Refractory Systemic Lupus ErythematosusPhase 1NCT07432334
CD19 CAR T-Cell Therapy for Refractory Systemic Lupus Erythematosus
A Phase 1 study testing Autologous CD19-Targeted CAR-T Cells in people with Refractory Systemic Lupus Erythematosus, run by Vinmec Research Institute of Stem Cell and Gene Technology at 1 site in Vietnam.
Trial summary
The goal of this Phase I clinical trial is to evaluate the safety and tolerability of autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy in adults with refractory systemic lupus erythematosus who have demonstrated inadequate response to standard-of-care immunosuppressive treatments. Read more about this study
- Who is it for?
- Ages 16 to 55 years
- Conditions the study is for
- Refractory Systemic Lupus Erythematosus
- What is being tested?
- Autologous CD19-Targeted CAR-T Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteVietnam
- Study dates
- 1 Mar 2026to Nov 2027
- 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 16 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 16 to 55 years, male or female
- Diagnosis of systemic lupus erythematosus (SLE) according to the 2019 EULAR/ACR classification criteria with a total score ≥ 10
- SLEDAI-2K score ≥ 8 at screening (with at least 4 points derived from laboratory parameters; excluding points attributable to central nervous system involvement)
- Positive antinuclear antibody (ANA ≥ 1:80) OR positive anti-dsDNA OR positive anti-Sm antibody at screening or documented in medical history
- Refractory systemic lupus erythematosus or refractory lupus nephritis defined as one of the following:
- Refractory SLE:
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- Failure to achieve adequate response, partial response, or stable disease control after ≥ 6 months of standard-of-care therapy (documented compliance). Standard therapy includes corticosteroids plus hydroxychloroquine and at least two of the following: calcineurin inhibitors, cyclophosphamide, mycophenolate mofetil, azathioprine, or B-cell-targeted therapy (e.g., rituximab, belimumab).
- Refractory Lupus Nephritis:
- Persistent active lupus nephritis after two induction regimens, including intravenous cyclophosphamide and mycophenolate mofetil administered for ≥ 6 months (with or without calcineurin inhibitors, rituximab, or belimumab), AND:
- Histopathologic confirmation of Class III or Class IV lupus nephritis, with or without Class V (ISN/RPS 2003 classification); isolated Class V is excluded
- Proteinuria > 1 g/24 hours OR urine protein-to-creatinine ratio > 1 mg/mg
- Adequate organ function:
- ALT ≤ 5 × upper limit of normal; total bilirubin ≤ 34 μmol/L (≤ 2.0 mg/dL)
- Pulmonary function: FVC ≥ 60% predicted OR FEV1 ≥ 60% predicted
- Cardiac function: LVEF ≥ 50%, no uncontrolled arrhythmia, no intracardiac thrombus, no heart failure
- Adequate hematologic parameters:
- Absolute neutrophil count ≥ 0.8 × 10⁹/L (without growth factor support)
- Absolute lymphocyte count ≥ 0.3 × 10⁹/L
- Platelet count ≥ 50 × 10⁹/L
- Hemoglobin ≥ 80 g/L (≥ 8.0 g/dL)
- Ability to provide written informed consent
- Agreement to use effective contraception during the study period (for participants of reproductive potential)
Who cannot join
- History of significant neurologic disorders (e.g., traumatic brain injury, seizure disorder, hemorrhagic conditions, impaired consciousness)
- Significant cardiovascular disease within 3 months prior to screening (e.g., uncontrolled hypertension, NYHA Class III-IV heart failure, severe arrhythmia, unstable angina, myocardial infarction)
- Prior kidney transplantation
- Severe asthma requiring long-term treatment or respiratory failure
- Severe hemolytic anemia requiring transfusion at intervals ≤ 7 days
- Active viral infections (e.g., hepatitis B or C, HIV, tuberculosis, malaria, syphilis, CMV, EBV) or other life-threatening infectious diseases
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- Active bacterial infection confirmed by clinical evaluation, imaging, or laboratory testing
- Use of the following prior to leukapheresis:
- Anti-CD20 therapy, cyclophosphamide, live or attenuated vaccines within 1 month
- Systemic corticosteroids > 10 mg/day (prednisone equivalent), T-cell-targeted therapy (e.g., mycophenolate mofetil, calcineurin inhibitors), immunosuppressive agents, or antimalarial agents within 7 days
- Prior anti-CD19 therapy
- Prior T-cell-based cellular therapy or gene therapy, including CAR-T therapy
- Current or prior malignancy
- Known hypersensitivity to study-related agents
- Pregnant or breastfeeding women
- Active antiphospholipid syndrome (stable antiphospholipid antibody positivity without active APS is permitted)
- Participation in another clinical trial at the time of screening
- Any condition that, in the investigator's judgment, would interfere with protocol compliance or study participation
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Vietnam1 site, 1 recruiting
- Recruiting
Vinmec Research Institute of Stem Cell and Gene Technology
Hanoi, 10000
Liem T Nguyen, PhD, +84 986 565 015, liem.nt@vinuni.edu.vn
About this study
Official title: Evaluation of the Safety and Efficacy of CD19 CAR T-Cell Therapy for the Treatment of Refractory Systemic Lupus Erythematosus: A Phase I Clinical Trial
The goal of this Phase I clinical trial is to evaluate the safety and tolerability of autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy in adults with refractory systemic lupus erythematosus who have demonstrated inadequate response to standard-of-care immunosuppressive treatments.
