Embryonal Tumor With Multilayered RosettesPhase 2PNOC031NCT06861244
Embryonal Tumor With Multilayered Rosettes
A Phase 2 study testing Radiotherapy (RT), Chemotherapy Drug, Cancer - Physician's Choice and Non-Investigational Surgical Resection in people with Embryonal Tumor With Multilayered Rosettes and Embryonal Tumor With Multilayered Rosettes, Nos, run by University of California, San Francisco at 8 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Embryonal Tumor With Multilayered Rosettes, Embryonal Tumor With Multilayered Rosettes, Nos
- What is being tested?
- Radiotherapy (RT), Chemotherapy Drug, Cancer - Physician's Choice and Non-Investigational Surgical Resection
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 8 sitesUnited States
- Study dates
- 6 Mar 2025to 31 Mar 2032
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants must have either a molecularly or histologically confirmed embryonal tumor with multilayered rosettes.
- 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.
- Prior Therapy:
- Cohort 1 participants must not have received any prior tumor-directed therapy other than surgical resection.
- Cohort 2 and 3 participants may receive tumor-directed therapy prior to enrollment. These participants must be discussed with study chairs prior to enrollment.
- Participants must not have received prior radiation for treatment of tumor.
Show all 29Show fewer
- Participants of any age are eligible.
- 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.
- Cohort specific eligibility
- Cohort 1: Gross-total resection, Eligible for early radiotherapy (please see age criteria below), and no evidence of metastatic disease.
- Cohort 2: Gross-total resection, high dose chemotherapy (please see age criteria below), and no evidence of metastatic disease.
- Cohort 3A: Metastatic or residual disease, and early radiotherapy.
- Cohort 3B: Metastatic or residual disease, and high dose chemotherapy.
- 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.
- 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.
- Organ Function Requirements:
- Peripheral absolute neutrophil count (ANC) > 75,000/mm3
- Platelet count > 75,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 72 hours prior to enrollment).
- Adequate Renal Function defined as:
- Serum creatinine < 1.5 x upper limit normal (ULN) based on age and gender.
- 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) < 3 x ULN,
- aspartate aminotransferase (AST) < 3 x ULN,
- Adequate Neurologic Function defined as:
- Participants with seizure disorder may be enrolled if well controlled. Participants on enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drugs.
- 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.
- Participants must be enrolled on PNOC COMP prior to enrollment on PNOC031 if PNOC COMP is open to accrual at the enrolling institution.
- A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.
Who cannot join
- Cohort 1 only: Participants who have received any prior tumor-directed therapy other than surgical intervention
- Participants who are receiving any other tumor directed investigational agents.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to the agents used in study.
- Uncontrolled intercurrent illness.
- Women of childbearing potential must not be pregnant or breast-feeding.
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 8 recruiting
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21218
Kenneth Cohen, MD, MBA, 410-614-5055, kcohen@jhmi.edu
Robyn Gartrell, MD, MS, 410-955-2548, rgartre1@jh.edu
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Girish Dhall, MD, 205-638-6194, gdhall@uabmc.edu
Laura K Metrock, MD, 205-638-9285, kmetrock@peds.uab.edu
- Recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Derek Hanson, MD, 551-996-5437, derek.hanson@hmhn.org
Katharine Offer, MD, 551-996-5437, katharine.offer@hmhn.org
- Recruiting
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
Brian Weiss, MD, 317-944-8784, weissbd@iu.edu
Alex Lion, DO, 317-944-8784, alion@iu.edu
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Emily Hanzlik, MD, 901-595-2615, emily.hanzlik@stjude.org
Amar Gajjar, MD, 901-595-2615, Amar.Gajjar@stjude.org
- Recruiting
University of Utah
Salt Lake City, Utah, 84113
Nicholas Whipple, MD, MPH, 801-662-4700, nicholas.whipple@hsc.utah.edu
Priya Chan, MD, 801-662-4700, priya.chan@hsc.utah.edu
- Recruiting
University of California, San Francisco
San Francisco, California, 94143
PNOC Operations Office, 415-502-1600, PNOC031@ucsf.edu
877-827-3222, cancertrials@ucsf.edu
- Recruiting
Washington University in St. Louis
St Louis, Missouri, 63130
Michael Angelo Huang, MD, 314-362-4563, huangm@wustl.edu
Mohamed AbdelBaki, MD, (314) 454-6018, Mohameda@wustl.edu
About this study
Official title: PNOC031: Protocol for Embryonal Tumor With Multilayered Rosettes (ETMR)
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).
