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Diffuse Intrinsic Pontine GliomaPhase 1NCT05768880

Study of B7-H3, EGFR806, HER2, And IL13-Zetakine (Quad) CAR T Cell Locoregional Immunotherapy For Pediatric Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, And Recurrent Or Refractory Central Nervous System Tumors

A Phase 1 study testing SC-CAR4BRAIN in people with Diffuse Intrinsic Pontine Glioma and Diffuse Midline Glioma, run by Seattle Children's Hospital at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This is a Phase 1 study of central nervous system (CNS) locoregional adoptive therapy with SC-CAR4BRAIN, an autologous CD4+ and CD8+ T cells lentivirally transduced to express to express combinations of B7-H3, EGFR806, HER2, and IL13-zetakine chimeric antigen receptors (CAR). Read more about this study

Who is it for?
Ages 1 year to 26 years
What is being tested?
SC-CAR4BRAIN
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
72 participants
Where
1 siteUnited States
Study dates
5 May 2023to 31 Dec 2043
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 26 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Subjects must be age ≥ 1 and ≤ 26 years (except for the first 3 subjects, who must be age ≥ 12 and ≤ 26 years).
  • Subject disease classified as one of the following:
  • DIPG at any timepoint following completion of standard radiotherapy
  • DMG at any timepoint following completion of standard radiotherapy
  • Evidence of refractory or recurrent CNS disease for which there is no routine therapy, defined by either of the following:
  • New site or sites of measurable or evaluable disease by radiographic imaging or histologic confirmation following completion of routine care first-line therapy for which curative salvage therapy is not available or amenable, OR ii. Measurable or evaluable disease that persists following completion of routine care first-line therapy for which curative salvage therapy is not available or amenable
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  • Able to tolerate apheresis or already has an apheresis product available for use in manufacturing
  • CNS reservoir catheter, such as an Ommaya or Rickham catheter, present in the proper location for CNS-directed therapy delivered as specified for BrainChild-04
  • Life expectancy ≥ 8 weeks
  • Lansky or Karnofsky score ≥ 60.
  • If patient does not have previously obtained apheresis product, patient must have discontinued, and recovered from acute toxic effects of, all prior chemotherapy, immunotherapy, and radiotherapy and discontinue the following prior to enrollment:
  • ≥ 7 days post last chemotherapy/biologic therapy administration
  • 3 half lives or 30 days, whichever is shorter post last dose of anti-tumor antibody therapy
  • Must be at least 30 days from most recent cellular infusion
  • All systemically administered corticosteroid treatment therapy must be stable or decreasing within 1 week prior to enrollment with maximum dexamethasone dose of 2.5 mg/m2/day. Corticosteroid physiologic replacement therapy is allowed.
  • Adequate organ function
  • Adequate laboratory values
  • Subjects of childbearing/fathering potential must agree to use highly effective contraception from the time of enrollment through 12 months following the last T cell infusion

Who cannot join

  • Presence of ≥ Grade 3 cardiac dysfunction or symptomatic arrhythmia requiring intervention
  • Presence of primary immunodeficiency/bone marrow failure syndrome
  • Presence of clinical and/or radiographic evidence of impending herniation in the CNS
  • For Arm A subjects only: Presence of > Grade 3 dysphagia
  • Presence of active malignancy other than the CNS tumor under study
  • Presence of active severe infection, defined as either of the following:
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  • Positive blood culture within 48 hours of enrollment, OR
  • Fever > 38.2ºC AND clinical signs of infection within 48 hours of enrollment
  • Pregnant or breastfeeding
  • Subject and/or authorized legal representative unwilling to provide consent/assent for study participation, including participation in the 15-year follow-up period, which is required if CAR T cell therapy is administered
  • Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Seattle Children's Hospital

    Seattle, Washington, 98105

Other trials in United States

About this study

Official title: Phase 1 Study of B7-H3, EGFR806, HER2, And IL13-Zetakine (Quad) CAR T Cell Locoregional Immunotherapy For Pediatric Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, And Recurrent Or Refractory Central Nervous System Tumors

This is a Phase 1 study of central nervous system (CNS) locoregional adoptive therapy with SC-CAR4BRAIN, an autologous CD4+ and CD8+ T cells lentivirally transduced to express to express combinations of B7-H3, EGFR806, HER2, and IL13-zetakine chimeric antigen receptors (CAR). CAR T cells are delivered via an indwelling catheter into the ventricular system in children and young adults with diffuse intrinsic pontine glioma (DIPG), diffuse midline glioma (DMG), and recurrent or refractory CNS tumors.

A child or young adult meeting all eligibility criteria, including having a CNS catheter placed into their ventricular system, and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a second-generation CAR T cell that target B7H3, EGFR806, HER2, and IL13-zetakine on tumor cells.

Read more

Patients will be assigned to 1 of 2 treatment Arms based on the type of their tumor:

  • Arm A is for patients with DIPG (meaning primary disease localized to the pons, metastatic disease is allowed) anytime after standard radiation OR after progression.
  • Arm B is for patients with non-pontine DMG (meaning DMG in other parts of the brain such as the thalamus or spine) anytime after standard radiation OR after progression. This Arm also includes other recurrent/refractory CNS tumors.
Being tested
SC-CAR4BRAIN
Keywords
CNS Tumor, DIPG, DMG, B7-H3, HER2, IL13-zetakine, EGFR806, CAR T cell, pediatric, brain tumor
Medical subject headings
Diffuse Intrinsic Pontine Glioma, Central Nervous System Neoplasms, Recurrence, Brain Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SC-CAR4BRAINBiological

    Courses of weekly intraventricular CNS administered SC-CAR4BRAIN infusions for 3 weeks, then 1 week off

Study groups in NCT05768880
Study groupRoleWhat they receive
Arm A - DIPGExperimentalNot described
Receives: Biological: SC-CAR4BRAIN
Arm B - DMG & recurrent/refractory tumorsExperimentalNot described
Receives: Biological: SC-CAR4BRAIN

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Manufacturing Feasibility

    Number and percent of subjects with sufficient therapeutic product generated to receive two courses on the intended dose regimen

    Measured: 42 days

  2. Safety of SC-CAR4BRAIN

    Establish the safety, defined by the adverse events of fractionated intraventricular CNS administration of adoptive therapy with SC-CAR4BRAIN in children and young adults with DIPG, DMG, or recurrent/refractory CNS tumors

    Measured: 28 days post-final SC-CAR4BRAIN infusion

  3. Dose level

    Establish the maximally tolerated dose regimen (MTDR) and recommended Phase 2 dose regimen (RP2DR) of fractionated intraventricular CNS administered SC CAR4BRAIN infusions.

    Measured: 28 days

  4. Administration feasibility

    Number of subjects meeting criteria for their initial CAR T infusion and number of subjects meeting criteria for at least 2 courses of CAR T infusions

    Measured: 98 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Mar 2023
  2. Study start5 May 2023
  3. Primary completion15 Jan 2028 (estimated)
  4. Study completion31 Dec 2043 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
72 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05768880
Sponsor's protocol ID
BrainChild-04
Status last verified
Oct 2026
First submitted
17 Feb 2023
Last updated
13 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing SC-CAR4BRAIN in people with Diffuse Intrinsic Pontine Glioma and Diffuse Midline Glioma, run by Seattle Children's Hospital at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Manufacturing Feasibility (time frame: 42 days). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05768880.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.

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