Systemic Lupus ErythematosusPhase 1NCT07233642
Study of Ultra-Fast CD19 CAR-T Therapy for Refractory SLE
A Phase 1 study testing CD19 CAR-T cells in people with Systemic Lupus Erythematosus, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.
Trial summary
This is an investigator-initiated trial aimed at assessing the safety and efficacy of ultra-fast autologous CD19-targeted CAR-T cells in the treatment of refractory systemic lupus erythematosus. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Systemic Lupus Erythematosus
- What is being tested?
- CD19 CAR-T cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- Nov 2025to Sep 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age: ≥ 5 years old, and no gender limitation;
- Diagnosed with SLE according to the 2019 EULAR/ACR SLE classification criteria, and still in moderate to severe disease activity despite ≥3M of high dose glucocorticoids(prednisone≥1mg/kg/d or other equivalent amount of other steriod), hydroxychloroquine and at least 2 DMARDs(include cyclophosphamide, mycophenolate mofetil, azathioprine, methotrexate, cyclosporin, tacrolimus, sirolimus, leflunomide, telitacicept, Beliumab, and rituximab) or intolerant to standard treatments;
- SLEDAI-2K score≥8 points;
- 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;
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- Liver function: AST and ALT≤3.0 ULN, total Bilirubin (TBIL) in serum ≤2.0×ULN;
- Lung function: no serious lung lesions, SpO2≥92%;
- Meet the standards of leukapheresis or intravenous blood collection, and no contraindication for leukapheresis;
- Negative pregnancy test for female subjects of childbearing age, and 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
- Central nervous system (CNS) disease: CNS neurolupus requires intervention within 60 days);
- Severe acute nephritis: patients who have accepted or was undergoing renal replacement therapy within 3 months prior to transfusion; or in the investgator's opinion, patients who is likely to have significant kidney disease within 3 moths of the study which need high dose glucocorticoid (prednisone dose≥1mg/kg/day or equivalent amount of other steriod), cyclophosphamide, or mycophenolate mofetil treatment;
- Have a history of congenital heart disease 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;
- Uncontrollable infection, or active infection that requires systemic treatment within 3 months prior to screening;
- Received organ transplantation or hematopoietic stem cell transplantation within 3 months prior to screening, 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;
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- Suffered from macrophage activation syndrome(MAS) within 1 month prior to screening (except for those whose safety risks have been ruled out by the researcher after treatment);
- Received CAR-T treatment (except for those whose safety risks have been ruled out by the researchers after treatment);
- Suffered from active pulmonary tuberculosis at screening;
- Received live vaccine within 4 weeks prior to screening;
- Positive in Blood pregnancy test;
- Previous or concurrent malignancy;
- Patients who participated in other clinical study within 3 months prior to screening;
- Any other conditions that the investigators deem it unsuitable for the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052
Jianhua Mao, Prof 0571-56555983, maojh88@zju.edu.cn
Xue He, 15088688407, hexue1119@163.com
About this study
Official title: Study of Ultra-Fast Autologous CD19-targeted Chimeric Antigen Receptor T (CAR- T) Therapy for Refractory Systemic Lupus Erythematosus
This is an investigator-initiated trial aimed at assessing the safety and efficacy of ultra-fast autologous CD19-targeted CAR-T cells in the treatment of refractory systemic lupus erythematosus.
Read the detailed description
Systemic lupus erythematosus (SLE) is a serious autoimmune disease that can lead to extensive damage in multiple organs and systems, ultimately resulting in disability and even death.
Currently, the primary treatment for SLE relies on glucocorticoids and immunosuppressants to alleviate symptoms. However, due to the absence of a curative treatment, patients typically need to remain on medication indefinitely. In recent years, biological agents such as belimumab and rituximab have been introduced for the treatment of SLE, but these treatments cannot completely eliminate autoimmune B cells in the bone marrow, leading to unsatisfactory overall outcomes. Furthermore, discontinuing these drugs can lead to disease relapse, and there is still no cure for SLE, leaving patients facing the challenges of lifelong medication and an incurable disease.
CAR-T therapy is an adoptive cell therapy that uses genetic modification technology to reprogram T cells and eliminate target cells expressing diseaserelated antigens through antigen-specific recognition. Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that CD19-targeted CAR-T cells hold significant therapeutic potential for SLE. Compared with traditional CAR-T cells, ultra-fast CAR-T, relying on an innovative CAR-T manufacturing system, can produce CAR-T cells in an extremely short period of time (with a preparation time of only 10 minutes).
