Recruiting
Phase 2

Observational Study

Sponsor:

UCLA

Code:

NCT06861244

Conditions

Embryonal Tumor With Multilayered Rosettes

Embryonal Tumor With Multilayered Rosettes, Nos

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Radiotherapy (RT)

Chemotherapy Drug, Cancer - Physician's Choice

Non-Investigational Surgical Resection

Temozolomide

Tumor Tissue Sample

Study Details

Brief summary:

This is an open-label, comprehensive, iterative investigation of evaluating the use of induction chemotherapy, high-dose chemotherapy, and focal radiation therapy in children with newly diagnosed Embryonal Tumor With Multilayered Rosettes (ETMR).

Conditions

Embryonal Tumor With Multilayered Rosettes

Embryonal Tumor With Multilayered Rosettes, Nos

Study ID

NCT06861244

Start date

Mar 6, 2025

Status verified date

Jun, 2026

Completion date

Mar 31, 2032

Anticipated

Primary completion date

Sep 30, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

The eligibility criteria listed below are interpreted literally and cannot be waived.

Inclusion Criteria:

1. Participants must have either a molecularly or histologically confirmed embryonal tumor with multilayered rosettes.
2. For enrollment, a confirmation of a minimum of 10-20 unstained formalin-fixed paraffin-embedded (FFPE) slides or 1 block (15-20 mg) with tumor content of 40% or greater is required. Anything less must be discussed and approved by the study chairs prior to enrollment.
3. Prior Therapy:

1. Cohort 1 participants must not have received any prior tumor-directed therapy other than surgical resection.
2. Cohort 2 and 3 participants may receive tumor-directed therapy prior to enrollment. These participants must be discussed with study chairs prior to enrollment.
4. Participants must not have received prior radiation for treatment of tumor.
5. Participants of any age are eligible.
6. Participants should begin induction chemotherapy within 28 days of the most recent definitive surgical procedure. Participants beginning therapy beyond 28 days from surgery, will need to discuss with study chairs.
7. Cohort specific eligibility

1. Cohort 1: Gross-total resection, Eligible for early radiotherapy (please see age criteria below), and no evidence of metastatic disease.
2. Cohort 2: Gross-total resection, high dose chemotherapy (please see age criteria below), and no evidence of metastatic disease.
3. Cohort 3A: Metastatic or residual disease, and early radiotherapy.
4. Cohort 3B: Metastatic or residual disease, and high dose chemotherapy.
5. Radiotherapy Age Criteria (at the time of planned radiation): >12 months of age for participants with infratentorial tumor OR >15 months of age for participants with supratentorial tumor. For participants being treated on radiotherapy-containing arms, the legal parent/guardian or patient and the physician must be willing to allow the use of radiotherapy for treatment.
8. Performance Score: 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.
9. Organ Function Requirements:

1. Peripheral absolute neutrophil count (ANC) > 75,000/mm3
2. Platelet count > 75,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 72 hours prior to enrollment).
10. Adequate Renal Function defined as:

a. Serum creatinine < 1.5 x upper limit normal (ULN) based on age and gender.
11. Adequate Liver Function defined as:

1. 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,
2. alanine aminotransferase (ALT) < 3 x ULN,
3. aspartate aminotransferase (AST) < 3 x ULN,
12. Adequate Neurologic Function defined as:

a. Participants with seizure disorder may be enrolled if well controlled. Participants on enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drugs.
13. As chemotherapeutic agents used in this trial are known to be teratogenic, women and men of child-bearing potential 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 study therapy. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
14. Participants must be enrolled on PNOC COMP prior to enrollment on PNOC031 if PNOC COMP is open to accrual at the enrolling institution.
15. A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.

