ONC201 for Glioblastoma

Recruiting in Palo Alto (17 mi)

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Chimerix

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This is an open-label, two arm study. The trial will enroll a total of up to 36 patients. Arm A will enroll up to a total of 6 evaluable patients and Arm B will enroll up to a total of 30 evaluable patients. Arm A will explore the intra-tumoral ONC201 concentrations and pharmacodynamic activity in adult EGFR-low glioblastoma patients. Arm B will determine the radiographic efficacy of ONC201 in adult recurrent EGFR-low glioblastoma patients. All patients will be treated with oral ONC201 (625 mg) twice weekly, 2 consecutive days on and 5 days off per week schedule.

Eligibility Criteria

Inclusion Criteria

- leukocytes ≥ 3,000/mcL

Patient must have histologically confirmed World Health Organization Grade IV glioblastoma.

Patient must have all of the following in their most recently resected archival tumor tissue: (1) absence of EGFR gene amplification by FISH, (2) absence of EGFRvIII expression by RT-PCR, and (3) low EGFR expression by IHC.

+28 more

Exclusion Criteria

Patient has a known IDH1 (isocitrate dehydrogenase 1) or IDH2 mutations or 1p/19q co-deletion.

History of allergic reactions attributed to compounds of similar chemical or biologic composition to ONC201 or its excipients (See Section 8).

Presence of diffuse leptomeningeal disease or evidence of CSF dissemination.

+15 more

Participant Groups

2Treatment groups

Experimental Treatment

Group I: Arm B: ONC201 in GlioblastomaExperimental Treatment1 Intervention

Unequivocal evidence of recurrence (progressive disease) on contrast-enhanced brain CT or MRI as defined by RANO criteria, or have documented recurrent glioma on diagnostic biopsy. ONC201 will be administered orally, twice a week (2 consecutive days on and 5 days off per week) schedule at a dose of 625mg.

Group II: Arm A: ONC201 with Surgical Resection in GlioblastomaExperimental Treatment1 Intervention

Patients must be eligible for salvage surgical resection as deemed by the site Investigator. ONC201 will be administered orally, twice a week (2 consecutive days on and 5 days off per week) schedule at a dose of 625mg.