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MedulloblastomaPNOC027NCT05057702

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

A study testing Specialized Tumor Board Treatment Plan and Combinations in people with Medulloblastoma and Medulloblastoma, Childhood, run by University of California, San Francisco at 9 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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… Read more about this study

Who is it for?
Ages 12 months to 39 years
What is being tested?
Specialized Tumor Board Treatment Plan and Combinations
Could I receive a placebo?
No, everyone receives an active treatment
How many people
74 participants
Where
9 sitesUnited States
Study dates
22 Feb 2022to 31 Jan 2028
Phase
Interventional
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 months to 39 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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
Show all 41Show fewer
  • 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.

Who cannot join

  • 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.

Trial locations and contacts

9 sites in 1 country. Contact the study team or a site near you to ask about taking part.

PNOC Operation Office contact

415-502-1600

PNOC027@ucsf.edu

9 sites in 1 country

United States9 sites, 9 recruiting

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027

    Ashley Margol, MD, 323-361-8147, amargol@chla.usc.edu

    Tom Davidson, MD, tdavidson@chla.usc.edu

    Recruiting

  • Children's Minnesota

    Minneapolis, Minnesota, 55404

    Anne Bendel, MD, 612-626-2778, anne.bendel@childrensmn.org

    Maggie Skrypek, MD, Mary.Skrypek@childrensmn.org

    Recruiting

  • New York University

    New York, New York, 10016

    Jessica Clymer, MD, 212-263-9949, Jessica.Clymer@nyulangone.org

    Recruiting

  • Columbia University Medical Center

    New York, New York, 10032

    James H Garvin Jr., MD, 212-305-9770, jhg1@cumc.columbia.edu

    James H Garvin Jr., MD

    Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Cassie Kline, MD, MAS, 267-426-9359 ext. x69359, klinec@chop.edu

    Jane Minturn, MD, PhD, 267-426-5026, minturn@chop.edu

    Recruiting

  • Rady Children's Hospital

    San Diego, California, 92123

    Jennifer Elster, MD, 858-576-1600, jelster@rchsd.org

    Megan Paul, MD, mpaul@rchsd.org

    Recruiting

  • University of California, San Francisco

    San Francisco, California, 94143

    PNOC Operations Office, 415-502-1600, PNOC027@ucsf.edu

    Sabine Mueller, MD, PhD

    Recruiting

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

    St Louis, Missouri, 63130

    Mohamed Abdelbaki, MD, 314-454-6018, MohamedA@wustl.edu

    Andrew Cluster, MD, acluster@wustl.edu

    Recruiting

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010

    Lindsay Kilburn, MD, 202-476-3854, lkilburn@childrensnational.org

    Roger Packer, MD, 202-476-5973, rpacker@cnmc.org

    Recruiting

Other trials in United States

About this study

Official title: 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

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.

Read the detailed 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):

  1. 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 specialized tumor board, in a clinically actionable timeframe, for children and young adults with recurrent medulloblastoma or ependymoma

For efficacy phase:

II. To determine the progression-free survival (PFS) in children and young adults with relapsed medulloblastoma and ependymoma who received the specialized tumor board recommendation and compare to historical cohorts.

SECONDARY OBJECTIVE:

For pilot (CLOSED TO ENROLLMENT) and efficacy phases:

  1. To determine the safety and describe the toxicity of treating children and young adults with relapsed medulloblastoma and ependymoma according to a specialized tumor board recommended treatment plan.

EXPLORATORY OBJECTIVES:

Pilot Phase (CLOSED TO ENROLLMENT):

  1. To estimate the objective response rate, progression free survival at 6 months (PFS-6) and overall survival (OS) of relapsed medulloblastoma in children and young adults treated with an individualized treatment regimen.

II. To assess Quality of Life (QOL) measures in participants with relapsed medulloblastoma treated with an individualized regimen.

III. To archive tumor and normal DNA from each participant along with serial blood draw following therapies as biospecimens for later studies to determine whether cfDNA sequences in the participant's blood serve as biomarkers of tumor burden, response to therapy, or development of drug resistance.

Efficacy Phase:

  1. To determine the overall survival (OS) and objective response rate (ORR) in children and young adults with relapsed medulloblastoma and ependymoma who received the specialized tumor board recommendation and compare to historical cohorts.

