Expanded Access Program for ONC201 to Treat Recurrent Histone H3 K27M Glioma
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The objective of this expanded access program is to provide ONC201 on a single patient compassionate use basis to eligible adult subjects with recurrent glioma that exhibits a missense histone H3 mutation .
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Ages Eligible for Study:
18 Years and older (Adult, Senior)
Sexes Eligible for Study:
Confirmed evidence of glioma with disease progression from immediately prior therapy or refractory to the immediately prior treatment.
Evidence of the H3 K27M mutation detected in the tumor by a CLIA assay, either by IHC or DNA sequencing.
Male or Female age ≥18 years.
Karnofsky Performance Status (KPS) ≥ 50%
ECOG performance status ≤ 2.
Adequate organ and marrow function as defined below:
Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment.
Hemoglobin >8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to treatment.
Total serum bilirubin <1.5 X upper limit of normal (ULN)
AST (SGOT)/ALT (SGPT) ≤2 X ULN; ≤ 5 X ULN if there is liver involvement secondary to tumor.
Ability to understand and the willingness to sign a written informed consent document.
Ability to swallow capsules
Female patients must be surgically sterile or be postmenopausal, or must agree to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception during the period of the trial 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 or a designated associate.
Known active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV).
Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related illness.
Any of the following in the previous 6 months: myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack or symptomatic pulmonary embolism.
Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results, or in the judgment of the investigator would make the patient inappropriate for entry into the study.