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CAR T-cell Therapy

CAR T Cell Therapy for Pediatric Brain Cancer

Phase 1

Recruiting

Research Sponsored by Seattle Children's Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age ≥ 1 and ≤ 26 years

Diagnosis of refractory or recurrent CNS disease for which there is no standard therapy, or diagnosis of DIPG or DMG at any time point following completion of standard therapy

Must not have

Presence of active severe infection

Receiving any anti-cancer agents or chemotherapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 7 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new cancer treatment for children and young adults with brain tumors. T cells are collected from the patient and engineered to create a CAR T cell that targets B7H3-expressing tumor cells. The patient is then administered the CAR T cells via an indwelling catheter, and the treatment is given in two courses. The hypothesis is that this new treatment is safe and effective.

Who is the study for?

This trial is for children and young adults aged 1 to 26 with specific brain tumors like DIPG, DMG, or other recurrent CNS tumors without standard treatment options. They must be able to undergo apheresis (a procedure to collect T cells), have a catheter in their CNS for drug delivery, expect to live at least 8 weeks, have decent performance status scores (Lansky/Karnofsky ≥60), recovered from prior treatments, stable on low-dose steroids, good organ function and lab values.

What is being tested?

The study tests B7-H3-specific CAR T cell therapy delivered directly into the brain or ventricular system through an indwelling catheter. It's divided into three arms based on tumor location/type. Patients receive two courses of treatment with weekly doses of engineered T cells targeting B7H3-expressing tumor cells. The goal is safe administration and interaction of these CAR T cells with the tumor.

What are the potential side effects?

Potential side effects include reactions related to immune response such as inflammation around the site where the CAR T cells are administered or throughout the body, symptoms due to cytokine release syndrome (fever, fatigue), neurological events (headaches, confusion), and potential risks associated with inserting a catheter into the brain.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 1 and 26 years old.

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My brain cancer has returned or is not responding to treatment, or I have DIPG/DMG after finishing standard therapy.

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I can undergo apheresis or have apheresis material ready for use.

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I can care for myself but may need occasional help.

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My steroid treatment dose has been stable or decreasing for the last week, and does not exceed 2.5 mg/m2/day.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently suffering from a severe infection.

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I am currently on cancer treatment drugs.

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I am showing signs of a possible herniation.

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I have cancer in parts of my body other than my brain.

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I have a condition that affects my immune system or bone marrow.

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I have severe heart issues or irregular heartbeats needing treatment.

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I have severe difficulty swallowing.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 7 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 7 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 7 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Establish the feasibility, defined by the ability to produce and administer CAR T cell product, of B7H3-specific CAR T cell product infusions delivered by a central nervous system (CNS) catheter into the tumor resection cavity or ventricular system

Establish the safety, defined by the adverse events, of B7H3-specific CAR T cell infusions delivered by a central nervous system (CNS) catheter into the tumor resection cavity or ventricular system

Secondary study objectives

Assess the distribution of CNS-delivered B7H3-specific CAR T cells distribution within the cerebrospinal fluid (CSF) and peripheral blood

Assessment of disease response of B7H3-expressing DIPG and DMG tumors to B7H3 specific CAR T cell therapy delivered into the CNS

Assessment of disease response of B7H3-expressing refractory or recurrent central nervous system (CNS) tumors to B7H3 specific CAR T cell therapy delivered into the tumor cavity or into the CNS

Other study objectives

Quantitative biomarker assessment of anti tumor CAR T cell functional activity