The primary questions this study aims to address are:
Read more
What is the incidence, nature, and severity of treatment-emergent adverse events following CD19 CAR-T cell infusion? Is administration of CD19 CAR-T cell therapy feasible and tolerable in patients with refractory systemic lupus erythematosus? This study is conducted as a single-arm trial without a comparison group.
Participants will:
Undergo leukapheresis for collection of autologous peripheral blood mononuclear cells Receive a protocol-defined lymphodepleting chemotherapy regimen prior to CAR-T cell infusion Receive a single intravenous infusion of approximately 1.0 × 10⁶ CD19 CAR-T cells per kilogram of body weight Undergo scheduled clinical evaluations, laboratory testing, and longitudinal follow-up to assess safety, tolerability, and clinical parameters
Read the detailed description
This is a Phase I, single-center, open-label clinical trial evaluating the safety of autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy in patients with refractory systemic lupus erythematosus (SLE).
Systemic lupus erythematosus is a chronic autoimmune disease characterized by immune dysregulation and pathogenic autoantibody production, with B lymphocytes playing a central role in disease pathophysiology. Targeting CD19-expressing B cells represents a potential therapeutic strategy for patients with disease refractory to standard immunosuppressive therapies.
Autologous CD19 CAR-T cells will be generated from peripheral blood T cells collected by leukapheresis. Cells will be genetically modified ex vivo to express a CD19-specific chimeric antigen receptor, expanded, and released for clinical administration following protocol-defined quality control testing and regulatory requirements.
Participants will receive a lymphodepleting chemotherapy regimen prior to a single intravenous infusion of CD19 CAR-T cells. Treatment administration and post-infusion monitoring will be conducted according to the protocol-specified safety and observation plan.
Following infusion, participants will be monitored for treatment-emergent adverse events, including CAR-T-associated toxicities such as cytokine release syndrome, immune effector cell-associated neurotoxicity, cytopenias, and infections. Safety evaluations will include serial clinical assessments and laboratory monitoring.
Exploratory assessments will evaluate immunological parameters, including B-cell depletion and reconstitution, autoantibody profiles, and selected biomarkers of disease activity. Participants will undergo longitudinal follow-up to assess early and delayed adverse events and the persistence of immunological effects, in accordance with regulatory guidance for gene-modified cell therapies.
- Conditions
- Refractory Systemic Lupus Erythematosus
- Being tested
- Autologous CD19-Targeted CAR-T Cells
- Keywords
- CD19 CAR-T, Autoimmune Disease
- Medical subject headings
- Lupus Erythematosus, Systemic, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous CD19-Targeted CAR-T CellsBiological
Autologous chimeric antigen receptor T cells targeting CD19, manufactured from participants' peripheral blood T cells collected by leukapheresis. Cells are genetically modified ex vivo to express a CD19-specific CAR, expanded, and administered as a single intravenous infusion following protocol-defined lymphodepleting chemotherapy. Participants undergo post-infusion monitoring for safety and immunological effects according to the study protocol.
| Study group | Role | What they receive |
|---|---|---|
| CD19 CAR-T Cell Therapy | Experimental | Participants will receive autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy following leukapheresis and protocol-defined lymphodepleting chemotherapy. A single intravenous infusion of CD19 CAR-T cells will be administered, with subsequent safety monitoring and follow-up according to the study protocol. Receives: Biological: Autologous CD19-Targeted CAR-T Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of Frequency and Severity of Adverse Events and Serious Adverse Events
Incidence, type, and severity of adverse events (AEs) and serious adverse events (SAEs) graded according to CTCAE v5.0. Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) will be graded according to ASTCT criteria.
