Systemic Lupus Erythematous (SLE)Phase 1NCT06710717
Autologous CD19 Car T-Cell Therapy For Severe Refractory Systemic Lupus Erythematosus (SLE)
A Phase 1 study testing CD19 CAR-T cells in people with Systemic Lupus Erythematous (SLE), run by National University of Malaysia at 1 site in Malaysia.
Trial summary
This pilot clinical study aims to evaluate the effectiveness of Chimeric Antigen Receptor (CAR) T-cell therapy in treating severe, refractory systemic lupus erythematosus (SLE), an autoimmune disease driven by autoreactive B-cells. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Systemic Lupus Erythematous (SLE)
- What is being tested?
- CD19 CAR-T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteMalaysia
- Study dates
- 3 Jan 2025to 2 Jan 2029
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- -
- Aged between ≥ 18 to ≤ 65 years Clinical Diagnosis of SLE according to the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria (Fanouriakis et al., 2019) Positive anti-nuclear antibody (ANA) (titer ≥1:80 ), anti-dsDNA (≥30 IU/mL on enzyme-linked immunosorbent assay [ELISA]), or anti-Smith at screening or by documented medical history Active disease (defined by not being in remission according to DORIS criteria or in a low disease activity state [LLDAS]) (Franklyn et al., 2016, van Vollenhoven et al., 2021) With at least one active organ system involvement Persistent active disease with insufficient response to glucocorticoids and at least 2 of the following treatments for at least 3 months each: cyclophosphamide, mycophenolate mofetil or its derivatives, belimumab, azathioprine, anifrolumab, methotrexate, rituximab, obinutuzumab, cyclosporin, tacrolimus or voclosporin.
- Serum ALT <5 times the normal value, serum bilirubin <3 times the normal value, Left ventricular ejection fraction >45% Life expectancy of more than 3 months Eastern Cooperative Oncology Group (ECOG) performance status ≤2. Psychological, sociological or geographical conditions precluding compliance A female of childbearing age must have a negative pregnancy test and is on two effective contraception methods A male must use two effective contraception methods
Who cannot join
- -
- Active cancer or receiving cancer treatment Evidence of severe lung, FVC <45% and/or DLCO (corrected for Hb) <30% predicted, heart (NYHA class III/IV, arrhythmia, AV block, uncontrolled hypertension), liver failure or severe neurologic disorders.
- Pre-existing irreversible kidney damage and creatinine clearance below 30 ml/min ( to review) Severe pancytopenia HIV positivity. Active Hepatitis B, C infection. Septicemia. Pregnant/nursing female. Receiving stem cell transplant within 12 weeks of enrolment, chemotherapy or radiotherapy within 8 weeks of enrolment Active CNS involvement.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Malaysia1 site, 1 recruiting
- Recruiting
University Kebangsaan Malaysia Medical Center
Bandar Tun Razak, Kuala Lumpur, 56000
S Fadilah Abdul Wahid, PhD, +60391456450, sfadilah@hctm.ukm.edu.my
About this study
Official title: Autologous CD19 Car T-Cell Therapy For Patients With Severe Refractory Systemic Lupus Erythematosus - A Pilot Study
This pilot clinical study aims to evaluate the effectiveness of Chimeric Antigen Receptor (CAR) T-cell therapy in treating severe, refractory systemic lupus erythematosus (SLE), an autoimmune disease driven by autoreactive B-cells. Current treatments for severe SLE, including glucocorticoids, cytotoxic, and immunosuppressive drugs, have significant limitations. These treatments do not adequately control the underlying autoimmune process and require long-term use, leading to chronic side effects and often failing to prevent permanent organ damage. Given the high prevalence and mortality rates associated with SLE in regions like Asia and Malaysia, there is a pressing need for more effective therapies.
Read the detailed description
This study seeks to investigate CAR-T cell therapy's potential for treating severe treatment-refractory systemic lupus erythematosus (SLE) in Malaysian patients.
STUDY OBJECTIVES
General Objective: To evaluate the safety and efficacy of autologous CD19 CAR T-cell infusion in patients with severe refractory SLE.
Specific Objectives:
To evaluate the safety and tolerability of treatment. To evaluate efficacy as measured by response rates and survival. To evaluate the quality of life based on the use of validated health questionnaire tools.
Hypothesis:
Intravenous autologous CD19 CAR T-cell therapy is safe, and efficacious in patients with severe refractory SLE.
