Actively Recruiting
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory ANCA-Associated Vasculitis
Led by The Children's Hospital of Zhejiang University School of Medicine · Updated on 2024-12-02
12
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
Sponsors
T
The Children's Hospital of Zhejiang University School of Medicine
Lead Sponsor
C
Children's Hospital of Chongqing Medical University
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of anti-CD19 CAR-T cells in treating children and young adults aged 5 to 25 with childhood-onset refractory ANCA-Associated Vasculitis (AAV), a serious autoimmune disease that damages small blood vessels and multiple organs, including the lungs and kidneys. Current treatments like glucocorticoids and immunosuppressants often require lifelong use and do not fully cure the disease. This study aims to explore a newer therapy that might offer better disease control. The study involves giving participants anti-CD19 CAR-T cells through intravenous injection. This therapy is being studied because it targets specific immune cells that contribute to AAV. Participants will be monitored for safety and treatment effects at 3 and 6 months, with additional assessments extending to 24 months to observe immune response and disease control over time. During the study, participants will undergo various assessments including organ function tests for heart, kidney, liver, and lung, as well as monitoring of immune cell activity and autoantibody levels. The main focus is on safety within the first 6 months, while effectiveness and long-term disease response will be evaluated over two years. Participants and their guardians will provide consent and be closely followed to understand the impact of this innovative treatment approach.
CONDITIONS
Brief Title
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Refractory AAV
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 5 and 25 years old (including limits)
- Diagnosed with ANCA-Associated Vasculitis according to 2022 EULAR/ACR criteria
- Disease not responding or relapsing after at least 3 months of treatment with glucocorticoids and immunosuppressants
- PVAS score ≥15 if under 18 years old; BVAS score ≥15 if 18 years or older
- Normal important organ functions including heart, kidney, liver, and lung as defined by study parameters
- Eligible for leukapheresis or intravenous blood collection without contraindications
- Negative pregnancy test for females of childbearing age and agreement to use contraception for 1 year after treatment
- Participant or guardian agrees to participate and provides informed consent
You will not qualify if you...
- Previous CAR T cell therapy
- Central nervous system disease requiring intervention within 60 days
- Pulmonary hemorrhage requiring ventilation support for more than 1 week
- History of congenital heart disease, recent myocardial infarction, serious arrhythmias, or unstable vital signs needing medication
- Other diseases requiring long-term glucocorticoids or high-dose immunosuppressants
- Uncontrolled or active infection needing systemic treatment within 1 week prior to screening
- History of organ or stem cell transplantation or recent graft-versus-host disease
- Positive tests for hepatitis B, hepatitis C, HIV, syphilis, or cytomegalovirus beyond normal levels
- Received live vaccine within 4 weeks before screening
- Positive pregnancy test
- Previous or current cancer
- Participation in another clinical study within 3 months prior to enrollment
- Any other conditions judged 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 - Up to 6 months
Participants receive intravenous injections of anti-CD19 CAR-T cells to treat refractory childhood-onset ANCA-Associated Vasculitis.
Multiple visits over 6 months for treatment administration and monitoring
Duration - Up to 24 months
Participants are monitored for long-term safety, efficacy, cellular kinetics, autoantibody levels, and duration of disease response following treatment.
Periodic visits for assessments up to 24 months
Trial Site Locations
Total: 1 location
1
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
J
Jianhua Mao, PhD
J
Junyi Chen, MD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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