Actively Recruiting
PNOC031: Protocol for Embryonal Tumor With Multilayered Rosettes (ETMR)
Led by University of California, San Francisco · Updated on 2026-04-23
70
Participants Needed
7
Research Sites
78 weeks
Total Duration
On this page
Sponsors
U
University of California, San Francisco
Lead Sponsor
P
Pediatric Neuro-Oncology Consortium
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating a treatment approach for children with newly diagnosed Embryonal Tumor With Multilayered Rosettes (ETMR). This open-label study investigates the use of induction chemotherapy, high-dose chemotherapy, and focal radiation therapy to improve outcomes. The study focuses on participants with both gross-totally resected, non-metastatic tumors and those with incompletely resected or metastatic disease, aiming to better understand survival rates and disease progression. Participants will be assigned to one of four groups based on their surgical results and metastatic status. Those with gross total resection and no metastasis receive either standard induction chemotherapy with early focal radiotherapy or high-dose chemotherapy with stem cell rescue, optionally followed by radiotherapy. Participants with metastatic or residual disease undergo similar chemotherapy regimens with or without early radiotherapy. Treatments last between 12 to 24 weeks depending on the group, with follow-up periods ranging from two to five years. Throughout the study, participants will have tumor tissue, blood, and cerebrospinal fluid collected for research. They will be monitored for progression-free survival and overall survival, with assessments continuing up to two years for some groups and up to five years for others. The study also aims to validate blood and cerebrospinal fluid markers and explore the genetic makeup of ETMR. Safety and treatment response are tracked according to a Pediatric Neuro-Oncology Consortium protocol until withdrawal or death.
CONDITIONS
Brief Title
Embryonal Tumor With Multilayered Rosettes
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must have a molecularly or histologically confirmed embryonal tumor with multilayered rosettes.
- Tumor tissue for enrollment requires at least 10-20 unstained slides or one block with at least 40% tumor content.
- Cohort 1 participants must not have received any prior tumor-directed therapy other than surgery.
- Cohort 2 and 3 participants may have had prior therapy but need approval from study chairs.
- No prior radiation treatment for tumor is allowed.
- Participants of any age are eligible.
- Participants should start induction chemotherapy within 28 days of their most recent surgery.
- Cohort 1: gross-total resection, eligible for early radiotherapy, no metastatic disease.
- Cohort 2: gross-total resection, eligible for high-dose chemotherapy, no metastatic disease.
- Cohort 3A: metastatic or residual disease, early radiotherapy.
- Cohort 3B: metastatic or residual disease, high-dose chemotherapy.
- Radiotherapy age criteria: over 12 months for infratentorial tumor or over 15 months for supratentorial tumor.
- Karnofsky performance score 50 or higher for participants over 16 years old; Lansky score 50 or higher for participants 16 or younger.
- Adequate organ function with specified blood counts, renal, liver, and neurologic function.
- Participants with well-controlled seizure disorders may be eligible.
- Women and men of child-bearing potential must use effective contraception during and 4 months after study therapy.
- Participants must enroll in PNOC COMP if available before enrolling in this study.
- Legal guardian or participant must be able to provide informed consent or assent.
You will not qualify if you...
- Cohort 1 participants who have received prior tumor-directed therapy other than surgery.
- Participants receiving any other investigational tumor therapies.
- History of allergic reactions to similar drugs used in the study.
- Uncontrolled illnesses that could interfere with participation.
- Women who are pregnant or breastfeeding and of childbearing potential.
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 - Variable depending on recovery
Participants undergo tumor resection surgery as part of planned care before enrollment.
1 surgical hospitalization
Duration - 12 to 24 weeks depending on cohort
Participants receive induction chemotherapy and may receive early focal radiotherapy or high-dose chemotherapy depending on cohort assignment.
Multiple visits weekly during chemotherapy and radiotherapy
Duration - Up to 5 years
Participants are followed for up to 2 years (Cohorts 1 and 2) or up to 5 years (Cohorts 3A and 3B) to assess progression-free and overall survival.
Regular follow-up visits as per Pediatric Neuro-Oncology Consortium (PNOC) COMP protocol
Trial Site Locations
Total: 7 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
3
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States, 46202
Actively Recruiting
4
Johns Hopkins University
Baltimore, Maryland, United States, 21218
Actively Recruiting
5
Washington University in St. Louis
St Louis, Missouri, United States, 63130
Actively Recruiting
6
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Actively Recruiting
7
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
P
PNOC Operations Office
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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