Phase 12 Study of CTX112, a CRISPR-Cas9 Engineered Anti-CD19 T Cell Therapy, in Adults with Relapsed or Refractory Hematologic Autoimmune Diseases Including ITP and Warm Autoimmune Hemolytic Anemia

What this Trial Is About

This trial evaluates CTX112, also called Zugocabtagene geleucel zugo-cel, an allogeneic CD19-targeted CAR T cell therapy, in adults with relapsed or refractory primary Immune Thrombocytopenia ITP and Warm Autoimmune Hemolytic Anemia wAIHA. These autoimmune blood disorders involve the immune system attacking blood cells, causing low platelet counts or anemia. The study aims to assess safety and initial effectiveness in patients who have not responded to other treatments. Participants receive CTX112 through an intravenous infusion following lymphodepleting chemotherapy. This therapy uses genetically modified donor T cells designed to target B cells involved in the autoimmune process. The trial is a single-arm, open-label Phase 12 study enrolling up to 60 adults. The study lasts up to 5 years to monitor outcomes and long-term effects. During the study, participants undergo scheduled visits for safety monitoring, laboratory tests, and assessments of how the therapy affects their disease. Researchers will measure safety outcomes up to 28 days after infusion and track pharmacodynamics, pharmacokinetics, and preliminary efficacy for up to 60 months. Participants agree to contraception guidelines and compliance with trial procedures throughout follow-up.

Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease