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Can a Tumor's genetic blueprint guide better treatment for kids with brain cancer?

NCT ID NCT03739372

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 11, 2026 · Last updated Aug 12, 2026 · Updated 1 time

Summary

This pilot trial explores whether using a tumor's molecular profile—its gene expression and DNA sequencing—can help doctors choose a personalized treatment plan for children and young adults with high-grade glioma (a type of brain cancer). Participants receive a recommendation from a specialized tumor board, which may include up to four FDA-approved drugs based on the tumor's unique characteristics. The study aims to see if this tailored approach can improve progression-free survival and overall survival at 12 months, while also monitoring safety.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Individualized treatment plan based on molecular profiling, with up to four FDA-approved drugs selected by a specialized tumor board
What this could lead to
If successful, this approach could show that tailoring treatment to each tumor's genetic makeup improves survival in children with high-grade glioma.
What could go wrong
This is a small pilot study, so results may not be conclusive. The approach depends on the accuracy of molecular profiling and the availability of effective drugs.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

44 people

The number who actually took part.

Started

Nov 2018

Finished

May 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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) * 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. Exclusion Criteria: * 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Children's Hospitals and Clinics of Minneapolis

    Minneapolis, Minnesota, 55404, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Johns Hopkins University

    Baltimore, Maryland, 21218, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • The Children's Hospital Of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • University of California, San Diego Rady Children's Hospital

    San Diego, California, 92123, United States

  • University of California, San Francisco

    San Francisco, California, 94158, United States

  • University of Florida

    Gainesville, Florida, 32611, United States

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • Washington University

    St Louis, Missouri, 63110, United States

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