In vivo CAR-T treatment for refractory Graves' disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This early phase 1 trial uses a CD8 antibody‑coated lipid nanoparticle to deliver CD19 CAR‑mRNA into the patient's own T cells in the body, converting them into functional CAR‑T cells. Eligible adults with refractory Graves' disease will receive three to five administrations of HN2301 and be followed closely for thyroid function, TRAb levels, clinical symptoms, and treatment‑related adverse events. The protocol emphasizes safety monitoring and preliminary signals of clinical remission or reduction in antibody levels. The single‑center study is conducted at Zhongshan Hospital, Fudan University in Shanghai.

Why it matters

Who can join: the full rules

Inclusion Criteria (Participants must meet all of the following criteria to be eligible for this study):

* 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 (Participants meeting any of the following criteria will be excluded from the study):

* 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
* Impaired liver function, defined as any of the following: Alanine aminotransferase (ALT) \> 3 × ULN, Aspartate aminotransferase (AST) \> 3 × ULN, Total bilirubin \> 2.5 × ULN.
* Impaired renal function: creatinine clearance rate (CrCl) \< 60 mL/min (Cockcroft-Gault formula).
* Left ventricular ejection fraction (LVEF) \< 55%.
* Coagulation abnormalities, defined as either: International normalized ratio (INR) \> 1.5 × ULN, Prothrombin time (PT) \> 1.5 × ULN
* Pregnant or breastfeeding women.
* Any other condition that, in the opinion of the investigator, would make the participant unsuitable for the study.

Where it's running

• Zhongshan Hospital Fudan University
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Study coordinator: Jingjing JIANG, MD, PhD
• Email: jiang.jingjing@zs-hospital.sh.cn
• Phone: 86-021-64041990

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.