EXPECTED OUTCOME OF THE STUDY:
CD19 CAR T-cell therapy is expected to result in:
Clinical remission as assessed by DORIS remission) with a good safety profile Serologic remission (defined as negative anti-dsDNA and normal complement C3 and C4 levels) at 3 months Sustained DORIS remission for at least 12 months Improvement in organ function Prolongation of duration of drug-free remission Improvement in quality of life Manageable adverse effects
STUDY POPULATION This pilot study will be conducted between 2025 and 2028 involving 5 patients diagnosed with severe, treatment-refractory SLE. Eligibility criteria were based on previously published studies and international guidelines for CAR treatment for patients with autoimmune disease (Boulougoura et al., 2023).
- Conditions
- Systemic Lupus Erythematous (SLE)
- Being tested
- CD19 CAR-T cells
- Keywords
- CAR T cells, SLE, treatment-refractory, severe SLE
- 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
Study participant will be given single infusion of autologous CD19 CAR-T cells following lymphodepletion chemotherapy
| Study group | Role | What they receive |
|---|---|---|
| autologous CD19 CAR T-cells | Experimental | Single Infusion of autologous CD19 CAR T- cells Receives: Biological: CD19 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Adverse events
Incidence of adverse event (AE), classified according to CTCAE version 5.0, and evaluation and classification of Cytokine Release Syndrome (CRS) and Immune Effector cell-associated Neurotoxicity Syndrome (ICANS)
Measured: Starting form day 0 up to 24 months after CAR-T cells infusion
SLEDAI Remission Rate
Remission is evaluated by fulfilment of SLEDAI remission criteria
Measured: Weeks 4, 8, 12, 16, 20, 24,28 after CAR-T cells infusion
Secondary outcomes (4)
Clinical Response Rate
Measured by BILAG, PGA, SLICC/ACR
Measured: Weeks 4, 8, 12, 16, 20, 24,28 after CAR-T cells infusion
Rate of B cell aplasia
Numbers of B cells in peripheral blood measured by flow cytometry
Measured: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 after CAR-T cells infusion
Rate of Immunological response
Changes in the levels of SLE-associated serum autoantibodies
Measured: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 after CAR-T cells infusion
Mean of Quality of life score
Quality of life Assessed by FACIT-F
Measured: Weeks 4, 8, 12, 16, 20, 24,28 after CAR-T cells infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2024
- Study start3 Jan 2025
- Primary completion2 Dec 2028 (estimated)
- Study completion2 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Lupus Erythematous (SLE)Studies 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
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National University of Malaysia
- Sponsor type
- University, hospital or other
- Collaborators
- Plutonet Sdn Bhd
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06710717
- Sponsor's protocol ID
- JEP-2024-754
- Status last verified
- May 2025
- First submitted
- 21 Nov 2024
- Last updated
- 29 May 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 29 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous CD19 Car T-Cell Therapy For Patients With Severe Refractory Systemic Lupus Erythematosus - A Pilot Study
- ClinicalTrials.gov ID
- NCT06710717
- Acronym / sponsor ID
- JEP-2024-754
- Conditions
- Systemic Lupus Erythematous (SLE)
- Keywords
- CAR T cells, SLE, treatment-refractory, severe SLE
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- National University of MalaysiaUniversity, hospital or other
- Collaborators
- Plutonet Sdn Bhd
- Study start
- 3 Jan 2025 (actual)
- Primary completion
- 2 Dec 2028 (estimated)
- Study completion
- 2 Jan 2029 (estimated)
- First posted
- 29 Nov 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 pilot clinical study aims to evaluate the effectiveness of Chimeric Antigen Receptor (CAR) T-cell therapy in treating severe, refractory systemic lupus erythematosus (SLE), an autoimmune disease driven by autoreactive B-cells. Current treatments for severe SLE, including glucocorticoids, cytotoxic, and immunosuppressive drugs, have significant limitations. These treatments do not adequately control the underlying autoimmune process and require long-term use, leading to chronic side effects and often failing to prevent permanent organ damage. Given the high prevalence and mortality rates associated with SLE in regions like Asia and Malaysia, there is a pressing need for more effective therapies.
Detailed description
This study seeks to investigate CAR-T cell therapy's potential for treating severe treatment-refractory systemic lupus erythematosus (SLE) in Malaysian patients.
STUDY OBJECTIVES
General Objective: To evaluate the safety and efficacy of autologous CD19 CAR T-cell infusion in patients with severe refractory SLE.
Specific Objectives:
To evaluate the safety and tolerability of treatment. To evaluate efficacy as measured by response rates and survival. To evaluate the quality of life based on the use of validated health questionnaire tools.
