NCT06861244

Embryonal Tumor With Multilayered Rosettes

PNOC031: Protocol for Embryonal Tumor With Multilayered Rosettes (ETMR)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 70 (estimated)
• Sponsor: University of California, San Francisco · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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).

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: I. 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

Interventions

Type	Name	Description
RADIATION	Radiotherapy (RT)	Undergo RT
DRUG	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.
PROCEDURE	Non-Investigational Surgical Resection	Undergo surgery directly before study enrollment as part of planned care.
DRUG	Temozolomide	Participants assigned to or whom receive optional RT will receive concurrent temozolomide
PROCEDURE	Tumor Tissue Sample	Tumor tissue will be collected for correlative studies
PROCEDURE	Blood Sample	Blood samples will be collected for correlative studies
PROCEDURE	Cerebrospinal Fluid (CSF) Sample	CSF samples will be collected for correlative studies

Timeline

• Start date: 2025-03-06
• Primary completion: 2030-09-30
• Completion: 2032-03-31
• First posted: 2025-03-06
• Last updated: 2026-04-13

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study