II. To generate PDX models of relapsed medulloblastoma and ependymoma to facilitate future in vivo testing of therapeutic agents.

III. To assess the correlation between available genomic data (WES and RNAseq) and patient outcomes, evaluating its potential complementary role in guiding therapeutic decisions alongside the primary drug screening approach.

IV. To archive tumor and normal DNA from each participant along with serial blood draw following therapies as biospecimens for later studies to determine whether cell free DNA (cfDNA) sequences in the participant's blood serve as biomarkers of tumor burden, response to therapy, or development of drug resistance.

  1. To assess patient and/or proxy satisfaction with study participation via patient-reported outcome (PRO) measures in the context of race ethnicity and other health related social risks.

VI. To assess on therapy toxicity in the context of race, ethnicity and other health related social risks.

Participants were initially enrolled in a pilot phase (CLOSED TO ENROLLMENT), and additional participants will be enrolled in the efficacy phase. Participants may continue treatment as tolerated for up to two years or until disease progression. After two years, patients may be eligible to continue therapy if they are experiencing clinical benefits, have disease control, and it is deemed appropriate to do so by their physician. Participants will be followed until progression, death, or up to 5 years from start of therapy.

Being tested
Specialized Tumor Board Treatment Plan, Combinations
Keywords
Individualized Treatment Plan, Genetic Screening, Screening
Medical subject headings
Medulloblastoma, Ependymoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Specialized Tumor Board Treatment PlanOther

    Also called Individualized Treatment Plan, Specialized 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

  • CombinationsOther

    Also called Multiple Treatments Physician Discretion

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

Study groups in NCT05057702
Study groupRoleWhat they receive
Individualized Treatment Recommendation (Pilot Phase)ExperimentalParticipants will receive an individualized treatment recommendation including a combination of up to four FDA-approved drugs within 21 business days of tissue acquisition using the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.
Receives: Other: Specialized Tumor Board Treatment Plan; Other: Combinations
Individualized Treatment Recommendation (Efficacy Phase)ExperimentalParticipants will receive an individualized treatment recommendation including a combination of up to four FDA-approved drugs within 21 business days of tissue acquisition using the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.
Receives: Other: Specialized Tumor Board Treatment Plan; Other: Combinations

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants for whom treatment recommendations are fully completed within 21 business days of tissue collection based on drug screening (Pilot Phase)

    Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days

    Measured: Up to 21 days

  2. Percentage of participants with treatment recommendations within 21 business days (Pilot Phase)

    Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days

    Measured: Up to 21 days

  3. Number of participants without adequate tissue

    The number of consented participants who do not have adequate tissue collection will be used to determine the feasibility.

    Measured: Up to 21 days

  4. Median Time from tissue collection to issued treatment plan from the specialized tumor board (Pilot Phase)

    Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days

    Measured: Up to 21 days

  5. Median Progression-free survival (PFS) (Efficacy Phase)

    The median months from the time of surgery for this recurrence to the first evidence of progression or death, using PFS at 10 months (PFS10) for the relapsed medulloblastoma cohort and PFS at 17 months for the relapsed ependymoma cohort (Arm B).

    Measured: Up to 5 years

Secondary outcomes (1)

  1. Proportion of participants with Adverse Events

    Proportion of participants with grade 3 or higher toxicities classified according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Adverse events and clinically significant laboratory abnormalities (meeting Grade 3 or higher criteria will be summarized by maximum grade and relationship to study drug(s).

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Sep 2021
  2. Study start22 Feb 2022
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion31 Jan 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Other
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
74 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05057702
Sponsor's protocol ID
21083
Other IDs
NCI-2021-09726
Acronym
PNOC027
Status last verified
Aug 2026
First submitted
15 Sep 2021
Last updated
11 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Individual participant data after de-identification.

Questions about this trial

What is this clinical trial studying?

A study testing Specialized Tumor Board Treatment Plan and Combinations in people with Medulloblastoma and Medulloblastoma, Childhood, run by University of California, San Francisco at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 41 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact PNOC Operation Office (415-502-1600, PNOC027@ucsf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants for whom treatment recommendations are fully completed within 21 business days of tissue collection based on drug screening (Pilot Phase) (time frame: Up to 21 days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05057702.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.

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