GliomaPNOC008NCT03739372
Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan for Patients With High Grade Glioma
A study testing Specialized tumor board recommendation in people with Glioma and Glioma of Brain, run by University of California, San Francisco at 12 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Glioma, Glioma of Brain, Cancer
- What is being tested?
- Specialized tumor board recommendation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 12 sitesUnited States
- Study dates
- 28 Nov 2018to 1 May 2026
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with newly diagnosed HGG (including midline HGG but excluding DIPG), who undergo tissue collection as part of standard of care. HGG is defined as either World Health Organization (WHO) grade III or IV, or testing positive for H3K27M mutation. Patients with disseminated disease are not eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician. Primary spinal cord tumors are eligible.
- Enrollment within 3 weeks of the start of radiation therapy.
- Start of radiation therapy within 6 weeks from initial tissue diagnosis.
- Age ≤ 21 years
- Karnofsky score ≥ 50 for patients ≥ 16 years of age and Lansky score ≥ 50 for patients ≤15 years of age. Patients who are unable to walk because of paralysis but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
- Adequate tissue for molecular profiling (see Section 8 of the protocol for full details)
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- The effects of the current treatment paradigm on the developing human fetus are unknown. For this reason, females of child-bearing potential and males must agree to use adequate contraception: hormonal or barrier method of birth control; abstinence prior to study entry and for the duration of study participation, and 30 days after completion of study drug administration. Should a female become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Males treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 30 days after completion of study drug administration.
- Adequate neurologic function defined as: Patients with seizure disorder may be enrolled if seizures are well controlled.
- Ability by patient or parent/legal guardian to understand a written informed consent document, and the willingness to sign it.
Who cannot join
- Patients who are currently enrolled on another therapeutic clinical trial. Individual cases should be discussed with the study chair.
- Patients who are currently taking any anti-cancer directed therapy. Steroids are not considered anti-cancer therapy. The use of temozolomide during radiation therapy is allowed at standard dosing (maximum 75 to 90 mg/m^2 daily for a total of 42 days). Any other schedule(s) need to be discussed with the study chair.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Female patients of childbearing potential must not be pregnant or breast-feeding. Female patients of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy (as clinically indicated).
- Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy. Telemedicine visits are acceptable.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Johns Hopkins University
Baltimore, Maryland, 21218
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Florida
Gainesville, Florida, 32611
Children's Hospitals and Clinics of Minneapolis
Minneapolis, Minnesota, 55404
The Children's Hospital Of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Utah
Salt Lake City, Utah, 84112
University of California, San Diego Rady Children's Hospital
San Diego, California, 92123
University of California, San Francisco
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults With High Grade Glioma (Excluding Diffuse Intrinsic Pontine Glioma)
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.
Read the detailed description
For children with High-grade gliomas (HGG) including HGG presenting within the midline structures of the brain and spine, outcome remains poor and the majority of children die from this disease. The current 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. This treatment strategy has shown promising results in adult patients with solid tumors and is currently being explored in children with DIPG, neuroblastoma and other solid tumors. The current study will test the efficacy of such an approach in children with HGG for which outcomes remain dismal.
- Conditions
- Glioma, Glioma of Brain, Cancer, Pediatric Cancer, Pediatric Brain Tumor, Astrocytoma
- Being tested
- Specialized tumor board recommendation
- Keywords
- High-grade glioma, whole exome sequencing, WES, genomics, RNA sequencing
- Medical subject headings
- Glioma, Neoplasms, Astrocytoma, Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Specialized tumor board recommendationOther
Based on the molecular profile, the specialized tumor board will determine an individualized treatment recommendation for each patient using up to four FDA approved drugs. In special circumstances, Investigational new drug (IND) study agents may be used.
| Study group | Role | What they receive |
|---|---|---|
| Newly diagnosed HGG (Stratum A) | Experimental | 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. Receives: Other: Specialized tumor board recommendation |
| Diffuse midline HGG (Stratum B) | Experimental | 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. Receives: Other: Specialized tumor board recommendation |
What is being measured
The primary outcome is the main question the study is designed to answer.
12 month Progression Free Survival (PFS) for Stratum A
Progression Free Survival will be determined from date of confirmation of response to first evidence of progression or death at the 12 month time point.
Measured: Up to 12 months
12 month Overall Survival (OS) for Stratum B
Overall survival will be determined from the date of histological diagnosis to time of death at the 12 month time point.
Measured: Up to 12 months
Secondary outcomes (2)
Frequency of Adverse Events
Toxicity will be described by reporting Adverse Events (AE). Events will be assessed according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) v5.0. All study-related and unrelated AEs will be collected and reported, together with their maximum intensity among all recorded respective AE.
Measured: From beginning of enrollment up to 30 days post end of treatment.
Frequency of Serious Adverse Events
Toxicity will be described by reporting Serious Adverse Events (SAE). Events will be assessed according to the NCI CTCAE v5.0. All study-related and unrelated Serious Adverse Events (SAE) will be collected and reported, together with their maximum intensity among all recorded respective SAE.
