Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors

Other illness

• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.

Lab values

• Women of childbearing potential must not be pregnant or breast-feeding. A negative serum or urine pregnancy test is required prior to start of therapy.

• Patients must have a relapse or recurrence of one of the following rare brain tumors:
  • CNS sarcoma (e.g. EWS, CIC, DICER1 altered)
  • BCOR altered tumors (e.g. BCOR ITD; BCOR, BCORL1, EP300, fused)
  • Mesenchymal tumors (e.g. FET::CREB altered tumors)
  • MN1::PATZ1 fused tumors
  • PLAG- altered tumors
  • Astroblastomas/MN-1 altered (e.g. EWSR2BEND2 altered)
  • Unclassifiable tumors
  • Other rare brain tumors: (e.g. recently described, poorly characterized, and/or ambiguous entities)
• Participant must be a candidate for surgical resection or biopsy.
  • Participants must have surgically accessible disease.
• Prior therapy
  • The participant must have a relapsed or recurrent rare brain tumor following at least one prior therapy for initial diagnosis or previous recurrence prior to study registration:
  • Surgery followed by observation.
  • Multi-modality therapy: surgery, radiation, and/or chemotherapy
• Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
  • Biologic agent: Participant must have recovered from any toxicity potentially related to the agent.
  • Last radiation at least 7 days prior to therapy start
• Karnofsky 50 for participants \> 16 years of age and Lansky 50 for participants \<16 years of age
• Corticosteroids: Subjects who are receiving dexamethasone or equivalent must be on a stable or decreasing dose for at least 1 week prior to therapy start.
• The effects of the agents used in this study on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 4 months after completion of therapy administration.
• Adequate neurologic function defined as:
  • Participants with seizure disorders may be enrolled if seizures are well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug.
• A legal parent or guardian must be able to understand and agree to sign a written informed consent and assent document, as appropriate.