Engineered immune cells injected directly into the brain take on childhood tumors
NCT ID NCT05768880
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a new treatment called SC-CAR4BRAIN for children and young adults with certain aggressive brain tumors, including DIPG and DMG. The treatment involves taking a patient's own immune T cells, engineering them in a lab to recognize and attack tumor cells, and then delivering them directly into the fluid-filled spaces of the brain through a small tube. The study aims to see if this approach is safe and to find the best dose for future studies.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- SC-CAR4BRAIN (a type of CAR T cell therapy that targets four proteins on tumor cells)
- What this could lead to
- If it works, this could point toward a new treatment option for children and young adults with hard-to-treat brain tumors like DIPG and DMG.
- What could go wrong
- This is an early Phase 1 trial, so the main goals are safety and finding the right dose. It is too soon to know if it will be effective, and there may be side effects from the treatment or the catheter used to deliver it.
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
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 72 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2023
- Expected to finish
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Dec 2043
An estimate. End dates often move.
- Lead sponsor
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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
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1 year to 26 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: 1. Subjects must be age ≥ 1 and ≤ 26 years (except for the first 3 subjects, who must be age ≥ 12 and ≤ 26 years). 2. Subject disease classified as one of the following: 1. DIPG at any timepoint following completion of standard radiotherapy 2. DMG at any timepoint following completion of standard radiotherapy 3. Evidence of refractory or recurrent CNS disease for which there is no routine therapy, defined by either of the following: i. 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 3. Able to tolerate apheresis or already has an apheresis product available for use in manufacturing 4. 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 5. Life expectancy ≥ 8 weeks 6. Lansky or Karnofsky score ≥ 60. 7. 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. 8. Adequate organ function 9. Adequate laboratory values 10. 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 Exclusion Criteria: 1. Presence of ≥ Grade 3 cardiac dysfunction or symptomatic arrhythmia requiring intervention 2. Presence of primary immunodeficiency/bone marrow failure syndrome 3. Presence of clinical and/or radiographic evidence of impending herniation in the CNS 4. For Arm A subjects only: Presence of \> Grade 3 dysphagia 5. Presence of active malignancy other than the CNS tumor under study 6. Presence of active severe infection, defined as either of the following: 1. Positive blood culture within 48 hours of enrollment, OR 2. Fever \> 38.2ºC AND clinical signs of infection within 48 hours of enrollment 7. Pregnant or breastfeeding 8. 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 9. Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol
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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.
Contacts and locations
Locations
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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