System Lupus ErythematosusEarly Phase 1NCT07301164
Clinical Study of BCT301 Cell Injection Therapy for Refractory Autoimmune Diseases
A Early Phase 1 study testing BCT301 in people with System Lupus Erythematosus and Systemic Sclerosis (SSc), run by Peking University Third Hospital at 1 site in China.
Trial summary
This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- System Lupus Erythematosus, Systemic Sclerosis (SSc), Inflammatory Myositis
- What is being tested?
- BCT301
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 11 Dec 2025to 31 Dec 2028
- Phase
- Early 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- General Inclusion Criteria
- Voluntarily sign the informed consent form.
- Male or female, aged 18-80 years (inclusive), with a body weight ≥40 kg.
- Female participants of childbearing potential and male participants with female partners of childbearing potential must use medically approved contraceptive methods or practice abstinence during the treatment period and for at least 6 months after the end of the treatment. Female participants of childbearing potential must have a negative serum human chorionic gonadotropin (HCG) test within 7 days prior to enrollment and must not be breastfeeding.
- Participants currently receiving one or more of the following treatments at stable doses: glucocorticoids, antimalarials, immunosuppressants:
- If the participant is receiving glucocorticoid therapy, the following conditions must be met: the maximum dose at screening and during the screening period is 30 mg/day of prednisone (or equivalent). The dose must have been stable for ≥7 days prior to screening, and adjustments during the screening period must not exceed 5 mg/day of prednisone (or equivalent);
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- If the participant is receiving antimalarials and/or conventional immunosuppressants: the treatment must have been initiated ≥12 weeks prior to screening. The dose must have been stable for ≥8 weeks prior to screening and remain stable during the screening period;
- If biological agents (belimumab, telitacicept, rituximab, etc.) were used prior to the screening period, a washout period of at least 5 half-lives must be completed before screening.
- Peripheral blood B cells must show positive CD19 expression as detected by flow cytometry.
- Disease-Specific Inclusion Criteria
- 1. Systemic Lupus Erythematosus (SLE)
- Meet the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria for SLE.
- Have moderate to severe disease activity at screening, with a Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2000) score >6.
- Have inadequate response to conventional therapy or experience disease relapse after remission. Conventional therapy includes glucocorticoids (at full or pulse doses), two or more immunosuppressants, or biologic agents for at least 6 months, including cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, azathioprine, leflunomide, methotrexate, belimumab, telitacicept, and rituximab.
- Have positive serological autoantibody tests: positive antinuclear antibodies (ANAs) and/or anti-ds-DNA antibodies and/or anti-Sm antibodies.
- 2. Systemic Sclerosis (SSc)
- Meet the 2013 EULAR/ACR classification criteria for SSc.
- Fulfill either (a) or (b) below:
- Inadequate response to conventional therapy or disease relapse after remission. Conventional therapy includes glucocorticoids (at full or pulse doses), two or more immunosuppressants, or biologic agents for at least 6 months, including cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, azathioprine, leflunomide, methotrexate, belimumab, telitacicept, and rituximab.
- Disease progression: skin progression with a modified Rodnan Skin Score (mRSS) ≥10; and/or interstitial lung disease evidenced by ground-glass opacities on high-resolution computed tomography (HRCT); a decline in forced vital capacity (FVC) ≥10%, or a decline in FVC ≥5% accompanied by a decline in diffusing capacity for carbon monoxide (DLCO) ≥15%.
- Have positive SSc-related autoantibodies. 3. Antiphospholipid Syndrome (APS)
- 1) Meet the 2006 Sydney criteria for primary antiphospholipid syndrome. 2) Have medium to high titers of antiphospholipid antibodies (lupus anticoagulant [LA], anti-β2-glycoprotein 1 [β2GP1] IgG/IgM, or anti-cardiolipin [aCL] IgG/IgM); 3) Fulfill either (a) or (b) below:
- Receiving standard treatment with warfarin or alternative vitamin K antagonists (maintaining target international normalized ratio [INR]), or standard therapeutic doses of low molecular weight heparin (LMWH), and/or glucocorticoids and immunosuppressants/biologics (e.g., cyclophosphamide, cyclosporine, tacrolimus, rituximab, etc.).
