Molecular Profiling for Brain Tumor

(PNOC008 Trial)

Not currently recruiting at 13 trial locations
AD
SM
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Overseen ByKrystal Pryor, BA
Age: < 65
Sex: Any
Trial Phase: Academic
Sponsor: University of California, San Francisco
Must be taking: Temozolomide
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on developing personalized treatment plans for children and young adults with high-grade gliomas (HGG), a type of brain tumor. By examining the genetic makeup of each patient's tumor, the researchers aim to identify the most effective treatment method for each individual. Participants will receive a treatment plan tailored by a specialized tumor board (a group of medical experts) based on their tumor's genetic profile. This trial suits those newly diagnosed with high-grade gliomas who have begun radiation therapy within the past six weeks. As an unphased trial, it offers the opportunity to receive a highly personalized treatment approach that could significantly impact health.

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 prior data suggests that this treatment approach is safe for children with high-grade gliomas?

Research has shown that recommendations from tumor boards are very effective. In one study, 91% of their treatment suggestions were followed. Tumor boards consist of experts who create personalized treatment plans based on the unique details of a patient's tumor. They don't rely on a single drug or treatment; instead, they develop a plan that may include various treatments, depending on the board's decisions.

As this is an early trial, specific safety data might be limited. However, guidance from a tumor board suggests that the plans are carefully considered, focusing on the safest options for each patient. Without specific treatment data, predicting how well each plan will be tolerated is challenging. Nonetheless, the expertise of the tumor board indicates that safety is a priority.12345

Why are researchers excited about this trial?

Unlike standard treatments for high-grade gliomas, which often involve surgery, radiation, and chemotherapy, the study treatment is unique because it tailors therapy based on the molecular profile of each patient's tumor. This personalized approach means that the Specialized Tumor Board recommends treatments specifically targeting the genetic characteristics of the tumor, potentially leading to more effective outcomes. Researchers are excited about this method because it allows for a customized plan that could improve response rates and potentially reduce side effects, offering hope for better management of these aggressive brain tumors in children and young adults.

What evidence suggests that this trial's treatments could be effective for high-grade gliomas?

Research has shown that diffuse midline gliomas (DMG), a type of aggressive brain tumor, are difficult to treat with standard methods like radiation and chemotherapy. In this trial, children and young adults with DMG will receive individualized treatment plans based on the molecular profile of their tumors, as recommended by the Specialized Tumor Board. A new drug, dordaviprone, has shown promise for DMG, with about 22% of patients responding to it and an average response lasting 10.3 months. This drug targets the genetic changes in DMG, offering new hope for those affected.

For newly diagnosed high-grade gliomas (HGG), participants will also receive personalized treatment plans. Treatments like tumor treating fields (TTFields) have been found to help patients live longer without the disease worsening and to improve overall survival. Recent studies indicate that about 36% of patients live for five years, which marks a significant improvement given the serious nature of these brain tumors. These advancements suggest that personalized treatment plans based on the tumor's genetic makeup could lead to better outcomes for children with these conditions.678910

Who Is on the Research Team?

Dr. Sabine Mueller | UCSF Benioff ...

Sabine Mueller, MD, PhD, MAS

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

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.
See 6 more

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.
See 2 more

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Newly diagnosed HGG (Stratum A)Experimental Treatment1 Intervention
Group II: Diffuse midline HGG (Stratum B)Experimental Treatment1 Intervention

Find a Clinic Near You

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+

Citations

Decoding Diffuse Midline Gliomas: A Comprehensive Review ...Unfortunately, DMG has a very poor prognosis, rendering traditional treatment such as radiation therapy and chemotherapy limited in controlling tumor growth.
Study Details | NCT05009992 | Combination Therapy for ...In cohort 1B, previous tumor tissue confirmation of DMG is mandatory and pathology must be consistent with a DMG including diffuse midline glioma Histone 3 ...
Therapeutic avenues for targeting treatment challenges of ...Diffuse midline glioma (DMG) is the leading cause of brain tumor-related deaths in children. DMG typically presents with variable neurologic symptoms ...
FDA Approves Dordaviprone for Diffuse Midline GliomaEfficacy was assessed in 50 patients across five open-label, nonrandomized U.S. trials, showing an ORR of 22% and a median DOR of 10.3 months.
Dordaviprone Receives Accelerated FDA Approval for ...FDA accelerates approval of dordaviprone, the first targeted therapy for aggressive diffuse midline glioma, offering hope for patients with H3 ...
SEOM-GEINO clinical guidelines for high-grade gliomas of ...Whereas the prognosis for GB is grim (5-year survival rate of 5–10%) outcomes for the other high-grade gliomas are typically better, depending ...
Quality improvement in neurology - PubMed Central - NIHOne study of brain tumor board discussions revealed that 91% of 1,516 clinical recommendations were implemented, and that nearly half of those were ...
BRAIN TUMOUR GROUP OVERVIEW AND DISCIPLINE ...Introduction. Tumours of the central nervous system (CNS) represent the most common group of solid neoplasms in children and adolescents.
Role of the tumor board when prescribing mutant isocitrate ...The RTOG 9802 trial demonstrated prolonged PFS and OS in patients with grade 2 gliomas by adding PCV chemotherapy to radiotherapy (54 Gy) ...
ASTRO updates guideline on radiation therapy for high ...ASTRO updates guideline on radiation therapy for high-grade diffuse glioma, the most common primary brain tumor in adults.
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