Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma

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.

Conditions

• Medulloblastoma
• Medulloblastoma, Childhood
• Medulloblastoma Recurrent
• Ependymoma
• Ependymoma Malignant
• Ependymoma of Brain

Interventions

OTHER

Specialized 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

OTHER

Combinations

Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board

Sponsors & Collaborators

• Washington University School of Medicine

  collaborator OTHER

• St. Baldrick's Foundation

  collaborator OTHER

• Ashion Analytics

  collaborator UNKNOWN

• University of Washington

  collaborator OTHER

• Pediatric Neuro-Oncology Consortium

  collaborator OTHER

• University of California, San Francisco

  lead OTHER

Principal Investigators

• Sabine Mueller, MD, PhD, MAS · University of California, San Francisco

• Robert Wechsler-Reya, PhD · Columbia University

• Margaret Shatara, MD · Children's Minnesota

• Megan Paul, MD · Rady Children's Hospital

• Lindsay Kilburn, MD · Children's National Research Institute

Study Design

Allocation

NON_RANDOMIZED

Purpose

OTHER

Masking

NONE

Model

SEQUENTIAL

Eligibility

Min Age

12 Months

Max Age

39 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2022-02-22

Primary Completion

2027-12-31

Completion

2028-01-31

Countries

• United States

Study Locations