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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.

In plain words, diffuse intrinsic pontine glioma is a childhood brain tumor that grows in the pons, a part of the brainstem. This area controls breathing, swallowing and movement, and the tumor weaves through healthy tissue so surgery cannot remove it.

Diffuse Intrinsic Pontine Glioma

Where treatments stand, from ClinicalTrials.gov and FDA labels

FDA review0

No decision pending. FDA calendar

FDA-approved medicines1

Modeyso

Diffuse Intrinsic Pontine Glioma has 1 FDA-approved medicine, 41 open or active clinical trials, and no pending FDA decision.

Labels checked October 21, 2026. Corticosteroids and immune globulins count once each.Download the data

More about Diffuse Intrinsic Pontine Glioma

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.

Keep reading about Diffuse Intrinsic Pontine Glioma

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.

What are the symptoms of Diffuse Intrinsic Pontine Glioma?

These are common symptoms of Diffuse Intrinsic Pontine Glioma, and how strong they are varies from person to person.

  • 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 gets Diffuse Intrinsic Pontine Glioma?

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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Which medicines are FDA-approved for Diffuse Intrinsic Pontine Glioma?

There is currently 1 FDA-approved medication for Diffuse Intrinsic Pontine Glioma.

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.

How to get help paying for Diffuse Intrinsic Pontine Glioma treatment

Charity funds and drugmaker programs for Diffuse Intrinsic Pontine Glioma, checked at the source.

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Side effects people have reported to the FDA

Side effects people have reported to the FDA

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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Diagnosis and genetic testing

Genetic testing for Diffuse Intrinsic Pontine Glioma

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, 13 test a treatment and 2 are observational studies or registries; each card says which. Every trial comes from ClinicalTrials.gov.

Jump to the trial list, the site map or the pipeline timeline.

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.

41 active trials worldwide
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RecruitingTreatment trialPhase 2Recently updatedNCT07680439

Illuminate: A Clinical Study Evaluating CAR T Immune Cell Therapy (BCB-276) for Patients With Diffuse Intrinsic Pontine Glioma (DIPG).

Intervention: BCB-276

Sponsor: BrainChild Bio, Inc

This study will evaluate BCB-276, an investigational B7-H3-targeted Chimeric Antigen Receptor (CAR) T cell therapy, in children and young adults with diffuse intrinsic pontine glioma (DIPG). DIPG is a rare and aggressive brain tumor with limited treatment options. CAR T cell ther...

Ages 1 Year to 26 Years6 locations
Started Sep 2026Updated yesterdayEst. Oct 2028 (~2 years)
RecruitingObservational study, no treatment givenRecently updatedNCT03101813

International Diffuse Intrinsic Pontine Glioma (DIPG)/Diffuse Midline Glioma (DMG) Registry and Repository

Sponsor: Children's Hospital Medical Center, Cincinnati

Doctors and other medical scientists want learn about the biology of DIPG/DMG and to develop better ways to diagnose and treat patients with DIPG/DMG. To do this, they need more information about the characteristics of DIPG/DMG tumors. Therefore, they want to establish a central ...

Ages not specified1 location
Started Jun 2012Updated 3 days agoEst. Jun 2037 (~10y 8m)
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 6 days 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)
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
+37 more

Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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Diffuse Intrinsic Pontine Glioma patient communities

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Companies developing Diffuse Intrinsic Pontine Glioma treatments

Drugmakers that list Diffuse Intrinsic Pontine Glioma in their rare disease portfolio, 7 in all.

Questions people ask about Diffuse Intrinsic Pontine Glioma