Phase 1 Study of CTX112, a CRISPR-Cas9 Engineered Anti-CD19 T Cell Therapy, for Adults With Difficult-to-Treat Autoimmune Diseases Including Lupus, Systemic Sclerosis, or Inflammatory Myopathy

What this Trial Is About

Researchers are evaluating the safety and preliminary effectiveness of CTX112, a CD19-directed CAR T cell immunotherapy, in adults with difficult-to-treat autoimmune diseases such as systemic lupus erythematosus SLE, systemic sclerosis SSc, and idiopathic inflammatory myopathy IIM. This Phase 1, open-label, multicenter study involves genetically modified allogeneic T cells from healthy donors using CRISPR-Cas9 gene editing technology. Participants receive CTX112 through an intravenous infusion following lymphodepleting chemotherapy. The study may include up to 80 subjects and focuses on escalating doses of the therapy to assess safety. The treatment period is followed by long-term monitoring to evaluate the drugs effects over time. During the study, participants undergo scheduled visits, laboratory tests, and evaluations to monitor safety and treatment response. Researchers will assess pharmacodynamics, pharmacokinetics, and preliminary efficacy for up to 60 months after infusion. The primary outcome is safety within the first 28 days post-infusion, with extended follow-up to understand longer-term effects.

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease