1. Patient must have glioma that is positive for the H3 K27M mutation (positive testing in CLIA laboratory) or have Grade III or IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord.
2. Patient must have had previous therapy that includes radiotherapy.
3. Patient must be at least 2 weeks from radiotherapy for DIPG, at least 4 weeks for non-DIPG glioma that has not recurred following prior treatment or recurrence is outside of high dose radiation field, and at least 12 weeks for non-DIPG glioma that has recurred following prior treatment.
4. Patient must be at least 3 years of age.
5. Patient must weigh at least 10kg.
6. Patient must be able to swallow capsules and retain orally administered medication.
7. From the projected start of scheduled study treatment, the following time periods must have elapsed from prior anti-cancer treatments: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide and 6 weeks from nitrosoureas), 6 weeks from anti-cancer antibodies (except 21 days for bevacizumab), or 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies.
8. Contrast-enhanced head CT or brain MRI within 21 days prior to start of study drug.
9. Adequate organ and marrow function as defined below: 1. Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1) 2. Hemoglobin >8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1 3. Total serum bilirubin <1.5 X upper limit of normal (ULN) 4. AST (SGOT)/ALT (SGPT) ≤2 X ULN; ≤ 5 X ULN if there is liver involvement secondary to tumor 5. Serum creatinine ≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)
10. For patients post pubertal: Female patients must agree to use effective contraception while taking ONC201 and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception while taking ONC201 and for at least 90 days after completion of treatment. The decision of effective contraception will be based on the judgment of the principal investigator.
11. Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the subjects age.
1. Qualifies for participation in an ongoing ONC201 clinical trial or is already participating in an ONC201 clinical trial.
2. Current or planned participation in a study of an investigational agent or using an investigational device.
3. More than two episodes of disease recurrence.
4. Evidence of diffuse leptomeningeal disease or CSF dissemination.
5. Any known systemic infection that, in the opinion of the investigator, could compromise the safety of the patient, while taking ONC201.
Philadelphia, Pennsylvania, 19104
Clinical Operations Oncoceutics
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.