Actively Recruiting
A Pilot Feasibility and Efficacy Trial of Real-Time Drug Screening and Genomic Testing to Create Individualized Treatment Plans 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
4 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
Researchers are evaluating a new treatment approach tailored to the molecular features of tumors in children and young adults with relapsed medulloblastoma or ependymoma. This pilot and efficacy trial aims to test if real-time drug screening of tumor tissue collected during surgery, combined with detailed genetic testing, can guide personalized treatment plans developed by a specialized tumor board. The goal is to offer each participant the most effective and least toxic FDA-approved therapies based on their tumor's unique characteristics, improving understanding and treatment of these tumors. Participants receive individualized treatment recommendations featuring up to four FDA-approved drugs chosen within 21 business days after tissue collection, based on results from drug screening, whole exome sequencing, and RNA sequencing. The trial started with a pilot phase to assess feasibility and has moved into an efficacy phase where treatment outcomes will be compared to historical data. Treatment can continue for up to two years or until disease progression, with the possibility of extended therapy if clinical benefits persist. Throughout the study, participants will be closely monitored with assessments including tumor and blood sample collections to study biomarkers, quality of life measures, and treatment-related side effects. The research team will track progression-free survival, overall survival, and treatment safety for up to five years from the start of therapy. Participants or their guardians will provide informed consent and may also take part in cognitive and quality of life studies as part of this comprehensive personalized medicine trial.
CONDITIONS
Brief Title
Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must have recurrent medulloblastoma or recurrent ependymoma confirmed by prior biopsy and be experiencing their first or second relapse.
- Participants must have surgically accessible tumors for resection or biopsy.
- Participants must have received at least one prior therapy for their diagnosis and fully recovered from acute toxic effects of prior treatments.
- Last chemotherapy or biologic agent must have been given at least 7 days before study entry; monoclonal antibody or bevacizumab treatments must be at least 21 days prior.
- Radiation therapy must have been completed at least 12 weeks before registration, with at least 14 days after local palliative radiation.
- Age must be between 1 month and 39 years.
- Performance status: Karnofsky score of 50 or higher if over 16 years old; Lansky score of 50 or higher if 16 or younger.
- Stable or decreasing corticosteroid dose for at least 1 week before registration.
- Adequate bone marrow, kidney, and liver function within 7 days before registration.
- Women of childbearing potential and men must agree to use contraception during and for 4 months after treatment.
- Participants with seizure disorders may enroll if seizures are well controlled.
- Participants must enroll in the cognitive and quality of life study if open at the institution.
- Ability to understand and sign informed consent and assent as appropriate.
You will not qualify if you...
- Chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to study entry or unresolved side effects from treatments given over 4 weeks ago.
- Receiving any other investigational agents.
- Less than 7 days since completion of therapy with biologic or small molecule agents or unresolved adverse events beyond expected time.
- Currently taking any anti-cancer direct therapy other than steroids.
- Uncontrolled illness including active infections.
- Pregnant or breastfeeding women; negative pregnancy test required before therapy.
- Receiving tumor-directed therapy other than surgery or biopsy after enrollment.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years or until disease progression
Participants receive an individualized treatment recommendation including a combination of up to four FDA-approved drugs based on real-time drug screening, whole exome sequencing, and RNA sequencing results, assigned by a specialized tumor board.
Visits as needed for treatment and monitoring during therapy
Duration - Up to 5 years
Participants are followed until disease progression, death, or up to 5 years from the start of therapy to monitor outcomes and safety.
Periodic visits for monitoring and assessments
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
Research Team
P
PNOC Operation Office
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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