Read the detailed description
PRIMARY OBJECTIVES I. To determine the six-month progression-free survival (PFS6) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR, treated using a regimen of induction chemotherapy and early focal radiotherapy (Cohort 1)
SECONDARY OBJECTIVES I. To determine the two-year progression-free survival (PFS) and overall survival (OS) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR (Cohort 1).
II. To determine the two-year progression-free survival (PFS) and overall survival (OS) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR (Cohort 2).
III. To determine the two-year progression-free survival (PFS), overall survival (OS) and objective response rate of participants with newly diagnosed, incompletely resected and/or metastatic ETMR (Cohort 3A and 3B)
EXPLORATORY OBJECTIVES:
- To validate the utility of a liquid miRNA biomarker in blood and Cerebral spinal fluid (CSF) as a correlative marker of a participant's disease status.
II. To better define the genomic landscape of ETMR.
OUTLINE:
Participants with newly diagnosed ETMR will obtain either gross total, or sub-total resection surgery prior to enrollment. After surgery, participants will be assigned to 1 of 4 possible cohorts:
Cohorts 1 and 2: Participants with newly diagnosed, gross-totally resected, non-metastatic ETMR.
Cohorts 3A and 3B: Participants with newly diagnosed, incompletely resected and/or metastatic ETMR.
Participants will be assessed for survival outcomes for up to 2 years. Follow-up procedures are to be captured under the PNOC COMP protocol. Participants will be followed under the Pediatric Neuro-Oncology Consortium (PNOC) COMP protocol until death or withdrawal from study.
- Conditions
- Embryonal Tumor With Multilayered Rosettes, Embryonal Tumor With Multilayered Rosettes, Nos
- Being tested
- Radiotherapy (RT), Chemotherapy Drug, Cancer - Physician's Choice, Non-Investigational Surgical Resection, Temozolomide, Tumor Tissue Sample
- Medical subject headings
- Neuroectodermal Tumors, Primitive, Pyloric Stenosis, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Radiotherapy (RT)Radiation
Also called Focal Radiotherapy, Radiation Therapy
Undergo RT
Chemotherapy Drug, Cancer - Physician's ChoiceDrug
Also called Standard of Care, High dose Chemotherapy
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 ResectionProcedure or surgery
Also called Standard of Care Surgical Resection, Standard of Care Surgical Resection of Tumor
Undergo surgery directly before study enrollment as part of planned care.
TemozolomideDrug
Also called Temodar
Participants assigned to or whom receive optional RT will receive concurrent temozolomide
Tumor Tissue SampleProcedure or surgery
Also called Tumor Specimen
Tumor tissue will be collected for correlative studies
Blood SampleProcedure or surgery
Also called Blood Specimen
Blood samples will be collected for correlative studies
Cerebrospinal Fluid (CSF) SampleProcedure or surgery
Also called CSF Specimen
CSF samples will be collected for correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Gross-total resection, non-metastatic, early radiotherapy | Experimental | 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. Receives: Radiation: Radiotherapy (RT); Drug: Chemotherapy Drug, Cancer - Physician's Choice; Procedure: Non-Investigational Surgical Resection; Drug: Temozolomide; Procedure: Tumor Tissue Sample; Procedure: Blood Sample; Procedure: Cerebrospinal Fluid (CSF) Sample |
| Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy | Experimental | 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. Receives: Radiation: Radiotherapy (RT); Drug: Chemotherapy Drug, Cancer - Physician's Choice; Procedure: Non-Investigational Surgical Resection; Drug: Temozolomide; Procedure: Tumor Tissue Sample; Procedure: Blood Sample; Procedure: Cerebrospinal Fluid (CSF) Sample |
| Cohort 3A: Metastatic or residual disease, early radiotherapy | Experimental | 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. Receives: Radiation: Radiotherapy (RT); Drug: Chemotherapy Drug, Cancer - Physician's Choice; Procedure: Non-Investigational Surgical Resection; Drug: Temozolomide; Procedure: Tumor Tissue Sample; Procedure: Blood Sample; Procedure: Cerebrospinal Fluid (CSF) Sample |
| Cohort 3B: Metastatic or residual disease, high-dose chemotherapy | Experimental | 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. Receives: Radiation: Radiotherapy (RT); Drug: Chemotherapy Drug, Cancer - Physician's Choice; Procedure: Non-Investigational Surgical Resection; Drug: Temozolomide; Procedure: Tumor Tissue Sample; Procedure: Blood Sample; Procedure: Cerebrospinal Fluid (CSF) Sample |
What is being measured
The primary outcome is the main question the study is designed to answer.
