Actively Recruiting

Phase Not Applicable
Age: 12Months - 39Years
All Genders
NCT05057702

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

Led by University of California, San Francisco · Updated on 2026-04-15

74

Participants Needed

8

Research Sites

309 weeks

Total Duration

On this page

Sponsors

U

University of California, San Francisco

Lead Sponsor

W

Washington University School of Medicine

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 12Months - 39Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must have recurrent medulloblastoma or ependymoma confirmed by pathology and be experiencing their first or second relapse.
  • Participants must have disease accessible for surgery or biopsy with enough tissue expected for study.
  • Participants must have received at least one prior therapy at initial diagnosis.
  • Participants must have fully recovered from acute side effects of prior chemotherapy, immunotherapy, or radiotherapy before entering the study.
  • Participants must have had the last chemotherapy or biologic agent at least 7 days before registration.
  • Participants who received monoclonal antibody treatments must have had their last dose more than 21 days before registration.
  • Participants must have had the last fraction of local or craniospinal irradiation at least 12 weeks before registration.
  • Participants must be between 12 months and 39 years old.
  • Karnofsky score of 50 or higher for those over 16 years old, Lansky score of 50 or higher for 16 years or younger.
  • Participants on corticosteroids must be on a stable or decreasing dose for at least one week before registration.
  • Adequate bone marrow, kidney, and liver function as defined by specific laboratory values within 7 days before registration.
  • Women of childbearing potential and men must agree to use effective contraception during and for 4 months after therapy.
  • Participants with well-controlled seizure disorders may enroll.
  • Participants must enroll in the PNOC COMP study if it is open at the enrolling institution.
  • Participants or their legal guardians must be able to understand and sign informed consent or assent.
Not Eligible

You will not qualify if you...

  • Participants who had chemotherapy or radiotherapy within 3 weeks prior to study entry (6 weeks for nitrosoureas or mitomycin C) or who have not recovered from previous treatment side effects.
  • Participants receiving other investigational drugs.
  • Participants who completed biologic or small molecule agent therapy less than 7 days before enrollment or during adverse event risk periods.
  • Participants currently receiving any anti-cancer therapy other than steroids.
  • Participants with uncontrolled illness or active infections.
  • Women who are pregnant or breastfeeding; a negative pregnancy test is required before starting therapy.
  • Participants must not receive tumor-directed therapy after enrollment except surgery or biopsy.

AI-Screening

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Trial Site Locations

Total: 8 locations

1

Children's Hospital of Los Angeles

Los Angeles, California, United States, 90027

Actively Recruiting

2

Rady Children's Hospital

San Diego, California, United States, 92123

Actively Recruiting

3

University of California, San Francisco

San Francisco, California, United States, 94143

Actively Recruiting

4

Children's National Hospital

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

5

St. Louis Children's Hospital / Washington University in St. Louis

St Louis, Missouri, United States, 63130

Actively Recruiting

6

New York University

New York, New York, United States, 10016

Actively Recruiting

7

Columbia University Medical Center

New York, New York, United States, 10032

Actively Recruiting

8

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

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Research Team

P

PNOC Operation Office

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

OTHER

Number of Arms

2

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