- Meet all four criteria for catastrophic APS:
- Involvement of three or more organs, systems, and/or tissues; ii) Development of manifestations within one week; iii) Histopathological confirmation of small vessel occlusion in at least one organ or tissue; iv) Positive antiphospholipid antibodies (aPL).
- 4. Inflammatory Myopathy (IM)
- Meet the 2017 EULAR/ACR classification criteria for inflammatory myopathy (including dermatomyositis, polymyositis, anti-synthetase syndrome, and immune-mediated necrotizing myopathy).
- Have positive myositis-specific autoantibodies.
- For participants with muscle involvement: a Manual Muscle Test-8 (MMT-8) score <142, and at least two of the following five core abnormalities: Physician Global Assessment ≥2, Patient Global Assessment ≥2, or extramuscular disease activity score ≥2; Health Assessment Questionnaire (HAQ) total score ≥0.25; muscle enzyme levels ≥1.5 times the upper limit of normal; or MMT-8 ≥142 but with active interstitial lung disease (ground-glass opacities on HRCT).
- 5. Sjögren's Syndrome (SS)
- Meet the 2016 ACR/EULAR classification criteria for Sjögren's syndrome.
- Have a EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) score ≥5.
- Have positive anti-SSA/Ro antibodies.
- Have inadequate response to conventional therapy or disease relapse after remission. Conventional therapy includes glucocorticoids (at full or pulse doses), two or more immunosuppressants, or biologic agents for at least 6 months, including cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, azathioprine, leflunomide, methotrexate, belimumab, telitacicept, and rituximab.
- 6. Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV)
- Meet the 2022 ACR/EULAR classification criteria for ANCA-associated vasculitis (AAV), including microscopic polyangiitis, granulomatosis with polyangiitis, and eosinophilic granulomatosis with polyangiitis.
- Have a history of or currently positive ANCA.
- Have a Birmingham Vasculitis Activity Score (BVAS) ≥15 (total score 63).
- Have inadequate response to conventional therapy or disease relapse after remission. Conventional therapy includes glucocorticoids (at full or pulse doses), two or more immunosuppressants, or biologic agents for at least 6 months, including cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, azathioprine, leflunomide, methotrexate, belimumab, telitacicept, and rituximab.
Who cannot join
- Study participants who meet any of the following criteria will be excluded from the study:
- Any medical condition that, in the opinion of the investigator, would contraindicate participation in the study, such as a life-threatening illness.
- Decreased organ function reserve not attributable to the primary disease:
- Neutrophil count <1×10⁹/L; lymphocyte count <0.3×10⁹/L; hemoglobin <70 g/L; platelet count <50×10⁹/L; b) Alanine aminotransferase (ALT) >3 × upper limit of normal (ULN); aspartate aminotransferase (AST) >3 × ULN; total bilirubin >2 × ULN; c) Creatinine clearance <40 mL/min; estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m²; or serum creatinine >2.5 mg/dL; d) Left ventricular ejection fraction (LVEF) <45% as measured by echocardiography; e) Oxygen saturation <92% on room air.
- History of alcohol or substance abuse within the past 24 weeks.
- History of malignancy other than B-cell lymphoma.
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- Presence of infections including human immunodeficiency virus (HIV), hypogammaglobulinemia, T-cell deficiency, syphilis, chronic hepatitis B or C, or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
- Known active tuberculosis (TB) infection or active bacterial infection.
- History of myocardial infarction, coronary angioplasty or stenting, unstable angina, clinically significant arrhythmia, or other clinically significant cardiac disease within 6 months prior to screening.
- Symptomatic deep vein thrombosis or pulmonary embolism within 6 months prior to screening, except in cases of antiphospholipid syndrome (APS).
- History of severe allergic reaction to any component of cellular therapy or other immunotherapeutic agents.
- Prior organ transplant requiring ongoing immunosuppressive therapy.