Exclusion Criteria

1. Cohort 1 only: Participants who have received any prior tumor-directed therapy other than surgical intervention
2. Participants who are receiving any other tumor directed investigational agents.
3. History of allergic reactions attributed to compounds of similar chemical or biologic composition to the agents used in study.
4. Uncontrolled intercurrent illness.
5. Women of childbearing potential must not be pregnant or breast-feeding.

Study Design

Enrollment

70 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1: Gross-total resection, non-metastatic, early radiotherapy

Participants will undergo gross total resection of the tumor prior to enrollment into this cohort. Standard dose induction chemotherapy and 6-weeks of early focal radiotherapy, followed by and a second standard dose induction chemotherapy for a total of 12 weeks of chemotherapy; 18 weeks of treatments in all. Participants will be followed for up to 2 years.

experimental: Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy

Participants will undergo gross total resection of the tumor prior to enrollment into this cohort. Participants will receive 6 weeks of induction chemotherapy and 3 cycles (approximately 4 weeks each) of high-dose chemotherapy with stem cell rescue and will have the option to receive radiotherapy at the completion of therapy, for a total of 18-24 weeks. Participants will be followed for up to 2 years.

experimental: Cohort 3A: Metastatic or residual disease, early radiotherapy

Participants metastatic disease or residual disease following their initial surgical interventions prior to enrollment into this cohort will receive standard dose induction chemotherapy and 6-weeks of early focal radiotherapy, followed by and a second standard dose induction chemotherapy for a total of 12 weeks of chemotherapy; 18 weeks of treatments in all. Participants will be followed for up to 5 years.

experimental: Cohort 3B: Metastatic or residual disease, high-dose chemotherapy

Participants metastatic disease or residual disease following their initial surgical interventions prior to enrollment into this cohort. Participants will receive 6 weeks of induction chemotherapy and 3 cycles (approximately 4 weeks each) of high-dose chemotherapy with stem cell rescue and will have the option to receive radiotherapy at the completion of therapy, for a total of 18-24 weeks. Participants will be followed for up to 5 years.

Interventions

Radiotherapy (RT)

Undergo RT

Chemotherapy Drug, Cancer - Physician's Choice

One or more of the following may be assigned by the physician (physician's choice) per standard of care guidelines upon study enrollment following surgery: Cytarabine, Carboplatin, Cisplatin, Vincristine Sulfate injection (Vincristine PFS), Topotecan Hydrochloride, Dactinomycin, Thiotepa, Filgrastim, Cyclophosphamide, or Doxorubicin Hydrochloride. Not all participants will receive all possible drug regimens.

Non-Investigational Surgical Resection

Undergo surgery directly before study enrollment as part of planned care.

Temozolomide

Participants assigned to or whom receive optional RT will receive concurrent temozolomide

Tumor Tissue Sample

Tumor tissue will be collected for correlative studies

Blood Sample

Blood samples will be collected for correlative studies

Cerebrospinal Fluid (CSF) Sample

CSF samples will be collected for correlative studies

Primary outcome measure

  • Median Progression-free survival at 6 months (PFS6) (Cohort 1) [ Time Frame: Up to 6 months ]

Central Contacts and Locations

Central contacts

PNOC Operations Office

415-502-1600PNOC031@ucsf.edu

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

University of California, San Francisco

Recruiting

San Francisco, California, United States, 94143

Contacts

Principal Investigator:

Sabine Mueller, MD, Phd, MAS

Riley Hospital for Children at Indiana University Health

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21218

Contacts

Kenneth Cohen, MD, MBA

410-614-5055kcohen@jhmi.edu

Robyn Gartrell, MD, MS

410-955-2548rgartre1@jh.edu

Washington University in St. Louis

Recruiting

St Louis, Missouri, United States, 63130

Contacts

Michael Angelo Huang, MD

314-362-4563huangm@wustl.edu

Hackensack University Medical Center

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

More Information

Sponsor

University of California, San Francisco

Last update posted

Jun 29, 2026

Last verified

Jun, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by University of California, San Francisco on 2026-06-29.