Actively Recruiting
A Safety and Efficacy Study of in Vivo CAR-T (HN2301) for Refractory Graves' Disease
Led by Shanghai Zhongshan Hospital · Updated on 2026-03-19
5
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Graves' disease is an autoimmune thyroid disorder marked by the production of antibodies against the thyroid-stimulating hormone receptor, causing excessive thyroid hormone release and various symptoms. Some patients have refractory Graves' disease, meaning they do not achieve lasting remission despite extended antithyroid drug treatment. This trial aims to study the safety and effectiveness of HN2301, an in vivo CAR-T therapy, in treating refractory Graves' disease by evaluating its impact on thyroid function and antibody levels. Participants will receive three to five intravenous doses of HN2301, administered once every two days. This investigational treatment involves engineering the body's own T cells through a CD8 antibody-coated lipid nanoparticle delivery system carrying CD19 CAR-mRNA. The treatment period includes these repeated administrations, followed by ongoing monitoring for clinical response and adverse events. During the study, participants will be regularly assessed for changes in thyroid hormone levels, antibody levels including TRAb and thyroid stimulating immunoglobulin, thyroid gland size, and immune cell counts. Safety will be closely monitored from baseline through three months post-treatment, with remission evaluated over 12 months. Researchers will also track the generation of CAR-T cells and inflammatory markers to better understand treatment effects. The total participation may extend to one year after infusion.
CONDITIONS
Brief Title
In Vivo CAR-T for Refractory Graves' Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 75 years, male or female
- Diagnosis of refractory Graves' disease defined by either continuous antithyroid drug therapy for 3 or more years without remission or at least 2 relapses after stopping therapy
- Positive serum thyroid-stimulating hormone receptor antibody (TRAb)
- Willingness to use effective contraception for 12 months after receiving study treatment
- Voluntary agreement to participate, 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
- Presence or suspicion of uncontrolled active infection
- History of major organ or bone marrow transplantation
- Significant heart disease such as angina, heart attack, heart failure, or serious arrhythmias
- Receipt of any mRNA-LNP or lipid nanoparticle-based therapy within the past 2 years
- Receipt of live vaccine within 30 days before screening
- History of cancer
- Positive tests for hepatitis B, hepatitis C, HIV, or syphilis with active infection markers
- Psychiatric disorders or severe cognitive impairment
- Hematologic dysfunction including low neutrophil, hemoglobin, or platelet counts
- Impaired liver function defined by elevated liver enzymes or bilirubin
- Impaired kidney function with creatinine clearance below 60 mL/min
- Left ventricular ejection fraction below 55%
- Blood clotting abnormalities
- Pregnant or breastfeeding women
- Any other condition that investigator deems unsuitable for the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 6 to 10 days
Participants will receive 3 to 5 intravenous administrations of in vivo CAR-T therapy (HN2301), given once every 2 days according to the study dosing regimen.
3 to 5 visits (in-person) for administrations every 2 days
Duration - Up to 12 months after treatment
Participants are monitored for safety and efficacy outcomes, including adverse events and disease remission, for up to 12 months after the last infusion.
Regular visits during 12 months follow-up period
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
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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