The purpose of this study is to assess the safety and efficacy of the ultra-fast autologous CD19-targeted CAR-T cells in the treatment of refractory SLE.
- Conditions
- Systemic Lupus Erythematosus
- Being tested
- CD19 CAR-T cells
- Keywords
- CD19, CAR-T
- Medical subject headings
- Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD19 CAR-T cellsBiological
Three dose groups (1.5×10^5/kg, 5×10^5/kg, 10×10^5/kg) were set up, starting from the low dose group climbing to explore the safe and effective dose.
| Study group | Role | What they receive |
|---|---|---|
| CAR-T treatment group | Experimental | This trial was designed as an open, single-arm, single-center, dose-increasing trial. Receives: Biological: CD19 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety of CAR-T cell therapy in patients with refractory SLE [Safety]
Incidence and severity of AEs and SAEs, including changes in laboratory values, vital signs and other clinical manifestation assessed by CommonTerminology Criteria for Adverse Events (CTCAE) v5.0.
Measured: 3 months
The changes from baseline in SLEDAI-2K [efficacy]
Systemic Lupus Erythematosus Disease Activity Index 2000(SLEDAI-2K) is from 0 to 105 points. The higher score means the stronger disease activity.
Measured: 6 months
The changes from baseline in PGA [efficacy]
Physician Global Assessment(PGA) is a continuous visual analogue scale with 0, 1, 2, and 3 scales. "0" indicates no disease activity and "3" indicates the most severe disease activity.
Measured: 6 months
The changes from baseline in BILAG-2004 [efficacy]
British Isles Lupus Assessment Group Index 2004(BILAG-2004) consists of 8 systems, each of which is classified as A, B, C and D respectively. "A" indicates that the condition is highly active and requires active treatment. "B" indicates that the condition is active and requires close monitoring or symptomatic treatment. "C" indicates a stable condition. "D" indicates that the system is not involved.
Measured: 6 months
The number of patients with SRI-4 response [efficacy]
The definition of SRI-4 response: SLEDAI-2K ≥ 4-Point improvement; PGA with no worsening (<0.3-point increase); BILAG 2004 with no new A domain score and no more than 1 new B domain scores.
Measured: 3 months
The number of patients with LLDAS [efficacy]
The definition of LLDAS: SLEDAI-2K ≤ 4 and no disease activity in major organs (kidneys, central nervous system, heart and lungs), and no vasculitis or fever; no new disease activity symptoms were added compared with previous disease assessments; PGA ≤ 1; irrespective of serology; with permitted use of low-dose glucocorticoids (prednisolone ≤ 7.5 mg/day), and/or stable immunosuppressives and biologics.
Measured: 6 months
The number of patients with DORIS [efficacy]
The definition of DORIS: SLEDAI-2K = 0 ; PGA) < 0.5 ; irrespective of serology; with permitted use of antimalarials, low-dose glucocorticoids (prednisolone ≤ 5 mg/day), and/or stable immunosuppressives and biologics.
Measured: 6 months
Secondary outcomes (11)
The number of remission of lupus nephritis [efficacy]
The degree of remission of lupus nephritis includes complete response(CR), primary efficacy renal response(PERR) and partial response(PR).
Measured: 6 months
The changes of anti-ds-DNA antibody after infusion [efficacy]
Measured: 6 months
The changes of 24h urine protein after infusion [efficacy]
Measured: 6 months
The changes of C3 and C4 after infusion [efficacy]
Measured: 6 months
Cmax of CAR-T cells [PK parameter]
The peak plasma concentration (Cmax) of amplified CAR-T cells in peripheral blood after infusion.
Measured: 3 months
Tmax of CAR-T cells [PK parameter]
The time of amplified CAR-T cells in peripheral blood to reach the maximum concentration (Tmax).
Measured: 3 months
AUC28d/90d of CAR-T cells [PK parameter]
The area under the plasma concentration-time curve from 28 to 90 days after infusion (AUC28d/90d).
Measured: 3 months
The degree of B cell depletion [PD parameter]
The degree of B cell depletion at various time points.
Measured: 3 months
The concentration levels of IL-6 [PD parameter]
CAR-T-related serum cytokines include IL-6.
