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Genetic profiling for pediatric high-grade glioma treatment eligibility

Molecularly-Guided Phase II Umbrella Trial for Children, Adolescents, and Young Adults Newly Diagnosed With High-Grade Glioma, Including Diffuse Intrinsic Pontine Glioma

Nationwide Children's Hospital · NCT05839379

This study is testing if genetic testing of brain tumors in children and young adults with high-grade gliomas can help match them to the best treatment options.

Quick facts

Study typeObservational
Enrollment350 (estimated)
Ages12 Months to 39 Years
SexAll
SponsorNationwide Children's Hospital (other)
Drugs / interventionsbevacizumab, lorlatinib, radiation
Locations21 sites (Aurora, Colorado and 20 other locations)
Trial IDNCT05839379 on ClinicalTrials.gov

What this trial studies

This study aims to conduct genetic sequencing on brain tumors from children, adolescents, and young adults newly diagnosed with high-grade gliomas. It employs a comprehensive molecular screening approach using advanced techniques such as whole exome sequencing, RNA sequencing, and DNA methylation microarrays. Based on the genetic alterations identified, patients will be stratified into targeted treatment arms for a subsequent phase II clinical trial. The study also includes longitudinal evaluations of various biological samples to identify predictive biomarkers for treatment response and toxicity.

Who should consider this trial

Good fit: Ideal candidates are children, adolescents, and young adults aged 12 months to 39 years with newly diagnosed high-grade gliomas.

Not a fit: Patients with previously treated high-grade gliomas or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could lead to more personalized and effective treatment options for young patients with high-grade gliomas.

How similar studies have performed: Other studies utilizing molecular profiling for targeted therapies in pediatric cancers have shown promising results, indicating potential success for this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Age: Patients must be ≥12 months and ≤39 years of age at the time of enrollment onto this screening protocol.
2. Diagnosis: Patients with newly diagnosed HGG, including DIPG are eligible. Diagnosis must have histologic confirmation from biopsy or resection. The diagnosis of HGG must have been confirmed by pathology review at the local site. 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.
3. Disease Status: There are no disease status requirements for enrollment.

   * 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.

   Timing from surgery to start of RT: For patients who have started RT, radiation must have started \<42 days from definitive surgery or biopsy, however it is strongly recommended patients start RT within 31 days from definitive surgery (if patient had two surgeries, radiation must have started within 31 days from second surgery).
5. Tumor Sample Availability OR results from previous molecular profiling/targeted sequencing

   * 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).

However, it is important to note the following:

* 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

  #SCREENING OPTIONS
* 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 Criteria:

-Tumors that do not meet HGG and DIPG diagnoses specified above

Where this trial is running

Aurora, Colorado and 20 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

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

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.