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Loc3CAR: CAR T-Cell Therapy for Pediatric CNS Tumors

Locoregional Delivery of B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cells for Pediatric Patients with Primary CNS Tumors


CAR T-Cells

Brain Tumor

Diseases Treated:

Brain tumors

Eligibility Overview:

  • Up to 21 years old
  • Either B7-H3–positive relapsed or refractory non-brainstem primary CNS tumor or brainstem high-grade neoplasm
  1. Loc3CAR is a phase 1 clinical trial evaluating the safety and efficacy of autologous B7-H3–specific CAR T–cell therapy for children and young adults with relapsed or refractory brain tumors, or for patients with diffuse intrinsic pontine glioma who have undergone upfront radiation therapy.

    Treatment includes 6 B7-H3 CAR T–cell infusions over 8 weeks.

    B7-H3 CAR T cells will be locoregionally administered via a CNS reservoir catheter without lymphodepletion chemotherapy.

    The study will evaluate the safety and maximum tolerated dose of B7-H3 CAR T cells for 8 weeks. The study will last 1 year, after which patients will enroll in our existing institutional long-term follow-up protocol.

    Primary Objective

    To find the highest dose of B7-H3 CAR T cells that is safe to give patients with primary brain tumors

    Eligibility Criteria

    Inclusion criteria include:

    • Up to 21 years old
    • Primary CNS tumor that meets criteria for either cohort A or B:
      • Cohort A: B7-H3positive relapsed or refractory non-brainstem primary CNS tumor  
      • Cohort B: Brainstem high-grade neoplasm

    Exclusion criteria include:

    • Other clinically significant medical disorders such as serious infections or significant cardiac, pulmonary, hepatic, psychiatric, or other organ dysfunction

    Study Site

    St. Jude Children’s Research Hospital, Memphis, Tennessee

  2. About this study

    New treatments are needed to help children and young adults whose brain cancers return or are hard to treat. CAR T–cell therapy is a type of immunotherapy where the body’s T cells are changed in the lab so that they can attack cancer cells. Loc3CAR is a study that uses CAR T cells to treat children and young adults with brain tumors that have returned or are hard to treat. The changed T cells (Loc3CAR) recognize a protein called B7-H3, found in many childhood brain tumors.

    In this study, we will collect white blood cells (T cells) from the patient. We will change those cells in the lab to make the Loc3CAR cells. The Loc3CAR cells are returned to the patient so they can attack the tumors with a specific type of protein called B7-H3. The Loc3CAR cells are given using a small, flexible tube (catheter) that enters the brain and delivers the cells. An IV is not used.

    Purpose of this clinical trial

    The purpose of this study is to find:

    • The largest dose of Loc3CAR T cells that is safe to give patients with B7-H3positive brain tumors
    • The side effects of Loc3CAR T cells
    • The effect Loc3CAR T cells have on brain tumors

    This trial will help scientists better understand how the immune system fights this kind of tumor. What we learn from this study could help us create better treatments.

    Eligibility overview

    To take part in this study, you must:

    • Have a tumor that is positive for B7-H3
    • Be 21 years old or younger

    Patients must stay in Memphis while getting CAR T cells.

  3. Loc3CAR
    Sponsors: St. Jude Children’s Research Hospital identifier NCT05835687
    Trial Start Date: April 2023
    Estimated Enrollment: 36
    Study Phase: Phase 1
    Conditions: Relapsed/refractory brain tumors, high-grade brainstem neoplasms, DIPG
    Ages: 21 years or younger
    Principal investigators: Christopher DeRenzo, MD; Kelsey Bertrand, MBBS; Giedre Krenciute, PhD
    Study Sites: St. Jude Children’s Research Hospital
    For a consultation or to discuss  St. Jude Physician/Patient Referral Office

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.