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Small Molecule

ONC201 for Brain Tumor

Phase 2

Waitlist Available

Research Sponsored by Chimerix

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

Study Summary

This trial will test a new drug, ONC201, which has shown to kill cancer cells in lab studies, on patients with recurrent glioblastoma or recurrent WHO Grade IV gliomas with the H3 K27M mutation.

Eligible Conditions

Glioblastoma
Brain Stem Glioma
Brain Tumor
Diffuse Midline Glioma
Thalamic Glioma

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Progression-free survival

Side effects data

From 2021 Phase 2 trial • 30 Patients • NCT03394027

60%

Fatigue

40%

Alkaline phosphatase increased

40%

Aspartate aminotransferase increased

40%

Lymphocyte count decreased

40%

Dizziness

30%

Alanine aminotransferase increased

30%

Diarrhea

20%

Hypophosphatemia

20%

Abdominal pain

20%

Anemia

20%

Nausea

20%

Dyspnea

10%

Lethargy

10%

Pain

10%

Paresthesia

10%

Sepsis

10%

Proteinuria

10%

Pleural effusion

10%

Vaginal discharge

10%

Vaginal hemorrhage

10%

Edema limbs

10%

Flu like symptoms

10%

Gastroesophageal reflux disease

10%

Headache

10%

Atrial flutter

10%

Cardiac arrest

10%

Dehydration

10%

Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Death

10%

Sinus tachycardia

10%

Stroke

10%

Thromboembolic event

10%

Urinary tract infection

10%

Anorexia

10%

Arthralgia

10%

Back pain

10%

Constipation

10%

Lymphedema

10%

Malaise

10%

Mucosal infection

10%

Myalgia

10%

Rash maculo-papular

10%

Rhinorrhea

10%

Vascular access complication

10%

Weight loss

10%

Cough

10%

Creatinine increased

100%

80%

60%

40%

20%

Study treatment Arm

Cohort 3-Endometrial Cancer (Female Only)

Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female)

Cohort 2-Triple Negative Breast Cancer (Male and Female)

Trial Design

6Treatment groups

Experimental Treatment

Group I: F: Non-H3 K27M Diffuse Midline Glioma ONC201 Q1WExperimental Treatment1 Intervention

Group II: E: Diffuse Midline Glioma Surgical Cohort ONC201 Q1WExperimental Treatment1 Intervention

Group III: D: H3 K27M Glioma ONC201 Q1WExperimental Treatment1 Intervention

Group IV: C: GBM Surgical Cohort ONC201 Q1WExperimental Treatment1 Intervention

Group V: B: GBM ONC201 Q1WExperimental Treatment1 Intervention

Group VI: A: GBM ONC201 Q3WExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ONC201

2017

Completed Phase 2

~60

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ChimerixLead Sponsor

41 Previous Clinical Trials

4,050 Total Patients Enrolled

4 Trials studying Glioblastoma

358 Patients Enrolled for Glioblastoma

Media Library

ONC201 (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT02525692 — Phase 2

Glioblastoma Research Study Groups: A: GBM ONC201 Q3W, B: GBM ONC201 Q1W, C: GBM Surgical Cohort ONC201 Q1W, D: H3 K27M Glioma ONC201 Q1W, E: Diffuse Midline Glioma Surgical Cohort ONC201 Q1W, F: Non-H3 K27M Diffuse Midline Glioma ONC201 Q1W

Glioblastoma Clinical Trial 2023: ONC201 Highlights & Side Effects. Trial Name: NCT02525692 — Phase 2

ONC201 (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02525692 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the current status of ONC201 in regards to federal regulation?

"ONC201's safety was rated a 2 by our team at Power. This is because, currently, there is only data supporting ONC201's safety and no efficacy data to support its use."

Answered by AI

How can people sign up for this experiment?

"Unfortunately, this study is no longer recruiting patients. Although the listing on clinicaltrials.gov was last updated on April 8th, 2022, the trial stopped enrolling new participants as of an unknown date. There are other 739 trials that might be a better match for you."

Answered by AI

Is this a new clinical trial?

"Oncoceutics, Inc. sponsored the first ONC201 clinical trial in 2015. This study completed Phase 1 & 2 drug approval and involved 120 patients. Since then, 4 more trials have been conducted for ONC201 across 29 cities in 4 countries."

Answered by AI

Is ONC201 a novel treatment option?

"The first study for ONC201 was at University of Texas MD Anderson Cancer Center in 2015. There have been 4 completed trials thus far, with 10 more clinical trials underway as of now. Many of these active studies are based in Boston, Massachusetts."

Answered by AI

Can you tell me how many different places this research is being conducted?

"There are 8 total locations for this trial, with sites in California, Florida, and Massachusetts. The specific hospitals enrolling patients are Massachusetts General Hospital in Boston, Dana-Farber Cancer Institute in Los Angeles, and Miami Cancer Institute in Miami."

Answered by AI

Recent research and studies

Journal of Neuro-OncologyJournal

Pediatric and Adult H3 K27m-mutant Diffuse Midline Glioma Treated with the Selective DRD2 Antagonist ONC201

Chi, Andrew S., Rohinton S. Tarapore, Matthew D. Hall, Nicole Shonka, Sharon Gardner, Yoshie Umemura, Ashley Sumrall, et al.. 2019. “Pediatric and Adult H3 K27m-mutant Diffuse Midline Glioma Treated with the Selective DRD2 Antagonist ONC201”. Journal of Neuro-oncology. Springer Science and Business Media LLC. doi:10.1007/s11060-019-03271-3.

Pharmacology Research & PerspectivesJournal

Antipsychotic Drugs Elicit Cytotoxicity in Glioblastoma Multiforme in a Calcium‐dependent, Non‐d <sub>2</sub> Receptor‐dependent, Manner

Weissenrieder, Jillian S., Jessie L. Reed, George‐Lucian Moldovan, Martin T. Johnson, Mohamed Trebak, Jeffrey D. Neighbors, Richard B. Mailman, and Raymond J. Hohl. 2021. “Antipsychotic Drugs Elicit Cytotoxicity in Glioblastoma Multiforme in a Calcium‐dependent, Non‐d 2 Receptor‐dependent, Manner”. Pharmacology Research & Perspectives. Wiley. doi:10.1002/prp2.689.

clinicaltrials.govStudy

Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma

2016. "Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02525692.