- Concurrent participation in another clinical trial that may interfere with disease assessment or study treatment.
- Prior treatment with CD19- and/or BCMA-targeted therapy or any CAR-T cell product; except in cases where prior therapy is deemed to have clearly failed (e.g., no response, short duration of response, or disease progression) as assessed by the investigator, the current disease state warrants the study treatment, and there is no clear evidence that toxicity from prior therapy would compromise the safety of the current study.
- Severe psychiatric disorder or significant cognitive impairment.
- Pregnancy, lactation, or planned pregnancy.
- Any other condition that, in the judgment of the investigator, would make the participant unsuitable for enrollment in this clinical trial.
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
Peking University Third Hospital
Beijing
Xiaoying Zhang, 86+13810001444, zhang_xiaoying@pku.edu.cn
Xiaoying Zhang
About this study
Official title: A Study of BCT301 (Anti-CD19 Chemically Induced Pluripotent Stem Cell (CiPSC)-Derived CAR-iT Cells) Therapy for Refractory Autoimmune Diseases
This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.
Read the detailed description
Autoimmune diseases are a class of disorders caused by abnormal immune responses against the body's own tissues or organs. Although significant therapeutic advances have been made, such as the use of biologics (e.g., rituximab), most available drugs can only alleviate symptoms or slow disease progression, rather than achieve a complete cure.BCT301 cell injection is a CD19-targeted CAR-iT (chemically induced T-cell) therapy derived from human chemically induced pluripotent stem cells (CiPSCs) through chemical reprogramming technology. Starting from healthy donor cells, human CiPSCs are generated via small molecule-based reprogramming, followed by induced hematopoietic differentiation, iT-cell differentiation and expansion, and finally CAR lentiviral transduction to produce the off-the-shelf, CD19-targeted CAR-iT cell product (BCT301 cell injection). This approach holds promise as a potentially effective treatment strategy.
- Conditions
- System Lupus Erythematosus, Systemic Sclerosis (SSc), Inflammatory Myositis, Antiphospholipid Syndrome, ANCA Associated Vasculitis, Sjogren Syndrome
- Being tested
- BCT301
- Medical subject headings
- Lupus Erythematosus, Systemic, Scleroderma, Systemic, Antiphospholipid Syndrome, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Sjogren's Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCT301Biological
BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells.Participants will receive a single infusion of BCT301 cell injection at escalating dose levels.Prior to infusion, patients undergo a lymphodepleting conditioning regimen with fludarabine and cyclophosphamide given intravenously for three consecutive days.
| Study group | Role | What they receive |
|---|---|---|
| BCT301 | Experimental | Not described Receives: Biological: BCT301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-limiting toxicities of BCT301
Adverse events occurring within 28 days post-infusion that were possibly, probably, or definitely related to BCT301 cell injection (per NCI-CTCAE v5.0).
Measured: 28 days
Secondary outcomes (25)
Efficacy of refractory systemic lupus erythematosus（SLE）- The change of SLEDAI-2K
The primary efficacy endpoint will be the change from baseline in the SLEDAI-2K score. The SLEDAI-2K is a validated index for assessing SLE disease activity, with scores ranging from 0 to 105. A higher score indicates worse disease activity.
Measured: 90, 180, 360 days
Efficacy of refractory SLE-The change of PGA
The Physician Global Assessment (PGA) will be used to evaluate overall SLE disease activity. The PGA is a clinician's determination of disease activity based on history and physical exam, independent of laboratory results. It is scored on a scale from 0 to 3, where a higher score indicates worse disease activity.
Measured: 90, 180, 360 days
Efficacy of refractory SLE-The change of SF36
The SF-36 health survey will be employed as a patient-reported outcome to assess health-related quality of life. This 36-item questionnaire generates scores from 0 to 100 across eight health domains and two summary components (Physical and Mental). A lower score indicates a worse health state
Measured: 90, 180, 360 days
Efficacy of refractory SLE-The change of antibodies
Changes in autoantibody titers (e.g., anti-dsDNA) will be assessed as a serological outcome. A higher autoantibody titer reflects more severe underlying immunologic activity
Measured: 90, 180, 360 days
Efficacy of refractory systemic sclerosis (SSc)-The change of mRSS
The modified Rodnan Skin Score (mRSS) will be the primary endpoint to assess skin disease activity. The mRSS evaluates skin thickness in 17 body areas, with each scored from 0 (normal) to 3 (severe thickening). The total score ranges from 0 to 51, where a higher score indicates worse skin involvement and a poorer prognosis.