Measured: From CAR-T cell infusion through Day 360
Secondary outcomes (6)
Proportion of Participants Achieving Remission According to DORIS Criteria
Proportion of participants achieving complete or partial remission according to the Definitions of Remission in SLE (DORIS) criteria.
Measured: Day 90 and Day 360
Proportion of Participants Achieving Lupus Low Disease Activity State (LLDAS)
Proportion of participants meeting Lupus Low Disease Activity State (LLDAS) criteria.
Measured: Day 90 and Day 360
Proportion of Participants Experiencing Disease Relapse
Proportion of participants with disease relapse after achieving clinical response.
Measured: From Day 90 through Day 360
Manufacturing Success Rate of Autologous CD19 CAR-T Cells
Proportion of enrolled participants for whom autologous CD19 CAR-T cells are successfully manufactured and released for administration
Measured: From leukapheresis through product release, up to 12 days
Peripheral CD19+ B-Cell Depletion and Reconstitution
Quantitative assessment of peripheral blood CD19+ B-cell counts at specified time points following CAR-T cell infusion.
Measured: Baseline, Day 7, Day 14, Day 28, Day 90, Day 180, and Day 360
CAR-T Cell Expansion and Persistence
Quantitative assessment of circulating CAR-T cell levels in peripheral blood at specified time points following infusion.
Measured: Baseline, Day 7, Day 14, Day 28, Day 90, Day 180, and Day 360
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2026
- Study start1 Mar 2026
- Primary completionSep 2027 (estimated)
- Study completionNov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Systemic Lupus ErythematosusStudies of the same condition, open and under way.
- 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
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene Technology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Liem T Nguyen, PhD, Principal investigator, Vinmec Research Institute of Stem Cell and Gene Technology, VinUniversity
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07432334
- Sponsor's protocol ID
- ISC25.03
- Status last verified
- Feb 2026
- First submitted
- 12 Feb 2026
- Last updated
- 9 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Safety and Efficacy of CD19 CAR T-Cell Therapy for the Treatment of Refractory Systemic Lupus Erythematosus: A Phase I Clinical Trial
- ClinicalTrials.gov ID
- NCT07432334
- Acronym / sponsor ID
- ISC25.03
- Conditions
- Refractory Systemic Lupus Erythematosus
- Keywords
- CD19 CAR-T, Autoimmune Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene TechnologyUniversity, hospital or other
- Study start
- 1 Mar 2026 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Nov 2027 (estimated)
- First posted
- 25 Feb 2026 (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.
The goal of this Phase I clinical trial is to evaluate the safety and tolerability of autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy in adults with refractory systemic lupus erythematosus who have demonstrated inadequate response to standard-of-care immunosuppressive treatments.
The primary questions this study aims to address are:
What is the incidence, nature, and severity of treatment-emergent adverse events following CD19 CAR-T cell infusion? Is administration of CD19 CAR-T cell therapy feasible and tolerable in patients with refractory systemic lupus erythematosus? This study is conducted as a single-arm trial without a comparison group.
Participants will:
Undergo leukapheresis for collection of autologous peripheral blood mononuclear cells Receive a protocol-defined lymphodepleting chemotherapy regimen prior to CAR-T cell infusion Receive a single intravenous infusion of approximately 1.0 × 10⁶ CD19 CAR-T cells per kilogram of body weight Undergo scheduled clinical evaluations, laboratory testing, and longitudinal follow-up to assess safety, tolerability, and clinical parameters
Detailed description
This is a Phase I, single-center, open-label clinical trial evaluating the safety of autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy in patients with refractory systemic lupus erythematosus (SLE).
Systemic lupus erythematosus is a chronic autoimmune disease characterized by immune dysregulation and pathogenic autoantibody production, with B lymphocytes playing a central role in disease pathophysiology. Targeting CD19-expressing B cells represents a potential therapeutic strategy for patients with disease refractory to standard immunosuppressive therapies.
Autologous CD19 CAR-T cells will be generated from peripheral blood T cells collected by leukapheresis. Cells will be genetically modified ex vivo to express a CD19-specific chimeric antigen receptor, expanded, and released for clinical administration following protocol-defined quality control testing and regulatory requirements.
Participants will receive a lymphodepleting chemotherapy regimen prior to a single intravenous infusion of CD19 CAR-T cells. Treatment administration and post-infusion monitoring will be conducted according to the protocol-specified safety and observation plan.
