NCT07333677

In Vivo CAR-T for Refractory Graves' Disease

A Safety and Efficacy Study of in Vivo CAR-T (HN2301) for Refractory Graves' Disease

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Shanghai Zhongshan Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

Graves' disease is an autoimmune thyroid disorder characterized by the production of autoantibodies against the thyroid-stimulating hormone receptor (TRAb), leading to excessive thyroid hormone secretion and systemic manifestations. A subset of patients develop refractory disease, failing to achieve durable remission despite prolonged antithyroid therapy. This study aims to evaluate the safety and efficacy of HN2301, an in vivo CAR-T therapy in which host T lymphocytes are engineered and transformed to functional CAR-T cells via CD8 antibody-coated LNP delivery of CD19 CAR-mRNA. Participants with refractory Graves' disease will receive three to five administrations of HN2301 and will be regularly monitored for changes in thyroid function, TRAb levels, clinical response, and treatment-related adverse events. The study will provide preliminary evidence on whether HN2301 can induce sustained remission of refractory Graves' disease.

Conditions

• Graves' Disease

Interventions

Type	Name	Description
BIOLOGICAL	In Vivo CAR-T Therapy	Participants will receive 3 to 5 intravenous administrations of HN2301, given once every 2 days, according to the study dosing regimen.

Timeline

• Start date: 2026-01-29
• Primary completion: 2027-06-01
• Completion: 2027-12-01
• First posted: 2026-01-12
• Last updated: 2026-03-19

Locations

1 site across 1 country: China