84 Participants Needed

ONC201 for Brain Tumor

Recruiting at 4 trial locations
SR
EM
DA
RP
Overseen ByRamage, PhD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ONC201, a new type of treatment that has shown promise in fighting various cancers, including a specific type of brain cancer, in patients who have this cancer returning.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it does mention that you cannot use certain drugs that affect liver enzymes (CYP3A4/5 inhibitors and inducers) during the study and shortly before starting it. It's best to discuss your current medications with the study team to see if any adjustments are needed.

What makes the drug ONC201 unique for treating brain tumors?

ONC201 is unique because it targets a specific pathway in cancer cells, potentially offering a new approach for treating brain tumors that are resistant to standard therapies. Unlike other treatments, it may work by inducing stress in tumor cells, leading to their death, and is being explored for its effectiveness in various types of brain tumors.12345

Are You a Good Fit for This Trial?

Inclusion Criteria

Unequivocal evidence of progressive disease on contrast-enhanced brain computerized tomography (CT) or magnetic resonance imaging (MRI) as defined by Response Assessment in Neuro-Oncology Criteria (RANO), or have documented recurrent glioblastoma on diagnostic biopsy.
Previous first line therapy with at least radiotherapy and temozolomide. For Arms D, E, and F, previous first line therapy with at least radiotherapy
Male or Female age ≥16 years.
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Exclusion Criteria

Pregnant women because ONC201 is novel agent with unknown potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ONC201, breastfeeding should be discontinued if the mother is treated with ONC201.
Known HIV-positive test on combination antiretroviral therapy.
Known history of cardiac arrhythmias including atrial fibrillation, tachyarrhythmias or bradycardia. Receiving therapeutic agents known to prolong QT interval will be excluded. History of CHF, or MI or stroke in the last 3 months will be excluded.
See 14 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 625 mg oral dordaviprone (ONC201) every 1 or 3 weeks depending on the arm, with clinical evaluations after each cycle

6 months
Every 1 or 3 weeks (in-person)

Neuroimaging

Neuroimaging studies are performed at baseline, 8 weeks from treatment initiation, and then every 8 weeks thereafter

8 weeks
Baseline, 8 weeks, then every 8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • ONC201
How Is the Trial Designed?
6Treatment groups
Experimental Treatment
Group I: Arm FExperimental Treatment1 Intervention
Group II: Arm EExperimental Treatment1 Intervention
Group III: Arm DExperimental Treatment1 Intervention
Group IV: Arm CExperimental Treatment1 Intervention
Group V: Arm BExperimental Treatment1 Intervention
Group VI: Arm AExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Chimerix

Lead Sponsor

Trials
42
Recruited
4,100+

Oncoceutics, Inc.

Industry Sponsor

Trials
10
Recruited
500+

Published Research Related to This Trial

A study of 159 patients with grade II/III gliomas revealed significant over-expression of specific tumor-associated antigens (BCAN, CSPG4, IGF2BP3, PTPRZ1, and TNC) at both mRNA and protein levels, indicating their potential as targets for immunotherapy.
In a smaller group of 27 patients, spontaneous T cell responses to the IMA950 antigens were detected in all grade II patients and 71% of grade III patients, suggesting that these antigens are relevant for effective tumor targeting and could enhance the efficacy of immunotherapy when combined with other peptides.
Antigenic expression and spontaneous immune responses support the use of a selected peptide set from the IMA950 glioblastoma vaccine for immunotherapy of grade II and III glioma.Dutoit, V., Migliorini, D., Ranzanici, G., et al.[2021]
In a case series of 4 patients with BRAF V600E primary brain tumors, dual therapy with dabrafenib and trametinib led to near-complete or complete clinical responses in three patients after 8 weeks, demonstrating significant efficacy.
The combination therapy not only showed greater effectiveness than dabrafenib alone but also helped reduce skin-related side effects, such as keratosis, highlighting its potential to improve patient tolerability during treatment.
Dual BRAF/MEK therapy in BRAF V600E-mutated primary brain tumors: a case series showing dramatic clinical and radiographic responses and a reduction in cutaneous toxicity.Bernstein, A., Mrowczynski, OD., Greene, A., et al.[2023]
The phase I trial involving 33 patients with IDH1(R132H)+ astrocytomas demonstrated that the IDH1-specific peptide vaccine was safe, with only grade 1 adverse events reported, and induced immune responses in 93.3% of participants.
Patients who developed immune responses to the vaccine had significantly better outcomes, with a two-year progression-free rate of 0.82, compared to those without immune responses, highlighting the vaccine's potential efficacy in treating this specific type of glioma.
A vaccine targeting mutant IDH1 in newly diagnosed glioma.Platten, M., Bunse, L., Wick, A., et al.[2022]

Citations

Antigenic expression and spontaneous immune responses support the use of a selected peptide set from the IMA950 glioblastoma vaccine for immunotherapy of grade II and III glioma. [2021]
Dual BRAF/MEK therapy in BRAF V600E-mutated primary brain tumors: a case series showing dramatic clinical and radiographic responses and a reduction in cutaneous toxicity. [2023]
A vaccine targeting mutant IDH1 in newly diagnosed glioma. [2022]
Dabrafenib Treatment in a Patient with an Epithelioid Glioblastoma and BRAF V600E Mutation. [2022]
INTERCEPT H3: a multicenter phase I peptide vaccine trial for the treatment of H3-mutated diffuse midline gliomas. [2023]
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