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RECRUITINGEARLY_PHASE1

In Vivo CAR-T for Refractory Graves' Disease

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

NCT07333677•Shanghai Zhongshan Hospital

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.

Eligibility

Age

18 - 75 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Age 18-75 years (inclusive), male or female.
•Refractory Graves' disease, defined as meeting at least one of the following: a) Continuous antithyroid drug (ATD) therapy for ≥3 years without achieving criteria for ATD discontinuation; b) Meeting criteria for ATD discontinuation but experiencing ≥2 relapses after ATD withdrawal.
•Positive serum TRAb.
•Willing to use effective contraception for 12 months after study drug administration.
•Voluntarily agrees to participate in the study, has signed the informed consent form, and is able to comply with study procedures and follow-up requirements.

Exclusion Criteria

•History of severe drug allergy or known allergic predisposition.
•Presence or suspected presence of uncontrolled active infection.
•History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation.
•Presence of significant heart disease, such as angina, myocardial infarction, heart failure, or clinically significant arrhythmias.
•Receipt of any mRNA-LNP product or other lipid nanoparticle (LNP)-based therapy within the past 2 years.
•Receipt of a live vaccine within 30 days prior to screening.
•History of malignant tumors.
•Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA above the detection limit; positive hepatitis C virus (HCV) antibody with detectable HCV RNA; positive human immunodeficiency virus (HIV) antibody; or positive syphilis test.
•Presence of psychiatric disorders or severe cognitive impairment.
•Hematologic dysfuction at screening, defined as any of the following: a. Neutrophil count \< 1.8 × 10⁹/L, b. Hemoglobin \< 110 g/L, c. Platelet count \< 50 × 10⁹/L
+6 more criteria

Locations (1)

Zhongshan Hospital Fudan University

Shanghai, Shanghai Municipality, China

Key Dates

Start Date

January 29, 2026

Primary Completion Date

June 1, 2027

Completion Date

December 1, 2027

Last Updated

March 19, 2026

Enrollment

ESTIMATED participants

Conditions

Graves' Disease

Interventions

In Vivo CAR-T Therapy

BIOLOGICAL

Contacts

Jingjing JIANG, MD, PhD

CONTACT

86-021-64041990 jiang.jingjing@zs-hospital.sh.cn

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 19, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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In Vivo CAR-T for Refractory Graves' Disease

Inclusion Criteria

Exclusion Criteria

Graves' Disease Induced by Epstein-Barr Virus Lytic Reactivation

Early Levothyroxine Post Radioactive Iodine

Role of the Microbiome in Graves' Orbitopathy