Measured: 90, 180, 360 days
Efficacy of refractory SSc-The change of CRISS
The Combined Response Index for Systemic Sclerosis (CRISS) will be used as the primary composite endpoint. This index calculates a probability of improvement (score from 0.0 to 1.0) by integrating changes in skin thickness (mRSS), physician and patient global assessments, functional ability (HAQ-DI), and the absence of new organ damage. A higher score indicates a better treatment response, while a score of 0 indicates treatment failure.
Measured: 90, 180, 360 days
Efficacy of refractory SSc-The change of antibodies
Changes in autoantibody titers (e.g., anti-SCL70) will be assessed as a serological outcome. A higher autoantibody titer reflects more severe underlying immunologic activity.
Measured: 90, 180, 360 days
Efficacy of refractory Antiphospholipid Syndrome (APS)-new onset thrombotic event
The primary efficacy endpoint is the time to first new objectively confirmed arterial or venous thrombotic event. The occurrence of a new thrombotic event is considered a treatment failure, as it directly demonstrates the failure of the therapy.
Measured: 90, 180, 360 days
Efficacy of refractory APS-The change of antibodies
Reduction in titers of antiphospholipid antibodies (aCL, anti-β2GPI, and LA) will be assessed as a key biomarker of response. A decrease in these antibody levels is interpreted as evidence of a positive immunologic treatment effect.
Measured: 90, 180, 360 days
Efficacy of refractory APS-The change of platelet
In patients with baseline thrombocytopenia, an increase in platelet count is considered a positive indicator of treatment efficacy, signifying control of the underlying autoimmune and consumptive processes in APS.
Measured: 90, 180, 360 days
Efficacy of refractory APS- The change of fibrinogen
In patients with baseline hypofibrinogenemia, an increase in fibrinogen level is considered a positive indicator of treatment efficacy
Measured: 90、180、360 days
Efficacy of refractory inflammatory myositis (IM)-The change of TIS
Total Improvement ScoreThe Total Improvement Score (TIS) will be the primary endpoint to define treatment response. The TIS is a composite score (range 0-15) quantifying improvement from baseline across six core domains: muscle strength, physical function, physician and patient global assessments, muscle enzymes, and extramuscular activity. A TIS of ≥ 20 is defined as a clinically meaningful treatment response, with a higher score indicating a greater magnitude of improvement.
Measured: 90, 180, 360 days
Efficacy of refractory IM-The change of CK
Serum Creatine Kinase (CK) level will be assessed as a key biomarker of muscle inflammation. A reduction in CK from baseline, is a defined indicator of positive biochemical treatment response.
Measured: 90, 180, 360 days
Efficacy of refractory IM-The change of antibodies
Reduction in titers of antibodies (e.g. anti-MDA5) will be assessed as a key biomarker of response. A decrease in these antibody levels is interpreted as evidence of a positive immunologic treatment effect.
Measured: 90, 180, 360 days
Efficacy of refractory Sjögren's Disease（SjD）-The change of ESSDAI
The EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) will be the primary endpoint to assess systemic disease activity. The ESSDAI scores 12 organ domains, with a total range from 0 (no activity) to 123 (maximal activity). A reduction in the ESSDAI score of ≥3 points from baseline defines a clinically meaningful treatment response, indicating effective suppression of systemic disease.
Measured: 90, 180, 360 days
Efficacy of refractory SjD- Achieving STAR response or not
The Sjögren's Tool for Assessing Response (STAR) will be used as a key patient-reported outcome. The STAR quantifies symptom burden on a scale from 0 (none) to 10 (worst imaginable). A decrease from baseline of ≥1 point in the STAR score defines a clinically meaningful treatment response, indicating a significant reduction in the patient's experience of core symptoms like dryness, fatigue, and pain.