Median Progression-free survival at 6 months (PFS6) (Cohort 1)
The median PFS6 is defined as the median number of months for participants in Cohort 1 who have remained progression-free from the date of initial surgical resection until 6 months.
Measured: Up to 6 months
Secondary outcomes (2)
Median Progression-free survival at 2 years progression-free survival (PFS)
The median PFS6 is defined as the median number of months for participants by cohort who have remained progression-free from the date of initial surgical resection until 2 years post-surgery.
Measured: Up to 2 years
Median Overall Survival at 2 years (OS)
The median OS is defined as the median number of months for participants by cohort who are still alive from the date of initial surgical resection until 2 years post-surgery.
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.
- First posted6 Mar 2025
- Study start6 Mar 2025
- Primary completion30 Sep 2030 (estimated)
- Study completion31 Mar 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Embryonal Tumor With Multilayered RosettesStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- Pediatric Neuro-Oncology Consortium, Solving Kids' Cancer
- Responsible party
- Sponsor
- Investigators
- Sabine Mueller, MD, PhD, MAS, Principal investigator, University of California, San FranciscoDerek Hanson, MD, Study chair, Hackensack Meridian Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06861244
- Sponsor's protocol ID
- 240830
- Other IDs
- NCI-2025-01764
- Acronym
- PNOC031
- Status last verified
- Jun 2026
- First submitted
- 28 Feb 2025
- Last updated
- 29 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: De-identified data may be shared with study collaborators
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PNOC031: Protocol for Embryonal Tumor With Multilayered Rosettes (ETMR)
- ClinicalTrials.gov ID
- NCT06861244
- Acronym / sponsor ID
- PNOC031 / 240830
- Conditions
- Embryonal Tumor With Multilayered Rosettes, Embryonal Tumor With Multilayered Rosettes, Nos
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- Pediatric Neuro-Oncology Consortium, Solving Kids' Cancer
- Study start
- 6 Mar 2025 (actual)
- Primary completion
- 30 Sep 2030 (estimated)
- Study completion
- 31 Mar 2032 (estimated)
- First posted
- 6 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
PRIMARY OBJECTIVES I. To determine the six-month progression-free survival (PFS6) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR, treated using a regimen of induction chemotherapy and early focal radiotherapy (Cohort 1)
SECONDARY OBJECTIVES I. To determine the two-year progression-free survival (PFS) and overall survival (OS) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR (Cohort 1).
II. To determine the two-year progression-free survival (PFS) and overall survival (OS) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR (Cohort 2).
III. To determine the two-year progression-free survival (PFS), overall survival (OS) and objective response rate of participants with newly diagnosed, incompletely resected and/or metastatic ETMR (Cohort 3A and 3B)
EXPLORATORY OBJECTIVES:
- To validate the utility of a liquid miRNA biomarker in blood and Cerebral spinal fluid (CSF) as a correlative marker of a participant's disease status.
II. To better define the genomic landscape of ETMR.
OUTLINE:
Participants with newly diagnosed ETMR will obtain either gross total, or sub-total resection surgery prior to enrollment. After surgery, participants will be assigned to 1 of 4 possible cohorts:
Cohorts 1 and 2: Participants with newly diagnosed, gross-totally resected, non-metastatic ETMR.
Cohorts 3A and 3B: Participants with newly diagnosed, incompletely resected and/or metastatic ETMR.
