Evaluating Safety and Effectiveness of HN2301 In Vivo CAR-T Therapy for Adults With Refractory Graves Disease

What this Trial Is About

Graves disease is an autoimmune thyroid disorder marked by the production of antibodies against the thyroid-stimulating hormone receptor, causing excessive thyroid hormone release and various symptoms. Some patients have refractory Graves disease, meaning they do not achieve lasting remission despite extended antithyroid drug treatment. This trial aims to study the safety and effectiveness of HN2301, an in vivo CAR-T therapy, in treating refractory Graves disease by evaluating its impact on thyroid function and antibody levels. Participants will receive three to five intravenous doses of HN2301, administered once every two days. This investigational treatment involves engineering the bodys own T cells through a CD8 antibody-coated lipid nanoparticle delivery system carrying CD19 CAR-mRNA. The treatment period includes these repeated administrations, followed by ongoing monitoring for clinical response and adverse events. During the study, participants will be regularly assessed for changes in thyroid hormone levels, antibody levels including TRAb and thyroid stimulating immunoglobulin, thyroid gland size, and immune cell counts. Safety will be closely monitored from baseline through three months post-treatment, with remission evaluated over 12 months. Researchers will also track the generation of CAR-T cells and inflammatory markers to better understand treatment effects. The total participation may extend to one year after infusion.

In Vivo CAR-T for Refractory Graves' Disease