Measured: 3 months
The concentration levels of CRP [PD parameter]
Measured: 3 months
The concentration levels of ferritin [PD parameter]
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2025
- Study startNov 2025 (estimated)
- Primary completionMay 2026 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Lupus ErythematosusStudies 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Treatment of patients with refractory systemic lupus erythematosus using ultra-fast CD19-targeted CAR-T cells
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mao Jianhua, The Children's Hospital of Zhejiang University School of Medicine
- Investigators
- Jianhua Mao, MD, Principal investigator, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07233642
- Sponsor's protocol ID
- PBC097
- Status last verified
- Nov 2025
- First submitted
- 28 Sep 2025
- Last updated
- 18 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 18 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Ultra-Fast Autologous CD19-targeted Chimeric Antigen Receptor T (CAR- T) Therapy for Refractory Systemic Lupus Erythematosus
- ClinicalTrials.gov ID
- NCT07233642
- Acronym / sponsor ID
- PBC097
- Conditions
- Systemic Lupus Erythematosus
- Keywords
- CD19, CAR-T
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- The Children's Hospital of Zhejiang University School of MedicineUniversity, hospital or other
- Study start
- Nov 2025 (estimated)
- Primary completion
- May 2026 (estimated)
- Study completion
- Sep 2028 (estimated)
- First posted
- 18 Nov 2025 (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 ultra-fast autologous CD19-targeted CAR-T cells in the treatment of refractory systemic lupus erythematosus.
Detailed description
Systemic lupus erythematosus (SLE) is a serious autoimmune disease that can lead to extensive damage in multiple organs and systems, ultimately resulting in disability and even death.
Currently, the primary treatment for SLE relies on glucocorticoids and immunosuppressants to alleviate symptoms. However, due to the absence of a curative treatment, patients typically need to remain on medication indefinitely. In recent years, biological agents such as belimumab and rituximab have been introduced for the treatment of SLE, but these treatments cannot completely eliminate autoimmune B cells in the bone marrow, leading to unsatisfactory overall outcomes. Furthermore, discontinuing these drugs can lead to disease relapse, and there is still no cure for SLE, leaving patients facing the challenges of lifelong medication and an incurable disease.
CAR-T therapy is an adoptive cell therapy that uses genetic modification technology to reprogram T cells and eliminate target cells expressing diseaserelated antigens through antigen-specific recognition. Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that CD19-targeted CAR-T cells hold significant therapeutic potential for SLE. Compared with traditional CAR-T cells, ultra-fast CAR-T, relying on an innovative CAR-T manufacturing system, can produce CAR-T cells in an extremely short period of time (with a preparation time of only 10 minutes).
The purpose of this study is to assess the safety and efficacy of the ultra-fast autologous CD19-targeted CAR-T cells in the treatment of refractory SLE.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Treatment of patients with refractory systemic lupus erythematosus using ultra-fast CD19-targeted CAR-T cells
| Arm | Type | Receives |
|---|---|---|
| CAR-T treatment group | Experimental | Biological: CD19 CAR-T cellsThis trial was designed as an open, single-arm, single-center, dose-increasing trial. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Age: ≥ 5 years old, and no gender limitation;
- Diagnosed with SLE according to the 2019 EULAR/ACR SLE classification criteria, and still in moderate to severe disease activity despite ≥3M of high dose glucocorticoids(prednisone≥1mg/kg/d or other equivalent amount of other steriod), hydroxychloroquine and at least 2 DMARDs(include cyclophosphamide, mycophenolate mofetil, azathioprine, methotrexate, cyclosporin, tacrolimus, sirolimus, leflunomide, telitacicept, Beliumab, and rituximab) or intolerant to standard treatments;
- SLEDAI-2K score≥8 points;
- The functions of important organs are basically normal:
- Meet the standards of leukapheresis or intravenous blood collection, and no contraindication for leukapheresis;
Key exclusion criteria (14 listed)
- Central nervous system (CNS) disease: CNS neurolupus requires intervention within 60 days);
- Severe acute nephritis: patients who have accepted or was undergoing renal replacement therapy within 3 months prior to transfusion; or in the investgator's opinion, patients who is likely to have significant kidney disease within 3 moths of the study which need high dose glucocorticoid (prednisone dose≥1mg/kg/day or equivalent amount of other steriod), cyclophosphamide, or mycophenolate mofetil treatment;
- Have a history of congenital heart disease 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;
- Uncontrollable infection, or active infection that requires systemic treatment within 3 months prior to screening;
- Received organ transplantation or hematopoietic stem cell transplantation within 3 months prior to screening, or ≥Grade 2 GVHD within 2 weeks prior to screening;
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 |
|---|---|---|
| CD19 CAR-T cellsThree dose groups (1.5×10^5/kg, 5×10^5/kg, 10×10^5/kg) were set up, starting from the low dose group climbing to explore the safe and effective dose. | Biological | CAR-T treatment group |
Primary outcome
The safety of CAR-T cell therapy in patients with refractory SLE [Safety]
Time frame: 3 months
Incidence and severity of AEs and SAEs, including changes in laboratory values, vital signs and other clinical manifestation assessed by CommonTerminology Criteria for Adverse Events (CTCAE) v5.0.
