Actively Recruiting
An Exploratory Study of Anti-CD19/BCMA CAR Gene Vector Injection for Relapsed and Refractory Immune Thrombocytopenia
Led by Anhui Provincial Hospital · Updated on 2025-08-08
18
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying an investigational gene therapy called Anti-CD19/BCMA CAR gene vector injection for adults with relapsed and refractory immune thrombocytopenia (ITP), a condition where the immune system attacks platelets. This open-label, single-site, dose-escalation study aims to assess the safety and efficacy of this treatment in up to 18 participants. The research is sponsored by Anhui Provincial Hospital and monitors outcomes over a one-year period. Participants receive a single intravenous infusion of the Anti-CD19/BCMA CAR gene vector, with doses ranging from 1.0 to 4.0 million transducing units per kilogram. This experimental treatment is given once, and the study does not include placebo or comparison groups. The study focuses on how well the gene vector persists and the overall remission rate following treatment, alongside monitoring for adverse events. During the study, participants will undergo safety assessments, including tracking adverse events for up to one year. Researchers will monitor the persistence of the gene therapy in the body and evaluate the remission of ITP symptoms. Participants must agree to contraception requirements and will have regular clinical evaluations and laboratory tests to assess organ function and blood counts. The total study duration spans up to one year after treatment to ensure thorough safety and efficacy evaluation.
CONDITIONS
Brief Title
Anti CD19/BCMA CAR Gene Therapy for Relapsed/Refractory Immune Thrombocytopenia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older, any gender
- Clinical diagnosis of primary immune thrombocytopenia for at least 6 months
- Platelet count below 30 x 10^9/L within 48 hours before study participation
- Positive anti-platelet glycoprotein autoantibodies (e.g., GPIIb/IIIa)
- Prior second-line ITP therapy with ineffective response or relapse
- Bone marrow examination showing megakaryocytosis or normal
- Normal function of major organs, including echocardiography ejection fraction ≥50%, no significant ECG abnormalities, creatinine clearance ≥30 mL/min
- Liver enzymes ALT and AST ≤3 times upper limit of normal, bilirubin and alkaline phosphatase ≤2 times upper limit (with exceptions for Gilbert's syndrome)
- Adequate blood counts: lymphocyte count ≥0.5 x 10^9/L, neutrophil count ≥1 x 10^9/L, hemoglobin ≥60 g/L, platelet count ≥10 x 10^9/L
- Oxygen saturation above 92%
- ECOG performance status 0 to 2
- Males and women of childbearing potential must agree to use effective contraception from informed consent until 1 year post-treatment
- Women of childbearing potential must have a negative pregnancy test and not be breastfeeding
You will not qualify if you...
- Thrombocytopenia caused by other diseases such as myelodysplastic syndrome, aplastic anemia, thrombotic thrombocytopenic purpura
- Bone marrow showing myelofibrosis grade 2 or higher or other primary diseases causing thrombocytopenia
- History of hypersensitivity to study medication components
- Severe heart conditions including NYHA class III-IV heart failure, recent myocardial infarction or heart procedures, ventricular arrhythmias, severe cardiomyopathy
- Malignant disease within 3 years except well-treated cases without active disease
- Recent symptomatic deep vein thrombosis or pulmonary embolism or current anticoagulation
- Participation in other interventional clinical studies within 1 month prior
- Live attenuated vaccine received within 4 weeks prior
- Stroke or seizure within 6 months prior (excluding old lacunar infarction)
- Positive tests for hepatitis B, hepatitis C, HIV, or syphilis with abnormal viral loads
- Known bone marrow stem cell disease
- Any other conditions deemed unsuitable by investigators
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 - Single day
Participants receive a one-time intravenous infusion of the anti-CD19/BCMA CAR gene vector injection.
1 infusion visit (in-person)
Duration - Up to 1 year
Participants are monitored for safety and treatment effects including adverse events and persistence of the gene vector for up to 1 year after treatment.
Regular visits for monitoring during the year following treatment
Trial Site Locations
Total: 1 location
1
Hunan Siweikang Therapeutic Co.Ltd
Changsha, Hunan, China, 410119
Actively Recruiting
Research Team
B
bing xing wang, M.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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