Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Refractory AAV (NCT06508346) | Clinical Trial Compass
WithdrawnNot Applicable
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Refractory AAV
Stopped: no participants enrolled
0Started 2024-08-01
Plain-language summary
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset refractory ANCA-Associated Vasculitis.
Who can participate
Age range
5 Years – 25 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age:5-25 years old(including threshold);
. Diagnosed with AAV according to the 2022EULAR/ACR AAV classification criteria；despite of the Treatment with glucocorticoids (more than 1mg/kg/ day), cyclophosphamide, and rituximab for at least 3 months, still cannot achieve sustained response or disease recurred after response; Or use glucocorticoid combined with cyclophosphamide/rituximab plus more than one of the other immunosuppressants (including azathioprine, moxophenolate, methotrexate, leflunomide, tacrolimus, cyclosporine, beliuzumab, etc.) ≥3months，still failed to achieve sustained remission or relapsed after remission; Or meet the diagnostic criteria for severe vasculitis, clinical routine treatment is ineffective, the benefit is judged by the investigator to outweigh the risk, and the patient or guardian has fully informed consent, can be considered for inclusion。
. patient \<18 years old：PVAS≥15（total 63）；≥18 years old: BVAS≥15(total 63)
. The functions of important organs are basically normal: Cardiac function: Left ventricular ejection fraction (LVEF) ≥55% with no obvious abnormality in electrocardiogram; Renal function: eGFR≥30ML/min/1.73m2； Liver function: Asparagus cochinchinensis transase (AST) and Alanine Aminotransferase (ALT)≤3.0 ULN, Total Bilirubin (TBIL) in serum ≤2.0×ULN; Lung function: No serious lung lesions, SpO2≥92%;
. Met the standards of leukapheresis or intravenous blood collection, No contraindication for cell collection;
. Negative pregnancy test for female Subjects of childbearing age, agree to take effective contraceptive measures the first year after CAR-T infusion;
. Participants or their guardians agrees to participate in the clinical trial and sign the informed consent form which indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial for CAR-T cell therapy in children with refractory ANCA-associated vasculitis has been withdrawn — do you know why it was pulled, and does that affect whether CAR-T therapy is still a realistic option for my child?
2Since this study was withdrawn before completing enrollment, is there any other ongoing or upcoming trial investigating CAR-T cells or anti-CD19 approaches for childhood-onset ANCA-associated vasculitis that we should look into?
3Because this trial was listed as Phase NA and focused primarily on safety, what does that tell us about how early-stage this treatment approach still is, and how does that uncertainty compare to the risks of staying on our current treatment plan?
4Given that my child's vasculitis has been refractory — meaning it hasn't responded to standard treatments — what are the best available alternatives we should be actively considering right now while this trial is no longer enrolling?
5Are there any centers or specialists you would recommend consulting who have experience treating refractory childhood-onset ANCA-associated vasculitis with newer therapies like CAR-T, even outside of a formal clinical trial setting?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The safety of CAR-T cell in refractory childhood-onset ANCA-Associated Vasculitis
Timeframe: 3 months and 6 months
Trial details
NCT IDNCT06508346
SponsorThe Children's Hospital of Zhejiang University School of Medicine
. Central nervous system (CNS) disease: CNS neurolupus requires intervention within 60 days);
. Pulmonary hemorrhage that need for pulmonary ventilation support for more than 1 week;
. Have a history of congenital heart disease or acute myocardial infarction within 6 months prior to screening; Or severe arrhythmias (including multisource frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with moderate to massive pericardial effusion, serious myocarditis, etc; Or patients with unstable vital signs who need hypertensive drugs；
. Suffer from other diseases that require long-term use of glucocorticoid or high-dose of immunosuppressive agents;
. Uncontrollable infection, or active infection that requires systemic treatment within 1 week prior to screening；
. History of organ transplantation or hematopoietic stem cell transplantation, or ≥Grade 2 GVHD within 2 weeks prior to screening；
. Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal reference value range; Or hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Or positive for human immunodeficiency virus (HIV) antibodies; Or syphilis test positive; Or cytomegalovirus (CMV) DNA test positive;