Actively Recruiting

Phase 1
Age: 1Year - 26Years
All Genders
ID04185038

Phase 1 Study of B7-H3-Specific CAR T Cell Immunotherapy for Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, and Pediatric Recurrent or Refractory Central Nervous System Tumors

Led by Seattle Children's Hospital · Updated on 2026-04-13

90

Participants Needed

1

Research Sites

782 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating a Phase 1 study of a special immune therapy using B7H3-specific CAR T cells in children and young adults with certain brain tumors, including diffuse intrinsic pontine glioma (DIPG), diffuse midline glioma (DMG), and other recurrent or refractory central nervous system (CNS) tumors. The study aims to assess the safety and feasibility of delivering these engineered T cells directly into the brain or tumor cavity using an indwelling catheter, hoping to improve interaction with tumor cells and monitor their effects. Participants will have their own T cells collected and modified to target tumor cells expressing B7H3. They are assigned to one of three treatment groups based on tumor location. Patients with supratentorial tumors receive weekly doses into the tumor cavity, while those with infratentorial or metastatic tumors receive doses into the ventricular system. DIPG patients receive doses every other week into the ventricular system. Treatment courses include multiple doses followed by rest and examination periods, with possible additional courses if tolerated and cells remain available. During the study, participants undergo MRI scans and other evaluations to monitor the distribution of CAR T cells in cerebrospinal fluid and blood, as well as their impact on tumors. Safety is monitored through adverse event assessments up to seven months. The study also evaluates the ability to produce CAR T cells and their movement within the body. Participation may last several months, with ongoing follow-up and possible extended treatment depending on response and safety.

CONDITIONS

Brief Title

Study of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for Diffuse Intrinsic Pontine Glioma/Diffuse Midline Glioma and Recurrent or Refractory Pediatric Central Nervous System Tumors

Who Can Participate

Age: 1Year - 26Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age between 1 and 26 years
  • Diagnosis of refractory or recurrent CNS disease with no standard therapy, or DIPG or DMG at any time after standard therapy
  • Able to tolerate apheresis or have apheresis product available for manufacturing
  • Presence of a CNS reservoir catheter, such as an Ommaya or Rickham catheter
  • Life expectancy of at least 8 weeks
  • Lansky or Karnofsky performance score of 60 or higher
  • If no prior apheresis product, must have stopped and recovered from prior chemotherapy, immunotherapy, and radiotherapy with specified waiting periods
  • Stable or decreasing corticosteroid therapy within 1 week before enrollment with maximum dexamethasone dose of 2.5 mg/m2/day
  • Adequate organ function and laboratory values
  • Agreement to use highly effective contraception if of childbearing or fathering potential
Not Eligible

You will not qualify if you...

  • Grade 3 or higher cardiac dysfunction or symptomatic arrhythmia needing intervention
  • Primary immunodeficiency or bone marrow failure syndrome
  • Evidence of impending brain herniation
  • Grade higher than 3 dysphagia
  • Active malignancy other than the primary CNS tumor under study
  • Severe active infection
  • Currently receiving any anti-cancer agents or chemotherapy
  • Pregnant or breastfeeding
  • Unwilling or unable to consent to a 15-year follow-up period
  • Any condition that would prevent treatment under the study protocol as judged by the investigator

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 7 months

Participants receive up to 15 doses of B7H3-specific CAR T cells delivered via a central nervous system catheter into the tumor resection cavity or ventricular system.

Multiple visits for CAR T cell infusions and monitoring

Follow-up

Duration - Up to 6 months

Participants are monitored for safety, CAR T cell distribution, and disease response after completing treatment.

Periodic visits for assessments and monitoring

Trial Site Locations

Total: 1 location

1

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Actively Recruiting

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Research Team

R

Rebecca Ronsley, MD

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

5

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Frequently Asked Questions

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Published Research Related To This Trial

Tumor inflammation-associated neurotoxicity in children with diffuse intrinsic pontine glioma receiving B7-H3-targeting CAR T cells on BrainChild-03.

Rebecca Ronsley, Michelle Choe, Jason Wright...

https://pubmed.ncbi.nlm.nih.gov/41798119