Targeted Therapy for Pediatric Brain Tumor

Recruiting at 20 trial locations

Overseen By Kelsey H Troyer, PhD

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Nationwide Children's Hospital

Disqualifiers: Non-HGG tumors

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

Compensation: Varies

Number of Visits: 2

Duration: 10-35 days post-radiation therapy

350 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial studies brain tumors in children, teens, and young adults recently diagnosed with high-grade glioma, an aggressive brain cancer. Researchers will use genetic testing on participants' tumors to identify specific DNA changes. These genetic insights will guide the selection of treatment options, including targeted pediatric high-grade glioma therapy, for each person in a follow-up study. Ideal candidates are those newly diagnosed with high-grade glioma who can provide a tumor sample for testing. As a Phase 1 trial, this research focuses on understanding the treatment's effects in people, offering participants the opportunity to receive potentially groundbreaking therapy.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, certain prior cancer treatments are not allowed, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that treating children with high-grade glioma using the drug avapritinib appears safe. In early studies, most patients tolerated avapritinib well, experiencing no severe side effects. Specifically, about 3 out of 7 patients showed positive results on scans, suggesting the drug's potential effectiveness.

While avapritinib seems safe for many, all treatments can have some side effects. Overall, the early results are promising for those considering participation in a clinical trial. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Targeted Pediatric High-Grade Glioma Therapy is unique because it directly targets cancer cells in children with high-grade glioma, focusing on the specific genetic mutations present in these tumors. Unlike traditional treatments like surgery, radiation, and chemotherapy, which affect both healthy and cancerous cells, this therapy aims to minimize damage to healthy tissues. Researchers are excited because this precision approach could lead to more effective treatments with fewer side effects, offering a new hope for improved outcomes in young patients.

What evidence suggests that this treatment might be an effective treatment for high-grade glioma?

Research has shown that new treatments for children's brain tumors, known as high-grade gliomas, appear promising. Early results suggest that avapritinib might be safe and could benefit some patients. By analyzing the tumor's genetic makeup, doctors can customize treatments to fit each patient's needs, potentially improving effectiveness. Although these tumors have been difficult to treat in the past, new targeted treatments offer hope by focusing on specific genetic changes in the tumor. ^{1 5 6 7 8}

Who Is on the Research Team?

Margot Lazow, MD

Principal Investigator

Nationwide Children's Hospital

Maryam Fouladi, MD

Principal Investigator

Nationwide Children's Hospital

Are You a Good Fit for This Trial?

Inclusion Criteria

Age: Patients must be ≥12 months and ≤30 years of age at the time of enrollment onto this screening protocol.

Diagnosis: Patients with newly diagnosed HGG, including DIPG are eligible. The diagnosis of HGG must have been confirmed by local pathology review. for the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2/3 of the pons, with histopathology consistent with diffuse WHO grade 2-4 glioma (eg, diffuse astrocytoma, anaplastic astrocytoma, glioblastoma, H3K27-altered diffuse midline glioma). For all other tumors, histologic grade must be WHO grade 3-4.

Disease Status: There are no disease status requirements for enrollment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Molecular Profiling

Comprehensive molecular screening of tumor tissue using a multi-omic approach to determine eligibility for treatment arms

4 weeks

1 visit (in-person)

Treatment

Patients are stratified to biologically-targeted treatment arms based on tumor molecular profile and histopathology

10-35 days post-radiation therapy

Follow-up

Participants are monitored for safety and effectiveness after treatment, including longitudinal evaluation of biomarkers

4 years

What Are the Treatments Tested in This Trial?

Interventions

• Targeted Pediatric High-Grade Glioma Therapy

Who Is Running the Clinical Trial?

Nationwide Children's Hospital

Lead Sponsor

Trials

354

Recruited

5,228,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05839379

NCT05839379 | Targeted Pediatric High-Grade Glioma ... The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6682212/

Molecular profiling and targeted therapy in pediatric gliomas Chemotherapy has been shown to be an effective modality for the treatment of pediatric LGG. The most well-established regimen, first studied in the Children's ...

3. childrenshospital.org childrenshospital.org/newsroom/news-events/pediatric-high-grade-gliomas-research-reveals-effective-targeting

Pediatric High-grade Gliomas: Research Reveals Effective ... This early data suggests that avapritinib is generally safe and may trigger an initial clinical response in a small group of patients who have ...

4. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1504803/full

Targeted therapy in pediatric central nervous system tumors The present narrative review focuses on recent progress in targeted therapies for pediatric CNS tumors.

5. sciencedirect.com sciencedirect.com/science/article/pii/S2772610X24000709

European standard clinical practice recommendations for ... The outcome for these tumours is generally poor with 5-year survival rates of less than 20 %. Despite improved biological insights into pedHGGs and the promise ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10957575/

Therapeutically targeting the unique disease landscape of ... Glioblastoma multiforme (GBM) has been the most prevalent form of grade 4 pHGG and has an extremely poor 5-year survival rate of 1.2% (18). However as of the ...

7. trials.braintumor.org trials.braintumor.org/trials/NCT05839379

Targeted Pediatric High-Grade Glioma Therapy The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with ...

8. cell.com cell.com/cancer-cell/fulltext/S1535-6108(25)00070-4

Effective targeting of PDGFRA-altered high-grade glioma ... Our early data demonstrate that avapritinib is well tolerated and results in radiographic response in 3/7 cases, suggesting a potential role for ...