Hypothesis:
Intravenous autologous CD19 CAR T-cell therapy is safe, and efficacious in patients with severe refractory SLE.
EXPECTED OUTCOME OF THE STUDY:
CD19 CAR T-cell therapy is expected to result in:
Clinical remission as assessed by DORIS remission) with a good safety profile Serologic remission (defined as negative anti-dsDNA and normal complement C3 and C4 levels) at 3 months Sustained DORIS remission for at least 12 months Improvement in organ function Prolongation of duration of drug-free remission Improvement in quality of life Manageable adverse effects
STUDY POPULATION This pilot study will be conducted between 2025 and 2028 involving 5 patients diagnosed with severe, treatment-refractory SLE. Eligibility criteria were based on previously published studies and international guidelines for CAR treatment for patients with autoimmune disease (Boulougoura et al., 2023).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| autologous CD19 CAR T-cells | Experimental | Biological: CD19 CAR-T cellsSingle Infusion of autologous CD19 CAR T- cells |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- -
- Aged between ≥ 18 to ≤ 65 years Clinical Diagnosis of SLE according to the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria (Fanouriakis et al., 2019) Positive anti-nuclear antibody (ANA) (titer ≥1:80 ), anti-dsDNA (≥30 IU/mL on enzyme-linked immunosorbent assay [ELISA]), or anti-Smith at screening or by documented medical history Active disease (defined by not being in remission according to DORIS criteria or in a low disease activity state [LLDAS]) (Franklyn et al., 2016, van Vollenhoven et al., 2021) With at least one active organ system involvement Persistent active disease with insufficient response to glucocorticoids and at least 2 of the following treatments for at least 3 months each: cyclophosphamide, mycophenolate mofetil or its derivatives, belimumab, azathioprine, anifrolumab, methotrexate, rituximab, obinutuzumab, cyclosporin, tacrolimus or voclosporin.
- Serum ALT <5 times the normal value, serum bilirubin <3 times the normal value, Left ventricular ejection fraction >45% Life expectancy of more than 3 months Eastern Cooperative Oncology Group (ECOG) performance status ≤2. Psychological, sociological or geographical conditions precluding compliance A female of childbearing age must have a negative pregnancy test and is on two effective contraception methods A male must use two effective contraception methods
Key exclusion criteria (3 listed)
- -
- Active cancer or receiving cancer treatment Evidence of severe lung, FVC <45% and/or DLCO (corrected for Hb) <30% predicted, heart (NYHA class III/IV, arrhythmia, AV block, uncontrolled hypertension), liver failure or severe neurologic disorders.
- Pre-existing irreversible kidney damage and creatinine clearance below 30 ml/min ( to review) Severe pancytopenia HIV positivity. Active Hepatitis B, C infection. Septicemia. Pregnant/nursing female. Receiving stem cell transplant within 12 weeks of enrolment, chemotherapy or radiotherapy within 8 weeks of enrolment Active CNS involvement.
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 cellsStudy participant will be given single infusion of autologous CD19 CAR-T cells following lymphodepletion chemotherapy | Biological | autologous CD19 CAR T-cells |
Primary outcome
Rate of Adverse events
Time frame: Starting form day 0 up to 24 months after CAR-T cells infusion
Incidence of adverse event (AE), classified according to CTCAE version 5.0, and evaluation and classification of Cytokine Release Syndrome (CRS) and Immune Effector cell-associated Neurotoxicity Syndrome (ICANS)
SLEDAI Remission Rate
Time frame: Weeks 4, 8, 12, 16, 20, 24,28 after CAR-T cells infusion
Remission is evaluated by fulfilment of SLEDAI remission criteria
4 secondary outcomes
Clinical Response Rate
Time frame: Weeks 4, 8, 12, 16, 20, 24,28 after CAR-T cells infusion
Rate of B cell aplasia
Time frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 after CAR-T cells infusion
Rate of Immunological response
Time frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 after CAR-T cells infusion
Mean of Quality of life score
Time frame: Weeks 4, 8, 12, 16, 20, 24,28 after CAR-T cells infusion
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 29 May 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.
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 Erythematous (SLE), run by National University of Malaysia at 1 site in Malaysia.
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 3 inclusion and 3 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 29 May 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 Malaysia. 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 S Fadilah Abdul Wahid, PhD (+60391456450, sfadilah@hctm.ukm.edu.my), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Adverse events (time frame: Starting form day 0 up to 24 months after CAR-T cells infusion). The study lists 2 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 29 May 2025.