The changes from baseline in SLEDAI-2K [efficacy]
Time frame: 6 months
Systemic Lupus Erythematosus Disease Activity Index 2000(SLEDAI-2K) is from 0 to 105 points. The higher score means the stronger disease activity.
The changes from baseline in PGA [efficacy]
Time frame: 6 months
Physician Global Assessment(PGA) is a continuous visual analogue scale with 0, 1, 2, and 3 scales. "0" indicates no disease activity and "3" indicates the most severe disease activity.
The changes from baseline in BILAG-2004 [efficacy]
Time frame: 6 months
British Isles Lupus Assessment Group Index 2004(BILAG-2004) consists of 8 systems, each of which is classified as A, B, C and D respectively. "A" indicates that the condition is highly active and requires active treatment. "B" indicates that the condition is active and requires close monitoring or symptomatic treatment. "C" indicates a stable condition. "D" indicates that the system is not involved.
The number of patients with SRI-4 response [efficacy]
Time frame: 3 months
The definition of SRI-4 response: SLEDAI-2K ≥ 4-Point improvement; PGA with no worsening (<0.3-point increase); BILAG 2004 with no new A domain score and no more than 1 new B domain scores.
The number of patients with LLDAS [efficacy]
Time frame: 6 months
The definition of LLDAS: SLEDAI-2K ≤ 4 and no disease activity in major organs (kidneys, central nervous system, heart and lungs), and no vasculitis or fever; no new disease activity symptoms were added compared with previous disease assessments; PGA ≤ 1; irrespective of serology; with permitted use of low-dose glucocorticoids (prednisolone ≤ 7.5 mg/day), and/or stable immunosuppressives and biologics.
The number of patients with DORIS [efficacy]
Time frame: 6 months
The definition of DORIS: SLEDAI-2K = 0 ; PGA) < 0.5 ; irrespective of serology; with permitted use of antimalarials, low-dose glucocorticoids (prednisolone ≤ 5 mg/day), and/or stable immunosuppressives and biologics.
11 secondary outcomes
The number of remission of lupus nephritis [efficacy]
Time frame: 6 months
The changes of anti-ds-DNA antibody after infusion [efficacy]
Time frame: 6 months
The changes of 24h urine protein after infusion [efficacy]
Time frame: 6 months
The changes of C3 and C4 after infusion [efficacy]
Time frame: 6 months
Cmax of CAR-T cells [PK parameter]
Time frame: 3 months
Tmax of CAR-T cells [PK parameter]
Time frame: 3 months
AUC28d/90d of CAR-T cells [PK parameter]
Time frame: 3 months
The degree of B cell depletion [PD parameter]
Time frame: 3 months
The concentration levels of IL-6 [PD parameter]
Time frame: 3 months
The concentration levels of CRP [PD parameter]
Time frame: 3 months
The concentration levels of ferritin [PD parameter]
Time frame: 3 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2025.
- 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.
Jianhua Mao, MD principal investigator
The Children's Hospital of Zhejiang University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CD19 CAR-T cells in people with Systemic Lupus Erythematosus, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.
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 11 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jianhua Mao, MD (13516819071, maojh88@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The safety of CAR-T cell therapy in patients with refractory SLE [Safety] (time frame: 3 months). The study lists 7 primary outcomes in all. 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 18 Nov 2025; AzTrials last refreshed its copy on 6 Nov 2026.