ANCA Associated VasculitisNCT06508346
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Refractory AAV
An observational study of ANCA Associated Vasculitis and CAR-T Cell Therapy, run by The Children's Hospital of Zhejiang University School of Medicine.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 25 years
- Conditions the study is for
- ANCA Associated Vasculitis, CAR-T Cell Therapy
- What is being tested?
- anti-CD19-CAR-T cells
- Type of treatment
- Injection or infusion
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Aug 2024to 31 Jul 2027
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The trial consists of two phases, Dose Exploration (Part A) and Dose Expansion (Part B). In Part A, two dose groups (0.3×10^5/kg, 1×10^5/kg,) are set up, starting from the low dose group to explore the safe and effective dose. If the optimal effective dose is still not explored in the highest dose group, the dose can be increased according to the situation, and the highest dose is no more than 3×10^5/kg. Upon the completion of Part A, the optimal dose is selected by the comprehensive judgment of the investigator and the technical partner to enter into the Part B stage. with this dose , anther 3 cases are enrolled to continue to validate the safety and efficacy. A total enrollment of 9-12 patients is expected in the whole process of the trial.
Who may be able to join
- 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.
Who cannot join
- Received CAR T cell therapy previously;
- 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；
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- 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;
- Received live vaccine within 4 weeks before screening;
- Tested positive in Blood pregnancy test；
- Previous or concurrent malignancy；
- Patients who participated in other clinical study within 3 months prior to enrollment;
- Any other conditions that the investigators deem it unsuitable for the study
About this study
Official title: Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory ANCA-Associated Vasculitis
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.
Read the detailed description
The anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis （AAV ）is one of the most devastating and potentially fatal autoimmune diseases, characterized by involvement of small blood vessels (arterioles, tiny arteries, tiny static veins, and capillaries) and the presence of ANCA. The disease lead to extensive damage in multiple organs and systems,such as pulmonary hemorrhage and rapidly progressive glomerulonephritis (RPGN),ultimately resulting in disability and even death.Children with AAV are particularly at risk of organ damage, especially to the kidneys, and tend to more serious than that in adults.
Currently, the primary treatment for AAV relies on glucocorticoids and immunosuppressants to alleviate symptoms. However, due to the absence of a curative treatment, patients often require lifelong medication. In recent years, biological agents such as rituximab have been introduced for the treatment of AAV, but still cannot completely eliminate autoimmune B cells in the bone marrow, leading to unsatisfactory overall outcomes. Furthermore, stopping the drugs can lead to relapse, and there is still no cure for AAV, leaving patients facing the challenges of lifelong medication and an incurable disease.
Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that targeted CD19 CAR-T cells hold significant therapeutic potential for SLE. These cells effectively slow down the pathological progression of SLE and can also effectively treat severe cases. Furthermore, targeted CD19 CAR-T cells are also expected to restore the immune system in SLE patients, potentially allowing them to discontinue lifelong medication and avoid serious long-term side effects of drugs like hormones and immunosuppressants. Studies have reported that CAR-T has a good therapeutic effect on a variety of autoimmune diseases such as systemic sclerosis and idiopathic inflammatory dermatomyositis.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of childhood-onset refractory AAV.
- Conditions
- ANCA Associated Vasculitis, CAR-T Cell Therapy
- Being tested
- anti-CD19-CAR-T cells
- Keywords
- AAV, ANCA Associated Vasculitis, CAR-T cell therapy
- Medical subject headings
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-CD19-CAR-T cellsBiological
Intravenous injection
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety of CAR-T cell in refractory childhood-onset ANCA-Associated Vasculitis
The number of occurrence and proportion of adverse events and serious adverse events that occurred
Measured: 3 months and 6 months
Secondary outcomes (5)
The efficiency of CAR-T cell in refractory childhood-onset AAV
Percentage of patients achieving 50% response 100% response by the 3-month time
Measured: 3 months and 6 months
The efficiency of CAR-T cell in refractory childhood-onset AAV
the assessment of disease activity using PVAS/BVAS 3.0 ,the asseeement of the Irreversible damage by PVDI/VDI,and the changes in laboratory values
Measured: 3 months
Cellular kinetics
CAR transgene levels by quantitative polymerase chain reaction (qPCR) in peripheral blood.
Measured: 6 months
Autoantibody detection
Autoantibody detection up after CD19 CAR-T cells infusion.
Measured: 24 months
Duration of disease response (DOR)
the time between the first investigator assessment of remission and the first investigator assessment of progression or death from any cause
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2024
- Study start1 Aug 2024
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Jul 2027 (estimated)
Why it stopped: no participants enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ANCA Associated VasculitisStudies of the same condition, open and under way.
