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Diffuse Intrinsic Pontine Glioma (DIPG) Clinical Trials

Also called DIPG, diffuse midline glioma, DMG, H3 K27M-mutant glioma, pontine glioma, brainstem glioma

DIPG grows in the pons, the segment of the brainstem that runs breathing, heart rate, swallowing, and the nerves of the face. Unlike many brain tumors, it does not form a solid mass that surgeons can cut out; it threads diffusely through healthy tissue in a region where no tissue can be spared.

FDA-approved medicines

1

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Active trials

42

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Next FDA decision

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FDA calendar

Medicines are those whose current FDA label names Diffuse Intrinsic Pontine Glioma, checked October 14, 2026. Corticosteroids and immune globulins count once each. Download the data

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About Diffuse Intrinsic Pontine Glioma

DIPG grows in the pons, the segment of the brainstem that runs breathing, heart rate, swallowing, and the nerves of the face. Unlike many brain tumors, it does not form a solid mass that surgeons can cut out; it threads diffusely through healthy tissue in a region where no tissue can be spared. That single fact has shaped everything about the disease: diagnosis is made by MRI and, increasingly, a small biopsy, and treatment has historically been limited to radiation, which shrinks the tumor and improves symptoms for a time in most children but does not stop it from returning.

Most DIPG tumors carry a mutation called H3 K27M, and in 2021 the World Health Organization reclassified DIPG within a broader category named diffuse midline glioma, H3 K27-altered. The distinction matters for families because drug approvals and trial eligibility now often hinge on that mutation rather than on the tumor's location, which is one reason biopsy has become more common at diagnosis.

DIPG remains one of the hardest diagnoses in pediatric medicine, and families deserve straight answers: most children live less than a year after diagnosis, and fewer than 1 in 10 live 2 years. That reality is exactly why trial enrollment at diagnosis, not after other options fail, is the standard recommendation from pediatric neuro-oncologists, and why the first drug approval in 2025 mattered so much to this community.

Common Symptoms of Diffuse Intrinsic Pontine Glioma

Recognizing the signs of Diffuse Intrinsic Pontine Glioma early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Double vision or eyes that do not move together
  • Facial weakness or drooping, often on one side
  • Trouble swallowing or slurred speech
  • Weakness in an arm or leg, or loss of balance
  • Morning headaches or headaches with nausea
  • Symptoms that appear and worsen quickly, over weeks rather than months

Who Diffuse Intrinsic Pontine Glioma Affects

Primarily children between ages 5 and 10, affecting boys and girls equally. The broader tumor family it belongs to, diffuse midline glioma, also arises in the thalamus and spinal cord and can occur in adolescents and adults. No environmental or inherited cause has been established.

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Find trials I might fit Answer a few questions and see which open Diffuse Intrinsic Pontine Glioma trials fit your age, location and treatment history.

FDA-Approved Treatments for Diffuse Intrinsic Pontine Glioma

There is currently 1 FDA-approved medication for Diffuse Intrinsic Pontine Glioma. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

dordaviprone
Jazz Pharmaceuticals (developed by Chimerix, acquired by Jazz in April 2025)
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Help Paying for Diffuse Intrinsic Pontine Glioma Treatment

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Diffuse Intrinsic Pontine Glioma Resources

Reputable organizations and medical references for learning more about Diffuse Intrinsic Pontine Glioma, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Diffuse Intrinsic Pontine Glioma

Use this Diffuse Intrinsic Pontine Glioma clinical trial finder to see the 14 studies recruiting patients and 1 opening soon in the United States and worldwide, with eligibility criteria in plain English. Of these, 14 test a treatment and 1 is an observational study or registry; each card says which. These studies play a critical role in advancing care for rare cancers conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

New to trials? What Placebos and Randomization Actually Mean for You (In Plain English) · 4 min readWorried about cost? The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them) · 4 min read
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Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

42 active trials worldwide
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RECRUITINGTreatment trialPhase 1Recently updatedNCT07796061

Blood-Brain Barrier Disruption Using Exablate Magnetic Resonance-Guided Low Intensity Focused Ultrasound With Microbubbles in Combination With T-Cell Infusion in Patients With Newly Diagnosed Diffuse Midline Glioma (LIFT)

Intervention: TAA-T Cell, BBB Disruption

Sponsor: Children's National Research Institute

This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for Diffuse Midline Glioma (DMG), an aggressive brain tumor with a very poor prognosis - an average one-year overall survival. This study combines blood-brain barrier (BBB) disruptio...

Ages 3 Years to 25 Years1 location
Expected to start Oct 2026Updated 1 week agoEst. Dec 2031 (~5y 2m)
RECRUITINGTreatment trialPhase 2Recently updatedNCT06838676

ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas

Intervention: ACT001

Sponsor: Nationwide Children's Hospital · Accendatech USA Inc.

This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.

Ages 12 Months to 39 Years20 locations
Started Jul 2025Updated 3 weeks agoEst. Jul 2028 (~1y 9m)
RECRUITINGTreatment trialPhase 1/2Recently updatedNCT04870944

CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma

Intervention: Biospecimen Collection, Bone Marrow Aspirate, Bone Marrow Biopsy, Echocardiography Test, FACT Complex-targeting Curaxin CBL0137

Sponsor: Children's Oncology Group

This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with solid tumors, including central nervous system (CNS) tumors or lymphoma that has come back (relapsed) or does not respond to treatment (refractory). Drugs, such a...

Ages 12 Months to 21 Years35 locations
Started Jan 2022Updated 1 month agoEst. Dec 2026 (~2 months)
RECRUITINGTreatment trialPhase 1Recently updatedNCT07664176

NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain Tumors

Intervention: NeuroNavigation-Guided Focused Ultrasound (NgFUS), Lumason, Nivolumab

Sponsor: Children's National Research Institute

This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for progressive and relapsed diffuse midline glioma (DMG) and other progressive and relapsed high-grade brain tumors. This study combines intravenous nivolumab therapy infused follow...

Ages 3 Years to 25 Years1 location
Expected to start Sep 2026Updated 1 month agoEst. Dec 2030 (~4y 2m)
NOT YET RECRUITINGTreatment trialPhase 1Recently updatedNCT06946680

IL-8 Receptor-modified CD70 CAR T Cell Therapy in CD70+ Newly Diagnosed and Recurrent Pediatric High-grade Glioma (pHGG) and Newly Diagnosed Diffuse Intrinsic Pontine Glioma (ndDIPG)

Intervention: Ex-Vivo expanded autologous IL-8 receptor (CXCR2) modified CD70 CAR (8R-70CAR) T cells

Sponsor: University of Florida

This is a phase I study to assess the safety and feasibility of IL-8 receptor modified patient-derived activated CD70 CAR T cell therapy in newly diagnosed and recurrent CD70+ Pediatric High-Grade Gliomas (pHGG) and Diffuse Intrinsic Pontine Glioma (ndDIPG)

Ages 4 Years to 30 Years1 location
Expected to start Nov 2026Updated 1 month agoEst. Dec 2030 (~4y 2m)
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Active trial locations45 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Diffuse Intrinsic Pontine Glioma patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Diffuse Intrinsic Pontine Glioma treatments, clinical trial participation, and day-to-day disease management.

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Related Rare Cancers Conditions

Other rare diseases in the rare cancers category. Patients with Diffuse Intrinsic Pontine Glioma may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Diffuse Intrinsic Pontine Glioma Treatments

7 pharmaceutical companies have Diffuse Intrinsic Pontine Glioma in their rare disease portfolio

Frequently Asked Questions About Diffuse Intrinsic Pontine Glioma