Development at Fox Chase Cancer Center, is the Principal Investigator for the trial. 

Rutgers Cancer Institute of New Jersey

Phase I – Adults with Solid Tumors (NCT02250781)

The Phase I trial is investigating the use of ONC201 in patients with solid tumors not amenable to treatment with standard chemotherapy. The trial was originally being led by Mark Stein, MD, a medical oncologist and member of the Phase I/Investigational Therapeutics Program at the Cancer Institute of New Jersey. The current Principal Investigator of the study is Jyoti Malhotra, MD, MPH at Cancer Institute of New Jersey. This trial was the first in human trial with ONC201. The results from the initial Phase I portion can be found here.

LYMPHOMA

MD Anderson Cancer Center

Phase I/II – Adults with Lymphoma (NCT02420795)

The Phase I/II trial is investigating the use of ONC201 in patients with relapsed or refractory mantle cell lymphoma (MCL), cutaneous T-cell lymphoma (CTCL), diffuse large B-cell lymphoma (DLBCL) and transformed large cell lymphoma (TLCL). Michael Wang, MD, Professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center, is the Principal Investigator for the trial. This trial is conducted by MD Anderson as part of the alliance agreement between Oncoceutics and MD Anderson.

LEUKEMIA

MD Anderson Cancer Center

Phase I/II – Adults with Leukemia (NCT02392572)

The Phase I/II trial is investigating the use of ONC201 in patients with relapsed or refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS).  Gautam Borthakur, MD, Associate Professor in the Department of Leukemia at MD Anderson Cancer Center, is the Principal Investigator for the trial. This trial is conducted by MD Anderson as part of the alliance agreement between Oncoceutics and MD Anderson.