Participants will be assessed for survival outcomes for up to 2 years. Follow-up procedures are to be captured under the PNOC COMP protocol. Participants will be followed under the Pediatric Neuro-Oncology Consortium (PNOC) COMP protocol until death or withdrawal from study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Gross-total resection, non-metastatic, early radiotherapy | Experimental | Radiation: Radiotherapy (RT), Drug: Chemotherapy Drug, Cancer - Physician's Choice, Procedure: Non-Investigational Surgical Resection, Drug: Temozolomide, Procedure: Tumor Tissue Sample, Procedure: Blood Sample, Procedure: Cerebrospinal Fluid (CSF) SampleParticipants 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. |
| Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy | Experimental | Radiation: Radiotherapy (RT), Drug: Chemotherapy Drug, Cancer - Physician's Choice, Procedure: Non-Investigational Surgical Resection, Drug: Temozolomide, Procedure: Tumor Tissue Sample, Procedure: Blood Sample, Procedure: Cerebrospinal Fluid (CSF) SampleParticipants 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. |
| Cohort 3A: Metastatic or residual disease, early radiotherapy | Experimental | Radiation: Radiotherapy (RT), Drug: Chemotherapy Drug, Cancer - Physician's Choice, Procedure: Non-Investigational Surgical Resection, Drug: Temozolomide, Procedure: Tumor Tissue Sample, Procedure: Blood Sample, Procedure: Cerebrospinal Fluid (CSF) SampleParticipants 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. |
| Cohort 3B: Metastatic or residual disease, high-dose chemotherapy | Experimental | Radiation: Radiotherapy (RT), Drug: Chemotherapy Drug, Cancer - Physician's Choice, Procedure: Non-Investigational Surgical Resection, Drug: Temozolomide, Procedure: Tumor Tissue Sample, Procedure: Blood Sample, Procedure: Cerebrospinal Fluid (CSF) SampleParticipants 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (29 listed)
- Participants must have either a molecularly or histologically confirmed embryonal tumor with multilayered rosettes.
- 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.
- Prior Therapy:
- Participants must not have received prior radiation for treatment of tumor.
- Participants of any age are eligible.
Key exclusion criteria (5 listed)
- Cohort 1 only: Participants who have received any prior tumor-directed therapy other than surgical intervention
- Participants who are receiving any other tumor directed investigational agents.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to the agents used in study.
- Uncontrolled intercurrent illness.
- Women of childbearing potential must not be pregnant or breast-feeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Radiotherapy (RT)Also: Focal Radiotherapy, Radiation TherapyUndergo RT | Radiation | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
| Chemotherapy Drug, Cancer - Physician's ChoiceAlso: Standard of Care, High dose ChemotherapyOne 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. | Drug | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
| Non-Investigational Surgical ResectionAlso: Standard of Care Surgical Resection, Standard of Care Surgical Resection of TumorUndergo surgery directly before study enrollment as part of planned care. | Procedure or surgery | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
| TemozolomideAlso: TemodarParticipants assigned to or whom receive optional RT will receive concurrent temozolomide | Drug | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
| Tumor Tissue SampleAlso: Tumor SpecimenTumor tissue will be collected for correlative studies | Procedure or surgery | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
| Blood SampleAlso: Blood SpecimenBlood samples will be collected for correlative studies | Procedure or surgery | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
| Cerebrospinal Fluid (CSF) SampleAlso: CSF SpecimenCSF samples will be collected for correlative studies | Procedure or surgery | Cohort 1: Gross-total resection, non-metastatic, early radiotherapy, Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapy, Cohort 3A: Metastatic or residual disease, early radiotherapy, Cohort 3B: Metastatic or residual disease, high-dose chemotherapy |
Primary outcome
Median Progression-free survival at 6 months (PFS6) (Cohort 1)
Time frame: Up to 6 months
The median PFS6 is defined as the median number of months for participants in Cohort 1 who have remained progression-free from the date of initial surgical resection until 6 months.
2 secondary outcomes
Median Progression-free survival at 2 years progression-free survival (PFS)
Time frame: Up to 2 years
Median Overall Survival at 2 years (OS)
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2026.
- Individual participant dataSharing planned: Yes. De-identified data may be shared with study collaborators
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sabine Mueller, MD, PhD, MAS principal investigator
University of California, San Francisco
Derek Hanson, MD study chair
Hackensack Meridian Health
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Radiotherapy (RT), Chemotherapy Drug, Cancer - Physician's Choice and Non-Investigational Surgical Resection in people with Embryonal Tumor With Multilayered Rosettes and Embryonal Tumor With Multilayered Rosettes, Nos, run by University of California, San Francisco at 8 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 29 inclusion and 5 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 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Operations Office (415-502-1600, PNOC031@ucsf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Median Progression-free survival at 6 months (PFS6) (Cohort 1) (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Jun 2026.