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

NCT07333677 EARLY_PHASE1 RECRUITING

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.

Details

Lead sponsor	Shanghai Zhongshan Hospital
Phase	EARLY_PHASE1
Status	RECRUITING
Enrolment	5
Start date	2026-01-29
Completion	2027-12

Conditions

• Graves' Disease

Interventions

• In Vivo CAR-T Therapy

Primary outcomes

• Incidence and severity of Adverse Events (AEs) — From baseline to 3 months after infusion of HN2301
  Assessment of the incidence and severity of treatment-emergent adverse events (AEs) occurring within 3 months after drug administration at the recommended dose.
• Remission of Graves' disease — From baseline to 12 months after infusion of HN2301
  Proportion of remission will be calculated throughout 12 months after initial dose of HN2301. Remission is defined as euthyroid status without anti-thyroid medication.

Countries

China