Back to search results

Targeted Therapy for Pediatric High-Grade Glioma

This study aims to understand the genetic makeup of brain tumors in children, adolescents, and young adults (ages 12 months to 39 years) who have recently been diagnosed with high-grade glioma (a type of aggressive brain cancer), including DIPG (Diffuse Intrinsic Pontine Glioma), Anaplastic Astrocytoma, and Glioblastoma. Researchers will perform detailed genetic testing on tumor samples to identify specific changes. The goal is to see if these genetic findings can help decide which patients might be eligible for future treatment studies that target these specific genetic alterations. The study will also look at how feasible it is to use this genetic information to guide enrollment into those treatment studies. This is an observational study, meaning no specific treatments are given as part of this study itself. The study plans to enroll about 350 participants.

Study design
This is an observational study, meaning no specific treatments are given. It aims to enroll 350 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Molecular profiling and feasibility of enrollment to a treatment protocol will be measured at 4 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05839379

Targeted Pediatric High-Grade Glioma Therapy

Recruiting
Not specifiedAges 12–39Observational
Nationwide Children's Hospital
~350 participants
Updated 2026-06-10 on ClinicalTrials.gov

At a glance

Recruiting sites
12 of 21 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Molecular profiling
Measured over 4 years
+1 more outcome measured

Conditions

High Grade Glioma
Diffuse Intrinsic Pontine Glioma
Anaplastic Astrocytoma
Glioblastoma
Glioblastoma Multiforme
Diffuse Midline Glioma, H3 K27M-Mutant
Metastatic Brain Tumor
WHO Grade III Glioma
WHO Grade IV Glioma

Explore related trials

Anaplastic AstrocytomaDiffuse Intrinsic Pontine GliomaDiffuse Midline GliomaGlioblastomaMalignant Glioma

Where it's being run

21 sites across 20 states
Ohio2
Colorado1
District of Columbia1
Illinois1
Massachusetts1
Michigan1
North Carolina1
Pennsylvania1

Study leadership

  • Maryam Fouladi, MD · STUDY_CHAIR · Nationwide Children's Hospital
  • Margot Lazow, MD · STUDY_DIRECTOR · Nationwide Children's Hospital

Who to contact

Kelsey H Troyer, PhD
[email protected]
16147223284
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

Measurable disease is not required. Patients without measurable disease are eligible.
Patients with metastatic/disseminated or multifocal disease or gliomatosis cerebri are eligible.
Patients with a primary spinal tumor are eligible.
Patients with secondary, radiation related HGG are eligible. 4. Prior Therapy for HGG: Surgery, radiation, and/or dexamethasone are permissible. Temozolomide concurrent with radiation is permissible. Prior administration of avastin/bevacizumab is allowed (individual treatment arms have different washout period requirements, check individual arm eligibility). No other prior anticancer therapy for HGG will be allowed.
Participants screening for assignment to TarGeT-L may not have received radiation.
If a patient screens through OPTION #1, tumor sample in addition to normal comparator tissue (peripheral blood, saliva, or buccal swab) must be submitted for comprehensive molecular screening at the time of screening enrollment.
If a patient screens through OPTIONS #2 or #3, results from previously performed molecular profiling must be submitted following enrollment. It is highly recommended that results be uploaded within 7 days of enrollment (if results are available at time of enrollment) or within 7 days of results becoming available (if pending at time of enrollment) to allow adequate time for central review. 6. Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines. 7. Enrollment timeline: Patients are eligible to enroll on the TarGeT-SCR anytime between diagnosis and the following specific timepoints post completion of RT (if relevant)
Patients screening through OPTION #1 are eligible to enroll anytime between diagnosis and 10 days post RT (if completing RT).
Patients screening through OPTIONS #2 or #3 are eligible to enroll anytime between diagnosis and 21 days post RT (if completing RT).
Participants screening for TarGeT-L (lorlatinib) are eligible to enroll on TarGeT-SCR anytime between diagnosis and 31 days post definitive surgery (to allow time for molecular review).
For treatment protocols that include targeted therapy administered concurrently with RT, patients must start treatment within 10 calendar days of starting RT.
For treatment protocols that only include maintenance/adjuvant therapy (no systemic therapy given concurrently with radiation), patients must start treatment by 35 days post RT
OPTION1: Molecular screening through CONNECT TarGeT Clinical Testing Laboratories
OPTION2: Molecular screening through a national comprehensive tumor profiling program
OPTION3: Clinically validated targeted sequencing or focused profiling

Exclusion

Tumors that do not meet HGG and DIPG diagnoses specified above

What this trial measures

  • Molecular profiling4 years

    Utilize molecular, clinical, and histopathologic data to assess eligibility for specific biologically-guided treatment subprotocols among pediatric, adolescent and young adult patients with newly diagnosed HGG, including DIPG.

  • Feasibility of molecular profiling and enrollment to a TarGeT treatment protocol4 years

    Determine the percent of pediatric, adolescent, and young adult patients newly diagnosed with HGG, including DIPG, who undergo screening through one of three TarGeT-SCR screening mechanisms and are assigned to a TarGeT treatment arm.