Following infusion, participants will be monitored for treatment-emergent adverse events, including CAR-T-associated toxicities such as cytokine release syndrome, immune effector cell-associated neurotoxicity, cytopenias, and infections. Safety evaluations will include serial clinical assessments and laboratory monitoring.
Exploratory assessments will evaluate immunological parameters, including B-cell depletion and reconstitution, autoantibody profiles, and selected biomarkers of disease activity. Participants will undergo longitudinal follow-up to assess early and delayed adverse events and the persistence of immunological effects, in accordance with regulatory guidance for gene-modified cell therapies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD19 CAR-T Cell Therapy | Experimental | Biological: Autologous CD19-Targeted CAR-T CellsParticipants will receive autologous CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy following leukapheresis and protocol-defined lymphodepleting chemotherapy. A single intravenous infusion of CD19 CAR-T cells will be administered, with subsequent safety monitoring and follow-up according to the study protocol. |
Who may qualify
- Ages
- 16 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (22 listed)
- Age 16 to 55 years, male or female
- Diagnosis of systemic lupus erythematosus (SLE) according to the 2019 EULAR/ACR classification criteria with a total score ≥ 10
- SLEDAI-2K score ≥ 8 at screening (with at least 4 points derived from laboratory parameters; excluding points attributable to central nervous system involvement)
- Positive antinuclear antibody (ANA ≥ 1:80) OR positive anti-dsDNA OR positive anti-Sm antibody at screening or documented in medical history
- Refractory systemic lupus erythematosus or refractory lupus nephritis defined as one of the following:
Key exclusion criteria (18 listed)
- History of significant neurologic disorders (e.g., traumatic brain injury, seizure disorder, hemorrhagic conditions, impaired consciousness)
- Significant cardiovascular disease within 3 months prior to screening (e.g., uncontrolled hypertension, NYHA Class III-IV heart failure, severe arrhythmia, unstable angina, myocardial infarction)
- Prior kidney transplantation
- Severe asthma requiring long-term treatment or respiratory failure
- Severe hemolytic anemia requiring transfusion at intervals ≤ 7 days
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 |
|---|---|---|
| Autologous CD19-Targeted CAR-T CellsAutologous chimeric antigen receptor T cells targeting CD19, manufactured from participants' peripheral blood T cells collected by leukapheresis. Cells are genetically modified ex vivo to express a CD19-specific CAR, expanded, and administered as a single intravenous infusion following protocol-defined lymphodepleting chemotherapy. Participants undergo post-infusion monitoring for safety and immunological effects according to the study protocol. | Biological | CD19 CAR-T Cell Therapy |
Primary outcome
Assessment of Frequency and Severity of Adverse Events and Serious Adverse Events
Time frame: From CAR-T cell infusion through Day 360
Incidence, type, and severity of adverse events (AEs) and serious adverse events (SAEs) graded according to CTCAE v5.0. Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) will be graded according to ASTCT criteria.
6 secondary outcomes
Proportion of Participants Achieving Remission According to DORIS Criteria
Time frame: Day 90 and Day 360
Proportion of Participants Achieving Lupus Low Disease Activity State (LLDAS)
Time frame: Day 90 and Day 360
Proportion of Participants Experiencing Disease Relapse
Time frame: From Day 90 through Day 360
Manufacturing Success Rate of Autologous CD19 CAR-T Cells
Time frame: From leukapheresis through product release, up to 12 days
Peripheral CD19+ B-Cell Depletion and Reconstitution
Time frame: Baseline, Day 7, Day 14, Day 28, Day 90, Day 180, and Day 360
CAR-T Cell Expansion and Persistence
Time frame: Baseline, Day 7, Day 14, Day 28, Day 90, Day 180, and Day 360
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 9 Jul 2026.
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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.
Liem T Nguyen, PhD principal investigator
Vinmec Research Institute of Stem Cell and Gene Technology, VinUniversity
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 Autologous CD19-Targeted CAR-T Cells in people with Refractory Systemic Lupus Erythematosus, run by Vinmec Research Institute of Stem Cell and Gene Technology at 1 site in Vietnam.
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 22 inclusion and 18 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 9 Jul 2026. 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 Vietnam. 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 Liem T Nguyen, PhD (+84 986 565 015, liem.nt@vinuni.edu.vn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of Frequency and Severity of Adverse Events and Serious Adverse Events (time frame: From CAR-T cell infusion through Day 360). 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 9 Jul 2026.