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NCT05057702 · University of California, San Francisco

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

(PNOC027)

What this study is about

The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor.

View original scientific description

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.

Who can participate

This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.

Inclusion criteria

  • Participants must have recurrent medulloblastoma or recurrent ependymoma previously histologically confirmed. Participants must be experiencing their first or second relapse to be eligible.
  • Participants must have surgically accessible disease.
  • Prior Therapy:
  • The participant must have received at least one prior therapy at the time of initial diagnosis.
  • Relapsed medulloblastoma or relapsed ependymoma are eligible.
  • Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study and would be eligible for surgical resection per institutional guidelines
  • Participants must have received last chemotherapy or biologic agent at least 7 days prior to registration.
  • Monoclonal antibody treatment: \> 21 days prior to registration.
  • Bevacizumab participants must have received last dose \> 21 days prior to study registration
  • Participant must be a candidate for surgical resection or biopsy with anticipated ability to obtain the minimum tissue requirements for study.
  • Radiation - Participants must have:
  • Had their last fraction of local irradiation to primary tumor \>= 12 weeks prior to registration.
  • Had their last fraction of craniospinal irradiation or total body irradiation \>= 12 weeks prior to registration
  • At least 14 days after local palliative radiation (small-port)
  • Age \>=12 months to \<= 39 years of age.
  • Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>= 50 for participants \<= 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Corticosteroids: Participants who are receiving dexamethasone or equivalent must be on a stable or decreasing dose for at least 1 week prior to registration.
  • Organ Function Requirements (within 7 days prior to study registration)
  • Adequate Bone Marrow Function Defined as:
  • Peripheral absolute neutrophil count (ANC) \>= 750/mm\^3
  • Platelet count \>= 75,000/mm\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).
  • Hemoglobin \>= 8 g/dl.
  • Adequate Renal Function Defined as:
  • Creatinine clearance or radioisotope GFR \>= 70 milliliter/minute (mL/min) /1.73 m\^2 or
  • A serum creatinine based on age/sex as follows: Age / Maximum Serum Creatinine (mg/dL) Male / Maximum Serum Creatinine (mg/dL) Female.
  • 1 to \< 2 years / 0.6 / 0.6.
  • 2 to \< 6 years / 0.8 / 0.8.
  • 6 to \< 10 years / 1 / 1.
  • 10 to \< 13 years / 1.2 / 1.2.
  • 13 to \< 16 years / 1.5 / 1.4.
  • \>= 16 years / 1.7 / 1.4.
  • \- The threshold creatinine values in this table were derived from the Schwartz formula for estimating Glomerular filtration rate (GFR) utilizing child length and stature data published by the Center for Disease Control (CDC) (Schwartz GJ and Gauthier B 1985).
  • Adequate Liver Function Defined as:
  • Total Bilirubin \<= 1.5 x upper limit of normal (ULN) for age; in presence of Gilbert's syndrome, total bilirubin \< 3 x ULN or direct bilirubin \< 1.5 x ULN.
  • Alanine aminotransferase (ALT) \<= 3x ULN.
  • Aspartate aminotransferase (AST) \<= 3x ULN.
  • 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. Should a woman become pregnant or suspect pregnancy while participating in this study, the treating physician should be informed immediately.
  • Adequate neurologic function defined as participants with seizure disorder may be enrolled if seizures are well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug.
  • Participants must enroll on the Protocol for Children and Young Adults Diagnosed with a Central Nervous System (CNS) Tumor to Assess Cognitive, Quality of Life (QOL), and Comprehensive Effects of Therapies (PNOC COMP) study if PNOC COMP is open to accrual at the enrolling institution
  • A legal parent/guardian or participant must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.

Exclusion criteria

  • Participants who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
  • Participants who are receiving any other investigational agents.
  • Participants must be at least 7 days since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events that can occur beyond 7 days after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur. Such participants should also be discussed with study chairs.
  • Participants who are currently taking any anti-cancer direct therapy. Steroids are not considered anti-cancer therapy.
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.
  • 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.
  • Participants must not receive any tumor-directed therapy after enrollment, except for surgical resection/ biopsy. Important note: The eligibility criteria listed above are interpreted literally and cannot be waived.

Where

  • Los Angeles, California
  • San Diego, California
  • San Francisco, California
  • Washington D.C., District of Columbia
  • St Louis, Missouri
  • New York, New York
  • Philadelphia, Pennsylvania

Frequently asked questions

What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Data: ClinicalTrials.gov · synced Apr 15, 2026 · Source of record for eligibility and locations

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1 of 74 participants interested
1% interest

See if this study fits

A short prescreen based on this study's listed criteria. A coordinator confirms eligibility — this is not a medical assessment.

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Study locations

Choose your preferred location, or select flexible during enrollment.

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Los Angeles

California

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San Diego

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San Francisco

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Washington D.C.

District of Columbia

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St Louis

Missouri

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New York

New York

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New York

New York

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Philadelphia

Pennsylvania

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Express your interest

Share your contact details and a study coordinator can follow up about screening.

Secure & Confidential

Your information is protected and will only be shared with the research team.

What participation can include

  • Study-related care provided by the research team
  • Close monitoring by medical professionals
  • Possible compensation for time and travel*
  • The option to withdraw at any time
  • Contributing to medical research that may help future patients

*Compensation varies by study. Confirm details with coordinator.

Typical next steps

  1. 1.Submit this form
  2. 2.Phone screening
  3. 3.In-person assessment if eligible
  4. 4.Begin participation

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Looking for Medulloblastoma Treatment in Los Angeles?

Join others in California exploring innovative treatment options through clinical research

Medulloblastoma Treatment Options in Los Angeles, California

If you're searching for Medulloblastoma treatment in Los Angeles, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Los Angeles, San Diego, San Francisco and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Medulloblastoma. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 74 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Medulloblastoma?

Potential Benefits

  • Access to new treatment approaches before public availability
  • Close monitoring by experienced medical professionals
  • Study-related care provided at no cost
  • Contribute to medical research for Medulloblastoma

What to Expect

  • Initial screening to determine eligibility
  • Regular check-ups and monitoring visits
  • Possible compensation for time and travel
  • You can withdraw at any time

Frequently Asked Questions About This Medulloblastoma Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05057702. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.