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Experimental drug combo aims to fight deadly brainstem glioma

NCT ID NCT02887040

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 3 trial tests whether adding two experimental drugs (Atengenal and Astugenal) to standard radiation therapy helps people aged 3 and older with a newly diagnosed, aggressive brainstem tumor live longer. About 92 participants will be randomly assigned to get either radiation alone or radiation plus the drug combo. The main goal is to see if the combination improves overall survival and to check for side effects.

What this could mean

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

Active substance
Antineoplastons A10 (Atengenal) and AS2-1 (Astugenal)
What this could lead to
If it works, this could point toward a treatment that extends survival for children and young adults with a rare and aggressive brainstem tumor.
What could go wrong
This is an early-stage Phase 3 trial with only 92 participants, so results may not be conclusive. Antineoplastons are controversial and not widely accepted, so the treatment may not prove effective or safe.

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

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 92 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Oct 2025

An estimate. Start dates often move.

Expected to finish

Jun 2026

An estimate. End dates often move.

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

3 to 99 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: * Subjects with Diffuse, Intrinsic Pontine Glioma as defined by the following criteria are eligible: * A characteristic MRI appearance, including variable contrast enhancement after gadolinium administration, diffuse T2/FLAIR signal, and involvement of more than 50% of the pons. * Confirmation of anaplastic glioma (i.e., oligodendroglioma, astrocytoma, oligoastrocytoma) or GBM histology if there is less than 50% involvement of the pons. * Screening evaluation requires a MRI performed within 14 days prior to the start of ANP therapy. Study subjects must be on a fixed dose of steroids for at least five days prior to the screening MRI. If the steroid dose is changed between the date of imaging and the start of treatment, a new baseline MRI is required. All MRIs must be performed at an accredited radiology center. All MRIs should include at a minimum: T1-weighted images pre/post gadolinium administration, fluid attenuated inversion recovery (FLAIR), and T-2 weighted images. * Subjects 3-21 years of age must have a clinical history of disease of less than 6 months and at least two of the following clinical findings: cranial nerve deficit, long tract signs (i.e. hemiparesis) and ataxia are eligible. Subjects \> 21 years of age do not need to meet these criteria. * Subjects must be ≥ 3 years of age. RT is not recommended for subjects less than 3 years of age. * Subjects ≤ 16 years of age with a Lansky performance status of \> 40 are eligible. Subjects \> 16 years of age with a Karnofsky performance status of \> 40 are eligible. * Subjects with organ and marrow function (as defined below) are eligible. * Hemoglobin ≥ 9 g/dL * Leukocytes \> 2000/mm3 * Absolute neutrophil count \>1,000/ mm3 * Serum Na+ ≤ 150 mmol/L * Serum K+ ≤ 5.5 mmol/L * Serum creatinine ≤ 1.5 times institutional upper limit * Platelets \>50,000/ mm3 * Total bilirubin \< 2.5 mg/dL * AST (SGOT) / ALT (SGPT) \<5 times institutional upper limit * At the recommended therapeutic dose, the effects of ANP therapy on the developing human fetus are unknown. For this reason, women of child-bearing potential who agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to protocol study entry and for the duration of the protocol study are eligible. Should a woman become pregnant or suspect she is pregnant while participating in this protocol study, she will inform her treating physician immediately. * Subjects, parents, and/or guardians who are able to understand a written informed consent document, and are willing to sign it, are eligible. Exclusion Criteria: * No type of prior therapy, including other investigational agents, is allowable. A prior diagnostic biopsy or surgical shunt for hydrocephalus is permitted. * Subjects with disseminated disease, multicentric tumors, leptomeningeal disease, or the history of retrotumoral bleeding are not eligible. The screening / baseline MRI includes the spinal cord to rule out leptomeningeal disease. * Subjects with a known history of ganglioglioma are not eligible. * Subjects with a current diagnosis or family history of neurofibromatosis I or II are not eligible. - Subjects with a current diagnosis or family history of neurofibromatosis are not eligible. * Subjects with an uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, uncontrolled hypertension despite maximal medical management (three supine blood pressure measurements ≥ 150/99 taken at least one hour apart) or psychiatric illness/social situations that would limit compliance with protocol study requirements are not eligible. * Subjects with a history of New York Heart Association Class II congestive heart failure are not eligible. * Pregnant women are not eligible because the teratogenic and abortifacient effects of ANP therapy in humans are unknown. Because there is an unknown but potential risk for adverse events in nursing infants secondary to the mother receiving ANP therapy, breastfeeding is discontinued if the mother receives ANP therapy.

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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.

How to take part

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  1. The official record

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