Actively Recruiting
Loc3CAR: Locoregional Delivery of B7-H3-specific Chimeric Antigen Receptor Autologous T Cells for Pediatric Patients With Primary CNS Tumors
Led by St. Jude Children's Research Hospital · Updated on 2026-05-05
48
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating autologous B7-H3-CAR T cells in this Phase I clinical trial for participants 21 years old or younger with primary central nervous system (CNS) tumors, including relapsed or refractory non-brainstem tumors and diffuse midline gliomas (DMG). The study aims to find the highest safe dose of these specially engineered T cells given directly into the CNS to treat these brain tumors. It also monitors the safety and potential effects of this therapy on brain tumor patients. Participants will receive four infusions of B7-H3-CAR T cells over a four-week period. The cells are administered locoregionally through a CNS reservoir catheter, without prior lymphodepleting chemotherapy. The study uses a 3+3 dose escalation design to identify the maximum tolerated dose. After the treatment period, participants will be followed for one year on this protocol, then continue long-term monitoring for up to 15 years to observe lasting effects and safety. During the study, participants will have regular assessments including safety evaluations and imaging to monitor tumor response four weeks after infusions. Researchers will track neurologic side effects and other toxicities. The primary outcome is determining the maximum tolerated dose within four weeks after the first infusion or seven days after the last infusion. Secondary outcomes include radiographic tumor response. Participants will also undergo performance, organ function, and laboratory testing, and must have a CNS reservoir catheter for treatment delivery. The full study duration includes treatment and extended follow-up, ensuring comprehensive monitoring of patient health and therapy effects.
CONDITIONS
Brief Title
Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 21 years or younger
- Diagnosis of primary CNS tumor
- For Cohort A: evidence of relapsed or refractory non-brainstem CNS tumor
- For Cohort B: diagnosis of diffuse midline glioma with available tumor tissue for B7-H3 or H3K27M evaluation, or presumptive brainstem high-grade neoplasm with imaging
- Life expectancy greater than 12 weeks
- Ability to understand and sign informed consent
- Primary CNS tumor with measurable or evaluable disease
- Tumor is B7-H3 positive for Cohort A; for Cohort B, tumor is B7-H3 positive if non-pontine, or H3K27-altered diffuse midline pontine glioma, or classic DIPG confirmed by imaging
- Completion of standard radiation therapy at least 6 weeks prior to treatment
- Available autologous T-cell product meeting release criteria
- Presence of CNS reservoir catheter or programmable shunt
- First CAR T cell infusion scheduled at least 5 days after CNS surgery
- Discontinuation of specified therapies for required washout periods before enrollment
- Karnofsky or Lansky performance score of 50 or higher
- Adequate organ function and laboratory values
- Agreement to use contraception if of childbearing or child-fathering potential
- Negative pregnancy test and not lactating if female of childbearing potential
- Recovery from prior severe toxicities
- Ability to provide consent for 15 year long-term follow-up
You will not qualify if you...
- Clinically significant medical disorders interfering with treatment or study procedures
- Known primary or acquired immunodeficiency
- HIV positive status
- Severe active infections
- Rapidly progressive disease
- Underlying medical conditions that could limit or confound assessments
- Non-programmable ventricular shunt or reservoir catheter placement compromising therapy or safety
- Unwillingness or inability to consent to 15 year long-term follow-up
- Recent live vaccinations within 30 days
- Uncontrolled hypertension or uncontrolled seizures
- Recent myocardial infarction, unstable angina, severe heart failure, myocarditis, or ventricular arrhythmias within 6 months
- Receiving prohibited therapies during washout periods
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 weeks
Participants receive four infusions of B7-H3-CAR T cells locoregionally via a CNS reservoir catheter over a 4 week period without lymphodepleting chemotherapy.
4 infusions over 4 weeks
Duration - 1 year and then up to 15 years
Participants are followed for safety and treatment effects for 1 year after the final B7-H3-CAR T cell infusion, with continued long-term follow-up up to 15 years post-infusion.
Periodic visits during the first year; long-term follow-up as per institutional protocol
Trial Site Locations
Total: 1 location
1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
T
Tabatha E. Doyle, RN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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