Ultra-Rapid Drug Screening Guide for Relapsed Rare Brain Tumors

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

After surgical tissue is obtained, the trial performs rapid high-throughput drug screening and comprehensive molecular profiling to identify potential therapies. A specialized tumor board reviews the screening results, molecular data, prior treatments, and clinical context to issue a personalized management plan within about 28 days. The treating physician remains responsible for final treatment decisions, and additional molecular results may be incorporated as they become available. The goal is to give each patient the most effective and least toxic therapy based on their tumor’s unique biology and to establish a model for personalized medicine in rare CNS tumors.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. 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)
2. Participant must be a candidate for surgical resection or biopsy.

   • Participants must have surgically accessible disease.
3. 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
4. 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
5. Karnofsky 50 for participants \> 16 years of age and Lansky 50 for participants \<16 years of age
6. 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.
7. 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.
8. 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.
9. A legal parent or guardian must be able to understand and agree to sign a written informed consent and assent document, as appropriate.

Exclusion Criteria:

1. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.
2. 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.

Where it's running

• Children's National Hospital
  Washington D.C., District of Columbia, United States Recruiting

Study contacts

• Principal investigator: Adriana Fonseca, MD — Children's National Research Institute
• Study coordinator: Rachel Reed, MS, CCRP
• Email: rreed@childrensnational.org
• Phone: 202-476-5111

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Registry for rare brain tumors in children and young adults

  This study is collecting samples and health information from children and young adults with rare brain tumors to learn more about these conditions and improve treatment over the next 10 years.

  Recruiting at 13 sites Children's National Research Institute · Los Angeles, California and 12 other locations · NCT05697874

• GPC2-targeted CAR T cell therapy for relapsed or refractory medulloblastoma in children and young adults

  This trial will test whether autologous GPC2-targeted CAR T cells delivered into the brain ventricle are safe and active for children and young adults with relapsed or refractory medulloblastoma or similar CNS embryonal tumors.

  Recruiting Phase 1 Stanford University · Palo Alto, California · NCT07087002

• Botox to improve scar after cleft lip repair

  This study tests whether a Botox injection given during cleft lip repair in infants can lead to better-looking scars in the first year after surgery.

  Recruiting Phase 2 Children's National Research Institute · Washington D.C., District of Columbia · NCT07880860

• AI care summaries to help caregivers understand their child’s hospitalization

  This trial tests whether an AI-generated plain-language summary of inpatient notes helps caregivers of hospitalized children understand their child’s care better.

  Recruiting Children's National Research Institute · Washington D.C., District of Columbia · NCT07831018

• Blood-brain barrier opening with focused ultrasound plus T-cell therapy for young DMG patients

  This phase 1 study tests whether opening the blood-brain barrier with focused ultrasound and microbubbles can be safely combined with infusion of tumor-targeted T cells in children and young adults with newly diagnosed diffuse midline glioma.

  Recruiting Phase 1 Children's National Research Institute · Washington D.C., District of Columbia · NCT07796061

• Pupillary Dilation Reflex in Healthy Adults

  A single-site, non-interventional study will see how healthy adults’ pupils dilate when exposed to different levels of neurostimulation using the AlgometRx device.

  Recruiting Children's National Research Institute · Washington D.C., District of Columbia · NCT07746167

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.