Measured: 90, 180, 360 days
Efficacy of refractory SjD-The change of antibodies
Changes in autoantibody titers (e.g., anti-SSA) will be assessed as a serological outcome. A reduction in titer is considered a favorable immunologic response to therapy.
Measured: 90, 180, 360 days
Efficacy of refractory SjD-The change of immune globulin
Changes in immune globulin will be assessed as a serological outcome. A reduction in titer is considered a favorable immunologic response to therapy.
Measured: 90, 180, 360 days
Efficacy of refractory ANCA associated vasculitis (AAV)-The change of BVAS
The Birmingham Vasculitis Activity Score (BVAS) will be the primary endpoint to assess disease activity. The BVAS quantifies new or worsening vasculitis symptoms across nine organ systems. The total score ranges from 0 (no activity) to over 50, where a higher score indicates worse disease. A reduction in BVAS, particularly to 0, defines treatment response.
Measured: 90, 180, 360 days
Efficacy of refractory AAV-The change of antibody
Serial quantitative ANCA titers (anti-PR3 and anti-MPO) will be assessed as a serological biomarker of response. A significant decrease in ANCA titer is considered a favorable indicator of treatment efficacy.
Measured: 90, 180, 360 days
pharmacokinetic (PK)
time to peak (Tmax)
Measured: 0-28 days
pharmacokinetic (PK)
peak concentration (Cmax)
Measured: 0-28 days
pharmacokinetic (PK)
area under the concentration-time curve
Measured: 0-28 days
pharmacokinetic (PK)
area under the concentration-time curve
Measured: 0-90 days
pharmacodynamic (PD)
The count of B cells in peripheral blood post-infusion.
Measured: 0-28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2025
- Study start11 Dec 2025
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for System Lupus ErythematosusStudies of the same condition, open and under way.
- More trials run by Peking University Third HospitalThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University Third Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07301164
- Sponsor's protocol ID
- BCT301
- Status last verified
- Dec 2025
- First submitted
- 17 Nov 2025
- Last updated
- 23 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan to share IPD.
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of BCT301 (Anti-CD19 Chemically Induced Pluripotent Stem Cell (CiPSC)-Derived CAR-iT Cells) Therapy for Refractory Autoimmune Diseases
- ClinicalTrials.gov ID
- NCT07301164
- Acronym / sponsor ID
- BCT301
- Conditions
- System Lupus Erythematosus, Systemic Sclerosis (SSc), Inflammatory Myositis, Antiphospholipid Syndrome, ANCA Associated Vasculitis, Sjogren Syndrome
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Peking University Third HospitalUniversity, hospital or other
- Study start
- 11 Dec 2025 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 24 Dec 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 study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.
Detailed description
Autoimmune diseases are a class of disorders caused by abnormal immune responses against the body's own tissues or organs. Although significant therapeutic advances have been made, such as the use of biologics (e.g., rituximab), most available drugs can only alleviate symptoms or slow disease progression, rather than achieve a complete cure.BCT301 cell injection is a CD19-targeted CAR-iT (chemically induced T-cell) therapy derived from human chemically induced pluripotent stem cells (CiPSCs) through chemical reprogramming technology. Starting from healthy donor cells, human CiPSCs are generated via small molecule-based reprogramming, followed by induced hematopoietic differentiation, iT-cell differentiation and expansion, and finally CAR lentiviral transduction to produce the off-the-shelf, CD19-targeted CAR-iT cell product (BCT301 cell injection). This approach holds promise as a potentially effective treatment strategy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BCT301 | Experimental | Biological: BCT301 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (39 listed)
- General Inclusion Criteria
- Voluntarily sign the informed consent form.
- Male or female, aged 18-80 years (inclusive), with a body weight ≥40 kg.
