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Participants who will be undergoing surgery to remove their meningioma will receive two doses of ONC201 prior to their surgery. ONC201 is taken by mouth at 625 mg per dose. ONC201 will be taken once per week with the second dose taken approximately 24 hours prior to surgery.

Participants will receive one dose of ONC201 per week until progression. ONC201 is taken by mouth at 625 mg per dose.

ONC201 is an oral medication given once per week

ONC201

Erin Rogers, MS

Nicole Shonka, MD

The goal of this clinical trial is to learn about treatment for a type of brain tumor called a meningioma. This study will enroll two groups of people. One group will be for people who will receive surgery to remove their brain tumor. The other group will be for people who have previously received treatment for their brain tumor but do not have any other available options for treatment.

The primary goals of this study are:

1. To measure how much of the study drug is present in tumor tissue taken from patients during surgery to remove their brain tumor
2. To measure the length of time between a study participant's first dose of study treatment until the time when their brain tumor gets worse or their death

Patients will be enrolled in one of two arms, either the presurgical arm or the treatment arm. Patients who will be undergoing surgery to remove their meningioma will be enrolled to Arm I. Patients who have previously received treatment for their meningioma but have exhausted all reasonable treatment options will be enrolled to Arm II.

All patients will take the study medication, ONC201, by mouth once per week at a dose of 625 mg.

* Arm I patients will receive two doses of ONC201 prior to surgery, with the second dose occurring approximately 24 hours before surgery.
* Arm II patients will receive one 625 mg dose of ONC201 per week until disease progression or intolerable toxicity.

Primary Objectives

1. To evaluate the concentration of ONC201 in resected meningioma tissue (Arm I)
2. To measure progression-free survival in patients receiving ONC201 who have exhausted all other reasonable treatment options (Arm II)

Secondary Objectives

1. To evaluate tumor response via radiographic imaging following treatment with ONC201
2. To correlate dopamine receptor D2 (DRD2) expression with tumor response to treatment via molecular studies performed on archival and fresh tissue
3. To determine overall survival
4. To determine the efficacy of adding bevacizumab to ONC201 in patients who have progressed on ONC201 alone

A Study of ONC201 for Refractory Meningioma

A Pilot Study, Open-Label Study of ONC201 for Refractory Meningioma

Tumor tissue will be collected from Arm I participants at the time of surgical resection. The samples will be analyzed for the concentration of ONC201 in the tissue.

PFS at 6 months will be evaluated in participants assigned to Arm II. PFS is defined as the time from the start of study treatment to the time of disease progression or death. Participants without progression and who are alive at the time of last follow-up will be censored.

Tumor response from ONC201 on imaging will be measured by contrast-enhanced brain MRI completed every 12 weeks. Response will be determined using Macdonald criteria. The Macdonald criteria evaluates the status of lesions present on baseline imaging, the appearance of new lesions, corticosteroid use, and clinical status to determine the appropriate response category. The possible response categories include complete response, partial response, stable disease, and progressive disease.

Archival formalin-fixed paraffin embedded (FFPE) tumor tissue samples will be collected from Arm II participants at the time of study enrollment. The tissue samples will be tested for the level of DRD2 expression. The mechanism of action for the study medication, ONC201, is the through the inhibition of DRD2 receptors, a type of dopamine receptor. DRD2 expression will subsequently be correlated with best response and compared by response categories with Kruskal-Wallis test or Wilcoxon rank sum test, depending on the number of response levels.

Tumor response from ONC201 with bevacizumab on imaging will be measured by contrast-enhanced brain MRI completed every 12 weeks. Response will be determined using Macdonald criteria. The Macdonald criteria evaluates the status of lesions present on baseline imaging, the appearance of new lesions, corticosteroid use, and clinical status to determine the appropriate response category. The possible response categories include complete response, partial response, stable disease, and progressive disease.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Study of ONC201 for Refractory Meningioma	NCT06012929	Meningioma Refractory Meni... Relapsed Mening...	ONC201	19 Years -	University of Nebraska
A Study of ONC201 for Refractory Meningioma	NCT06012929	Meningioma Refractory Meni... Relapsed Mening...	ONC201	19 Years -	University of Nebraska
A Study of ONC201 for Refractory Meningioma	NCT06012929	Meningioma Refractory Meni... Relapsed Mening...	ONC201	19 Years -	University of Nebraska

Spots Global Cancer Trial Database for relapsed meningioma