Actively Recruiting
In Vivo CAR-T for Refractory Graves' Disease
Led by Shanghai Zhongshan Hospital · Updated on 2026-03-19
5
Participants Needed
1
Research Sites
95 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
In Vivo CAR-T for Refractory Graves' Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 18 and 75 years, male or female
- Diagnosed with refractory Graves' disease defined by either continuous antithyroid drug treatment for 3 or more years without remission or at least 2 relapses after stopping antithyroid drugs
- Positive serum thyroid-stimulating hormone receptor antibody (TRAb) test
- Willingness to use effective contraception for 12 months after treatment
- Voluntary agreement to participate with signed informed consent and ability to follow study procedures and visits
You will not qualify if you...
- History of severe drug allergy or known allergic predisposition
- Uncontrolled active infection or suspected infection
- History of major organ (heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation
- Significant heart disease such as angina, myocardial infarction, heart failure, or serious arrhythmias
- Received any mRNA-LNP or lipid nanoparticle-based therapy within the last 2 years
- Received a live vaccine within 30 days before screening
- History of cancer
- Positive tests for hepatitis B, hepatitis C, HIV, or syphilis with detectable virus or antibodies
- Psychiatric disorders or severe cognitive impairment
- Blood abnormalities at screening including low neutrophil, hemoglobin, or platelet counts
- Impaired liver function defined by elevated ALT, AST, or total bilirubin
- Impaired kidney function with creatinine clearance below 60 mL/min
- Left ventricular ejection fraction below 55%
- Coagulation problems indicated by elevated INR or prothrombin time
- Pregnant or breastfeeding women
- Any other condition that would make participation unsafe or unsuitable according to the investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Zhongshan Hospital Fudan University
Shanghai, Shanghai Municipality, China, 200032
Actively Recruiting
Research Team
J
Jingjing JIANG, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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