A Pilot Feasibility and Efficacy (Phase 2) Trial of Real Time Drug Screening and Genomic Testing to Determine an Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma
University of California, San Francisco
Summary
The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in the pilot phase of performing real-time drug screening on tissue taken during surgery in patients with relapsed medulloblastoma or ependymoma and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed medulloblastoma and ependymoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer.
Description
This is a multi-center pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). Relapsed participants will receive an individualized treatment recommendation including up to four FDA-approved drugs based on the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing. PRIMARY OBJECTIVE: For pilot phase (CLOSED TO ENROLLMENT): I. To determine the feasibility of using the results of real-time in vitro drug screening, whole exome sequencing, and RNA sequencing of participant-derived specimens to guide treatment recommendations by a…
Eligibility
- Age range
- 1–39 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participants must have recurrent medulloblastoma or recurrent ependymoma previously histologically confirmed. Participants must be experiencing their first or second relapse to be eligible. 2. Participants must have surgically accessible disease. 3. Prior Therapy: 1. The participant must have received at least one prior therapy at the time of initial diagnosis. 2. Relapsed medulloblastoma or relapsed ependymoma are eligible. 3. Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to…
Interventions
- OtherSpecialized Tumor Board Treatment Plan
Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board
- OtherCombinations
Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board
Locations (8)
- Children's Hospital of Los AngelesLos Angeles, California
- Rady Children's HospitalSan Diego, California
- University of California, San FranciscoSan Francisco, California
- Children's National HospitalWashington D.C., District of Columbia
- St. Louis Children's Hospital / Washington University in St. LouisSt Louis, Missouri
- New York UniversityNew York, New York