Anti-tumor antibiotic

Various Treatments for Embryonal Tumor With Multilayered Rosettes (PNOC031 Trial)

San Francisco, CA

Phase 2

Recruiting

Led By Sabine Mueller, MD, PhD, MAS

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants must have either a molecularly or histologically confirmed embryonal tumor with multilayered rosettes.

Cohort 1 participants must not have received any prior tumor-directed therapy other than surgical resection.

Must not have

Cohort 1 only: Participants who have received any prior tumor-directed therapy other than surgical intervention.

Women of childbearing potential must not be pregnant or breast-feeding.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is looking at different treatments for children with a specific type of brain tumor called Embryonal Tumor With Multilayered Rosettes (ETMR). They will be testing the use of

Who is the study for?

This trial is for children with a newly diagnosed brain cancer called Embryonal Tumor With Multilayered Rosettes (ETMR). Participants must be suitable for chemotherapy, radiation therapy, and potential surgical procedures to remove the tumor. Specific eligibility criteria are not provided.

What is being tested?

The study tests a treatment plan that includes induction chemotherapy to shrink the tumor, high-dose chemotherapy to target cancer cells, and focal radiation therapy aimed directly at the tumor area after surgery.

What are the potential side effects?

Possible side effects include those common to chemotherapy such as nausea, hair loss, fatigue; from radiotherapy like skin irritation, headaches; and surgical risks like infection or bleeding.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cancer is confirmed to be a specific type with a certain cell pattern.

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I have only had surgery for my tumor, no other treatments.

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I had complete surgery to remove my tumor, can have early radiotherapy, and my cancer hasn't spread.

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I had complete surgery, high dose chemo, and no spread of cancer.

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My kidney function is within the normal range for my age and gender.

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I have not had radiation for my tumor.

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My liver functions are within the required range.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had cancer treatment other than surgery.

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Median Progression-free survival at 6 months (PFS6) (Cohort 1)

Secondary study objectives

Median Overall Survival at 2 years (OS)

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Cohort 3B: Metastatic or residual disease, high-dose chemotherapyExperimental Treatment7 Interventions

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.

Group II: Cohort 3A: Metastatic or residual disease, early radiotherapyExperimental Treatment7 Interventions

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.

Group III: Cohort 2: Gross-total resection, non-metastatic, high-dose chemotherapyExperimental Treatment7 Interventions

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.

Group IV: Cohort 1: Gross-total resection, non-metastatic, early radiotherapyExperimental Treatment7 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Blood Sample

2016

Completed Phase 4

~6480

Temozolomide

2010

Completed Phase 3

~1880