Phase 1 Study of CD70-CAR T-cell Therapy with Chemotherapy for Children and Young Adults Up to 21 Years With Relapsed or Refractory Blood Cancers

What this Trial Is About

Researchers are evaluating the safety and optimal dose of CD70-CAR T cell therapy in children and young adults aged 21 years or younger who have certain difficult-to-treat or returning blood cancers. These include acute myelogenous leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia, lymphoma, and mixed phenotype acute leukemia that test positive for CD70. This phase 1 study aims to find the highest dose patients can tolerate and to observe the treatments activity against cancer cells in bone marrow and other sites. Participants receive a lymphodepleting chemotherapy regimen consisting of fludarabine and cyclophosphamide over several days to prepare the body, followed by a single infusion of their own genetically modified CD70-CAR T cells. The study tests three different dose levels of the CAR T cells to assess safety and tolerability. Mesna is also given to reduce chemotherapy side effects. Treatment is carefully timed, with chemotherapy given on days -4 to -2 and a rest day on day -1, followed by the CAR T cell infusion on day 0 or day 1. During the study, participants are monitored closely for safety and treatment effects, with the primary focus on the maximum tolerated dose assessed within 28 days after the CAR T cell infusion. Researchers will collect data on the therapys impact on leukemia in bone marrow and other disease sites. The study measures participants health status, life expectancy, and performance scores, ensuring they are clinically stable and recovered from any prior treatments such as stem cell transplants. The total monitoring period and follow-up extend over several weeks post-infusion to evaluate outcomes and side effects.

CAR T-cell Therapy Directed to CD70 for Pediatric Patients With Hematological Malignancies