A Phase II Study Assessing a Second Infusion of Tisagenlecleucel CAR T-Cell Therapy in Children and Young Adults with B-Cell Acute Lymphoblastic Leukemia

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Full Title

Phase II Open Label Multicenter Study of Early Reinfusion of Tisagenlecleucel to Promote Durable B-Cell Aplasia in Pediatric and Young Adult Patients with Relapsed/Refractory CD19-Positive B-Cell Acute Lymphoblastic Leukemia (REFUEL)

Purpose

The purpose of this study is to assess the safety and effectiveness of giving a second infusion of tisagenlecleucel CAR T-cell therapy to children and young adults with B-cell acute lymphoblastic leukemia (B-ALL). CAR T-cell therapy is a type of cellular therapy that involves using a patient’s own T cells (a type of white blood cell), genetically modifying them in the laboratory to recognize a protein on cancer cells, and multiplying them. The modified T cells, known as “chimeric antigen receptor” (CAR) T cells, are then returned to the patient to find and kill cancerous cells throughout the body.

Tisagenlecleucel targets proteins found on the surface of B cells, allowing the CAR T cells to destroy the B cells. Because the targeted protein is found on all B cells, both cancerous and non-cancerous B cells are killed. This condition of low B-cell numbers or no B cells is called “B-cell aplasia” (BCA) and is an expected result of successful treatment with CAR T-cell therapy. BCA indicates that a patient’s CAR T cells are continuing to multiply and work in the body.

Researchers are doing this study to see if giving a second dose of tisagenlecleucel 1 to 2 months after the first dose can keep participants in BCA for at least 6 months after their first infusion. Tisagenlecleucel is given intravenously (by vein).

Eligibility

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have B-ALL that was treated with a first infusion of tisagenlecleucel, and there must have been enough CAR T cells produced for them to receive more than one dose.
  • Patients must be in a state of BCA and have no evidence of B-ALL.
  • Participants must have recovered from the serious side effects of the first tisagenlecleucel treatment.
  • This study is for people under age 26.

Contact

For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.

Protocol

22-220

Phase

II

Disease Status

Relapsed or Refractory

Investigator