Molecular Profiling for Brain Tumor

(PNOC008 Trial)

Recruiting at 11 trial locations

Overseen By Krystal Pryor, BA

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: University of California, San Francisco

Must be taking: Temozolomide

Must not be taking: Anti-cancer therapy

Disqualifiers: Other trial, Infection, Heart failure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 12 months

44 Participants Needed

Trial Summary

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot be on any anti-cancer therapy except for steroids and temozolomide during radiation therapy. It's best to discuss your specific medications with the study team.

What data supports the effectiveness of the treatment Specialized tumor board recommendation for brain tumors?

Research shows that using molecular profiling to guide treatment decisions can help select the most effective therapies for brain tumor patients. This approach has been shown to improve the likelihood of success in clinical trials by matching patients with targeted therapies based on their tumor's genetic makeup. ^{1 2 3 4 5}

Is molecular profiling for brain tumors safe for humans?

The research does not provide specific safety data for molecular profiling in humans, but it suggests that molecular profiling is used to guide treatment decisions in clinical settings, indicating it is generally considered safe. ^{1 2 3 5 6}

How does the treatment 'Specialized tumor board recommendation' differ from other treatments for brain tumors?

This treatment is unique because it involves a specialized tumor board that uses molecular profiling to tailor recommendations specifically for each patient's brain tumor. This approach allows for personalized treatment plans based on the genetic makeup of the tumor, which can lead to more effective and targeted therapies compared to standard treatments. ^{3 7 8 9 10}

What is the purpose of this trial?

This is a 2 strata pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The study will use a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq. The current study will test the efficacy of such an approach in children with High-grade gliomas HGG.

Research Team

Sabine Mueller, MD, PhD, MAS

Principal Investigator

University of California, San Francisco

Eligibility Criteria

This trial is for children and young adults up to 21 years old with newly diagnosed high-grade gliomas (HGG), including those with specific genetic mutations but not DIPG. Participants must start radiation therapy within six weeks of diagnosis, have a certain level of physical function, and be able to give consent. Pregnant or breastfeeding individuals are excluded, as well as those on other clinical trials or with serious health issues that could affect participation.

Inclusion Criteria

I am 21 years old or younger.

My seizures are under control.

Ability by patient or parent/legal guardian to understand a written informed consent document, and the willingness to sign it.

Exclusion Criteria

Patients who are currently enrolled on another therapeutic clinical trial. Individual cases should be discussed with the study chair.

I am not on any cancer treatment now, except possibly steroids or temozolomide at a specific dose.

I can attend follow-up visits or do them via telemedicine.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive an individualized treatment plan based on molecular profiling of their tumor

Up to 12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Specialized tumor board recommendation

Trial Overview The trial tests an individualized treatment plan based on each patient's tumor genetics using gene expression profiling and sequencing techniques. The goal is to see if tailoring the treatment according to the tumor's molecular characteristics can improve outcomes in pediatric patients with HGG.

Participant Groups

Group I: Newly diagnosed HGG (Stratum A) Experimental Treatment 1 Intervention

Children and young adults with newly diagnosed HGG receive an individualized treatment plan. Each treatment is different and depends on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.

Group II: Diffuse midline HGG (Stratum B) Experimental Treatment 1 Intervention

Children and young adults with diffuse midline high grade gliomas receive an individualized treatment plan. Each treatment is different and depends on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

The V Foundation for Cancer Research

Collaborator

Trials

21

Recruited

1,300+

Pacific Pediatric Neuro-Oncology Consortium

Collaborator

Trials

16

Recruited

840+

References

1. United States pubmed.ncbi.nlm.nih.gov

Prospective, high-throughput molecular profiling of human gliomas. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Molecular profile to guide personalized medicine in adult patients with primary brain tumors: results from the ProfiLER trial. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Significance of molecular diagnostics for therapeutic decision-making in recurrent glioma. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Genomic Molecular Classification of CNS Malignancies. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Case Report: Next generation sequencing identifies a NAB2-STAT6 fusion in Glioblastoma. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Toward Precision Oncology in Glioblastoma with a Personalized Cancer Genome Reporting Tool and Genetic Changes Identified by Whole Exome Sequencing. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Novel insights into the pathogenesis of gliomas based on large-scale molecular profiling approaches. [2015]

8. United States pubmed.ncbi.nlm.nih.gov

Molecular Profiling and Targeted Therapies in Gliomas. [2023]

9. Korea (South) pubmed.ncbi.nlm.nih.gov

Recent Advancement of the Molecular Diagnosis in Pediatric Brain Tumor. [2020]

10. United States pubmed.ncbi.nlm.nih.gov

Insights From Molecular Profiling of Adult Glioma. [2022]