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RECRUITING
NCT07333677
EARLY_PHASE1

In Vivo CAR-T for Refractory Graves' Disease

Sponsor: Shanghai Zhongshan Hospital

View on ClinicalTrials.gov

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.

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

Key Details

Gender

All

Age Range

18 Years - 75 Years

Study Type

INTERVENTIONAL

Enrollment

5

Start Date

2026-01-29

Completion Date

2027-12

Last Updated

2026-03-19

Healthy Volunteers

No

Conditions

Interventions

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.

Locations (1)

Zhongshan Hospital Fudan University

Shanghai, Shanghai Municipality, China