NCT06861244

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).

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P50-P75 for phase_2

Timeline
72mo left

Started Mar 2025

Longer than P75 for phase_2

Geographic Reach
1 country

7 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress17%
Mar 2025Mar 2032

First Submitted

Initial submission to the registry

February 28, 2025

Completed
6 days until next milestone

First Posted

Study publicly available on registry

March 6, 2025

Completed
Same day until next milestone

Study Start

First participant enrolled

March 6, 2025

Completed
5.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2030

Expected
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2032

Last Updated

April 23, 2026

Status Verified

April 1, 2026

Enrollment Period

5.6 years

First QC Date

February 28, 2025

Last Update Submit

April 20, 2026

Conditions

Embryonal Tumor With Multilayered RosettesEmbryonal Tumor With Multilayered Rosettes, Nos

Outcome Measures

Primary Outcomes (1)

  • 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.

    Up to 6 months

Secondary Outcomes (2)

  • Median Progression-free survival at 2 years progression-free survival (PFS)

    Up to 2 years

  • Median Overall Survival at 2 years (OS)

    Up to 2 years

Study Arms (4)

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.

Radiation: Radiotherapy (RT)Drug: Chemotherapy Drug, Cancer - Physician's ChoiceProcedure: Non-Investigational Surgical ResectionDrug: TemozolomideProcedure: Tumor Tissue SampleProcedure: Blood SampleProcedure: 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.

Radiation: Radiotherapy (RT)Drug: Chemotherapy Drug, Cancer - Physician's ChoiceProcedure: Non-Investigational Surgical ResectionDrug: TemozolomideProcedure: Tumor Tissue SampleProcedure: Blood SampleProcedure: 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.

Radiation: Radiotherapy (RT)Drug: Chemotherapy Drug, Cancer - Physician's ChoiceProcedure: Non-Investigational Surgical ResectionDrug: TemozolomideProcedure: Tumor Tissue SampleProcedure: Blood SampleProcedure: 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.

Radiation: Radiotherapy (RT)Drug: Chemotherapy Drug, Cancer - Physician's ChoiceProcedure: Non-Investigational Surgical ResectionDrug: TemozolomideProcedure: Tumor Tissue SampleProcedure: Blood SampleProcedure: Cerebrospinal Fluid (CSF) Sample

Interventions

Chemotherapy Drug, Cancer - Physician's ChoiceDRUG

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.

Also known as: Standard of Care, High dose Chemotherapy
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy
Blood SamplePROCEDURE

Blood samples will be collected for correlative studies

Also known as: Blood Specimen
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy
Cerebrospinal Fluid (CSF) SamplePROCEDURE

CSF samples will be collected for correlative studies

Also known as: CSF Specimen
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy
Radiotherapy (RT)RADIATION

Undergo RT

Also known as: Focal Radiotherapy, Radiation Therapy
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy
Non-Investigational Surgical ResectionPROCEDURE

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

Also known as: Standard of Care Surgical Resection, Standard of Care Surgical Resection of Tumor
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy
TemozolomideDRUG

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

Also known as: Temodar
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy
Tumor Tissue SamplePROCEDURE

Tumor tissue will be collected for correlative studies

Also known as: Tumor Specimen
Cohort 1: Gross-total resection, non-metastatic, early radiotherapyCohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyCohort 3A: Metastatic or residual disease, early radiotherapyCohort 3B: Metastatic or residual disease, high-dose chemotherapy

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.
  • 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.
  • +14 more criteria

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

University of California, San Francisco

San Francisco, California, 94143, United States

RECRUITING

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, 46202, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21218, United States

RECRUITING

Washington University in St. Louis

St Louis, Missouri, 63130, United States

RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

RECRUITING

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

MeSH Terms

Conditions

Neuroectodermal Tumors, PrimitivePyloric Stenosis, Hypertrophic

Interventions

RadiotherapyStandard of CareDrug TherapyTemozolomideBlood Specimen Collection

Condition Hierarchy (Ancestors)

Neoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissuePyloric StenosisGastric Outlet ObstructionStomach DiseasesGastrointestinal DiseasesDigestive System Diseases

Intervention Hierarchy (Ancestors)

TherapeuticsQuality Indicators, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and EvaluationDacarbazineTriazenesOrganic ChemicalsImidazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsSpecimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques

Study Officials

  • Sabine Mueller, MD, PhD, MAS

    University of California, San Francisco

    PRINCIPAL INVESTIGATOR

  • Derek Hanson, MD

    Hackensack Meridian Health

    STUDY CHAIR

Central Study Contacts

PNOC Operations Office

CONTACT

415-502-1600PNOC031@ucsf.edu

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 28, 2025

First Posted

March 6, 2025

Study Start

March 6, 2025

Primary Completion (Estimated)

September 30, 2030

Study Completion (Estimated)

March 31, 2032

Last Updated

April 23, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

De-identified data may be shared with study collaborators

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
Until study close out

Locations

US(7)