- Female participants of childbearing potential and male participants with female partners of childbearing potential must use medically approved contraceptive methods or practice abstinence during the treatment period and for at least 6 months after the end of the treatment. Female participants of childbearing potential must have a negative serum human chorionic gonadotropin (HCG) test within 7 days prior to enrollment and must not be breastfeeding.
- Participants currently receiving one or more of the following treatments at stable doses: glucocorticoids, antimalarials, immunosuppressants:
Key exclusion criteria (17 listed)
- Study participants who meet any of the following criteria will be excluded from the study:
- Any medical condition that, in the opinion of the investigator, would contraindicate participation in the study, such as a life-threatening illness.
- Decreased organ function reserve not attributable to the primary disease:
- History of alcohol or substance abuse within the past 24 weeks.
- History of malignancy other than B-cell lymphoma.
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 |
|---|---|---|
| BCT301BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells.Participants will receive a single infusion of BCT301 cell injection at escalating dose levels.Prior to infusion, patients undergo a lymphodepleting conditioning regimen with fludarabine and cyclophosphamide given intravenously for three consecutive days. | Biological | BCT301 |
Primary outcome
Dose-limiting toxicities of BCT301
Time frame: 28 days
Adverse events occurring within 28 days post-infusion that were possibly, probably, or definitely related to BCT301 cell injection (per NCI-CTCAE v5.0).
25 secondary outcomes
Efficacy of refractory systemic lupus erythematosus（SLE）- The change of SLEDAI-2K
Time frame: 90, 180, 360 days
Efficacy of refractory SLE-The change of PGA
Time frame: 90, 180, 360 days
Efficacy of refractory SLE-The change of SF36
Time frame: 90, 180, 360 days
Efficacy of refractory SLE-The change of antibodies
Time frame: 90, 180, 360 days
Efficacy of refractory systemic sclerosis (SSc)-The change of mRSS
Time frame: 90, 180, 360 days
Efficacy of refractory SSc-The change of CRISS
Time frame: 90, 180, 360 days
Efficacy of refractory SSc-The change of antibodies
Time frame: 90, 180, 360 days
Efficacy of refractory Antiphospholipid Syndrome (APS)-new onset thrombotic event
Time frame: 90, 180, 360 days
Efficacy of refractory APS-The change of antibodies
Time frame: 90, 180, 360 days
Efficacy of refractory APS-The change of platelet
Time frame: 90, 180, 360 days
Efficacy of refractory APS- The change of fibrinogen
Time frame: 90、180、360 days
Efficacy of refractory inflammatory myositis (IM)-The change of TIS
Time frame: 90, 180, 360 days
Efficacy of refractory IM-The change of CK
Time frame: 90, 180, 360 days
Efficacy of refractory IM-The change of antibodies
Time frame: 90, 180, 360 days
Efficacy of refractory Sjögren's Disease（SjD）-The change of ESSDAI
Time frame: 90, 180, 360 days
Efficacy of refractory SjD- Achieving STAR response or not
Time frame: 90, 180, 360 days
Efficacy of refractory SjD-The change of antibodies
Time frame: 90, 180, 360 days
Efficacy of refractory SjD-The change of immune globulin
Time frame: 90, 180, 360 days
Efficacy of refractory ANCA associated vasculitis (AAV)-The change of BVAS
Time frame: 90, 180, 360 days
Efficacy of refractory AAV-The change of antibody
Time frame: 90, 180, 360 days
pharmacokinetic (PK)
Time frame: 0-28 days
pharmacokinetic (PK)
Time frame: 0-28 days
pharmacokinetic (PK)
Time frame: 0-28 days
pharmacokinetic (PK)
Time frame: 0-90 days
pharmacodynamic (PD)
Time frame: 0-28 days
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 23 Sep 2026.
- Individual participant dataSharing planned: No. There is no plan to share IPD.
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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 Early Phase 1 study testing BCT301 in people with System Lupus Erythematosus and Systemic Sclerosis (SSc), run by Peking University Third Hospital 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 39 inclusion and 17 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 23 Sep 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 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-limiting toxicities of BCT301 (time frame: 28 days). 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 23 Sep 2026.