- More trials run by The Children's Hospital of Zhejiang University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital of Chongqing Medical University
- Responsible party
- Principal investigator, Mao Jianhua, The Children's Hospital of Zhejiang University School of Medicine
- Investigators
- Jianhua Mao, PhD, Principal investigator, The Children's Hospital of Zhejiang University School of MedicineMo Wang, PhD, Principal investigator, Children's Hospital of Chongqing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06508346
- Sponsor's protocol ID
- 2024-IRB-0163-P-01
- Status last verified
- Jun 2026
- First submitted
- 14 Jul 2024
- Last updated
- 16 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory ANCA-Associated Vasculitis
- ClinicalTrials.gov ID
- NCT06508346
- Acronym / sponsor ID
- 2024-IRB-0163-P-01
- Conditions
- ANCA Associated Vasculitis, CAR-T Cell Therapy
- Keywords
- AAV, ANCA Associated Vasculitis, CAR-T cell therapy
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: no participants enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- The Children's Hospital of Zhejiang University School of MedicineUniversity, hospital or other
- Collaborators
- Children's Hospital of Chongqing Medical University
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Jul 2027 (estimated)
- First posted
- 18 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis （AAV ）is one of the most devastating and potentially fatal autoimmune diseases, characterized by involvement of small blood vessels (arterioles, tiny arteries, tiny static veins, and capillaries) and the presence of ANCA. The disease lead to extensive damage in multiple organs and systems,such as pulmonary hemorrhage and rapidly progressive glomerulonephritis (RPGN),ultimately resulting in disability and even death.Children with AAV are particularly at risk of organ damage, especially to the kidneys, and tend to more serious than that in adults.
Currently, the primary treatment for AAV relies on glucocorticoids and immunosuppressants to alleviate symptoms. However, due to the absence of a curative treatment, patients often require lifelong medication. In recent years, biological agents such as rituximab have been introduced for the treatment of AAV, but still cannot completely eliminate autoimmune B cells in the bone marrow, leading to unsatisfactory overall outcomes. Furthermore, stopping the drugs can lead to relapse, and there is still no cure for AAV, leaving patients facing the challenges of lifelong medication and an incurable disease.
Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that targeted CD19 CAR-T cells hold significant therapeutic potential for SLE. These cells effectively slow down the pathological progression of SLE and can also effectively treat severe cases. Furthermore, targeted CD19 CAR-T cells are also expected to restore the immune system in SLE patients, potentially allowing them to discontinue lifelong medication and avoid serious long-term side effects of drugs like hormones and immunosuppressants. Studies have reported that CAR-T has a good therapeutic effect on a variety of autoimmune diseases such as systemic sclerosis and idiopathic inflammatory dermatomyositis.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of childhood-onset refractory AAV.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 5 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The trial consists of two phases, Dose Exploration (Part A) and Dose Expansion (Part B). In Part A, two dose groups (0.3×10^5/kg, 1×10^5/kg,) are set up, starting from the low dose group to explore the safe and effective dose. If the optimal effective dose is still not explored in the highest dose group, the dose can be increased according to the situation, and the highest dose is no more than 3×10^5/kg. Upon the completion of Part A, the optimal dose is selected by the comprehensive judgment of the investigator and the technical partner to enter into the Part B stage. with this dose , anther 3 cases are enrolled to continue to validate the safety and efficacy. A total enrollment of 9-12 patients is expected in the whole process of the trial.
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (13 listed)
- Received CAR T cell therapy previously;
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| anti-CD19-CAR-T cellsIntravenous injection | Biological | – |
Primary outcome
The safety of CAR-T cell in refractory childhood-onset ANCA-Associated Vasculitis
Time frame: 3 months and 6 months
The number of occurrence and proportion of adverse events and serious adverse events that occurred
5 secondary outcomes
The efficiency of CAR-T cell in refractory childhood-onset AAV
Time frame: 3 months and 6 months
The efficiency of CAR-T cell in refractory childhood-onset AAV
Time frame: 3 months
Cellular kinetics
Time frame: 6 months
Autoantibody detection
Time frame: 24 months
Duration of disease response (DOR)
Time frame: 24 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jianhua Mao, PhD principal investigator
The Children's Hospital of Zhejiang University School of Medicine
Mo Wang, PhD principal investigator
Children's Hospital of Chongqing Medical University
Questions about this trial
What is this clinical trial studying?
An observational study of ANCA Associated Vasculitis and CAR-T Cell Therapy, run by The Children's Hospital of Zhejiang University School of Medicine.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 16 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The safety of CAR-T cell in refractory childhood-onset ANCA-Associated Vasculitis (time frame: 3 months and 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jun 2026.