Measured: From beginning of enrollment up to 30 days post end of treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2018
- Study start28 Nov 2018
- Primary completion31 Dec 2023
- Study completion1 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GliomaStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- Pacific Pediatric Neuro-Oncology Consortium, The V Foundation for Cancer Research
- Responsible party
- Sponsor
- Investigators
- Sabine Mueller, MD, PhD, MAS, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03739372
- Sponsor's protocol ID
- 170827
- Other IDs
- PNOC008
- Acronym
- PNOC008
- Status last verified
- Aug 2026
- First submitted
- 2 Nov 2018
- Last updated
- 11 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data after de-identification.
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults With High Grade Glioma (Excluding Diffuse Intrinsic Pontine Glioma)
- ClinicalTrials.gov ID
- NCT03739372
- Acronym / sponsor ID
- PNOC008 / 170827
- Conditions
- Glioma, Glioma of Brain, Cancer, Pediatric Cancer, Pediatric Brain Tumor, Astrocytoma
- Keywords
- High-grade glioma, whole exome sequencing, WES, genomics, RNA sequencing
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- Pacific Pediatric Neuro-Oncology Consortium, The V Foundation for Cancer Research
- Study start
- 28 Nov 2018 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 1 May 2026 (actual)
- First posted
- 13 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
For children with High-grade gliomas (HGG) including HGG presenting within the midline structures of the brain and spine, outcome remains poor and the majority of children die from this disease. The current 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. This treatment strategy has shown promising results in adult patients with solid tumors and is currently being explored in children with DIPG, neuroblastoma and other solid tumors. The current study will test the efficacy of such an approach in children with HGG for which outcomes remain dismal.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Newly diagnosed HGG (Stratum A) | Experimental | Other: Specialized tumor board recommendationChildren 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. |
| Diffuse midline HGG (Stratum B) | Experimental | Other: Specialized tumor board recommendationChildren 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 may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Patients with newly diagnosed HGG (including midline HGG but excluding DIPG), who undergo tissue collection as part of standard of care. HGG is defined as either World Health Organization (WHO) grade III or IV, or testing positive for H3K27M mutation. Patients with disseminated disease are not eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician. Primary spinal cord tumors are eligible.
- Enrollment within 3 weeks of the start of radiation therapy.
- Start of radiation therapy within 6 weeks from initial tissue diagnosis.
- Age ≤ 21 years
- Karnofsky score ≥ 50 for patients ≥ 16 years of age and Lansky score ≥ 50 for patients ≤15 years of age. Patients who are unable to walk because of paralysis but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
Key exclusion criteria (5 listed)
- Patients who are currently enrolled on another therapeutic clinical trial. Individual cases should be discussed with the study chair.
- Patients who are currently taking any anti-cancer directed therapy. Steroids are not considered anti-cancer therapy. The use of temozolomide during radiation therapy is allowed at standard dosing (maximum 75 to 90 mg/m^2 daily for a total of 42 days). Any other schedule(s) need to be discussed with the study chair.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Female patients of childbearing potential must not be pregnant or breast-feeding. Female patients of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy (as clinically indicated).
- Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy. Telemedicine visits are acceptable.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Specialized tumor board recommendationBased on the molecular profile, the specialized tumor board will determine an individualized treatment recommendation for each patient using up to four FDA approved drugs. In special circumstances, Investigational new drug (IND) study agents may be used. | Other | Diffuse midline HGG (Stratum B), Newly diagnosed HGG (Stratum A) |
Primary outcome
12 month Progression Free Survival (PFS) for Stratum A
Time frame: Up to 12 months
Progression Free Survival will be determined from date of confirmation of response to first evidence of progression or death at the 12 month time point.
12 month Overall Survival (OS) for Stratum B
Time frame: Up to 12 months
Overall survival will be determined from the date of histological diagnosis to time of death at the 12 month time point.
2 secondary outcomes
Frequency of Adverse Events
Time frame: From beginning of enrollment up to 30 days post end of treatment.
Frequency of Serious Adverse Events
Time frame: From beginning of enrollment up to 30 days post end of treatment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2026.
- Individual participant dataSharing planned: Yes. Individual participant data after de-identification.
Related trials for Glioma
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Active, not recruitingPhase 3
EF-41/KEYNOTE D58: Phase 3 Study of Optune Concomitant With Temozolomide Plus Pembrolizumab in Newly Diagnosed Glioblastoma
Glioblastoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 3
Beginning Radiation Immediately With GammaTile at GBM Excision Versus Standard of Care
Glioblastoma
RecruitingPhase 1/2
Testing the Addition of an Anti-cancer Drug, Selinexor, to the Usual Chemotherapy Treatment (Temozolomide) for Brain Tumors That Have Returned After Previous Treatment
Recurrent Glioblastoma, IDH-Wildtype, Recurrent MGMT-Methylated Glioblastoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sabine Mueller, MD, PhD, MAS principal investigator
University of California, San Francisco
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Specialized tumor board recommendation in people with Glioma and Glioma of Brain, run by University of California, San Francisco at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
12 month Progression Free Survival (PFS) for Stratum A (time frame: Up to 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.