CD19-BCMA Dual-Target CAR-T Cell Therapy for Refractory Autoimmune Disease Clinical Trial
A Phase 1/2 single-arm study at Beijing GoBroad Hospital testing CD19-BCMA dual-target CAR-T cell therapy in patients with refractory lupus nephritis, systemic sclerosis, or primary Sjögren syndrome with pulmonary artery hypertension. CancerFax helps international patients organize records, coordinate translation, and request preliminary review by the trial team.
About This Clinical Trial
Refractory systemic lupus erythematosus with proliferative lupus nephritis (SLE-LN), systemic sclerosis (SSc), and primary Sjögren syndrome combined with pulmonary artery hypertension (pSS-PAH) are severe, life-altering autoimmune diseases. Many patients continue to progress despite long courses of glucocorticoids, cyclophosphamide, mycophenolate mofetil, tacrolimus, and other standard immunosuppressive therapies. For these patients, organ damage accumulates and treatment options become very limited.
This trial (NCT06947460) is a single-center, open-label, non-randomized, single-arm Phase 1/2 study at Beijing GoBroad Hospital. It evaluates an autologous dual-target CAR-T cell therapy directed against both CD19 (a pan B-cell marker) and BCMA (a marker on plasmablasts and long-lived plasma cells). The study plans to enroll up to 45 patients aged 10 to 65 across four refractory disease groups: lupus nephritis, systemic sclerosis, Sjögren-associated PAH, and a broader autoimmune disease (AID) cohort defined by serologic and clinical criteria.
The scientific rationale for dual-target CD19/BCMA CAR-T in autoimmune disease is straightforward. Single-target anti-CD19 CAR-T has shown deep B-cell depletion and durable drug-free remission in early autoimmune cohorts, but autoantibodies are produced largely by BCMA-expressing plasma cells that are not eliminated by CD19 alone. Combining CD19 and BCMA targeting is intended to deplete both the B-cell precursor pool and the antibody-producing plasma cell compartment, potentially achieving deeper immunologic reset than either target alone.
The primary objective is safety, measured as the type and incidence of dose-limiting toxicity within 28 days of infusion and the overall adverse event profile within 30 days. Secondary objectives include overall remission rate at 90 and 120 days and serious adverse events tracked out to 2 years. As of this writing the trial is actively recruiting; no published efficacy results are available yet. ⚠ VERIFY: ~45 enrollment target and active-recruiting status confirmed from registry on the date this page was prepared.
Is dual-target CD19-BCMA CAR-T cell therapy safe and tolerable in patients with severe refractory autoimmune disease, and does it produce meaningful clinical and serologic remission?
Trial at a Glance
A quick summary of this trial. This is not a confirmation that any individual patient is eligible.
Final eligibility is determined only by the trial investigators after reviewing complete medical records.
Treatment Being Studied
The investigational therapy is an autologous CAR-T cell product engineered to recognize two targets at once: CD19 (found on most B cells) and BCMA (found on plasmablasts and antibody-producing plasma cells). The patient's own T cells are collected, modified in a manufacturing facility to express both receptors, expanded, and then infused back after a short course of preparatory chemotherapy.
The goal is a deeper immune reset than what is achievable with conventional immunosuppression or single-target CAR-T. By depleting both the B-cell precursor compartment and the plasma cells that produce pathogenic autoantibodies, the therapy aims to interrupt the disease at multiple points in the autoimmune cascade.
How the therapy works (in simple terms)
T cells are immune cells that recognize and kill specific targets. In this trial, a patient's own T cells are reprogrammed in the lab to carry two synthetic receptors — one that recognizes CD19 and another that recognizes BCMA. When these cells are returned to the body, they hunt down and destroy CD19-positive B cells and BCMA-positive plasma cells. Because these cells are responsible for producing the autoantibodies driving disease, removing them may allow the immune system to reset. New B cells eventually regenerate from bone marrow precursors, ideally without the autoreactive programming that drove the original disease.
T cells are collected from the patient's blood through a leukapheresis procedure, typically over a few hours. A minimum lymphocyte count of 0.5×10⁹/L is required.
The collected T cells are sent to a GMP facility where they are genetically modified to express both the CD19 and BCMA CAR receptors, then expanded. Manufacturing typically takes 2–4 weeks.
A short course of conditioning chemotherapy (typically fludarabine and cyclophosphamide) is given to make room for the CAR-T cells to expand and engraft.
The manufactured CAR-T cells are infused back into the patient as a single-time intravenous administration. Vital signs are monitored closely during and after infusion.
Patients remain hospitalized for close monitoring of cytokine release syndrome, neurologic effects, cytopenias, infection, and disease-specific response. The 28-day post-infusion window is the primary safety observation period.
After discharge, patients return for scheduled assessments at 90 and 120 days for response evaluation, and continue follow-up for serious adverse events out to 2 years.
CD19-BCMA dual-target CAR-T cell therapy is not an approved treatment for autoimmune disease anywhere in the world. It is being studied for safety and early efficacy. Results from this trial are not yet available.
Who This Trial May Be For
This trial is being studied in patients with severe, treatment-refractory autoimmune disease who have already tried standard immunosuppressive therapy without adequate response. The four disease groups have distinct eligibility profiles, and final suitability is determined by the trial investigators.
Lupus nephritis patients who have failed at least one standard immunosuppressive regimen over 3 to 6 months. Systemic sclerosis, Sjögren-PAH, and broader AID patients with documented refractory disease or intolerance to long-term immunosuppression.
Diagnosis confirmed using accepted international classification criteria — 2019 ACR/EULAR for SLE, ACR/EULAR for SSc, and 2002 AECG or 2016 ACR/EULAR for primary Sjögren syndrome. For lupus nephritis, renal biopsy showing active Class III or IV disease (excluding chronic-only lesions).
ECOG 0–2 (for LN cohort), WHO functional class I–II (for pSS-PAH cohort), expected survival ≥90 days, lymphocyte count >0.5×10⁹/L, eGFR ≥30 ml/min/1.73m², and acceptable liver function.
Lupus nephritis and the broader AID cohort accept patients aged 10–65, including pediatric and adolescent patients with parental/guardian informed consent. SSc and pSS-PAH cohorts are 18–65 only.
No active uncontrolled infection, no positive hepatitis B/C/HIV/syphilis screening, no symptomatic heart failure or severe arrhythmia, no active intracranial pathology, no other malignancy, and no prior CAR-T therapy other than CD19-CAR-T.
Final eligibility is determined only by the Beijing GoBroad Hospital trial team after reviewing complete records. International patients must be willing to travel to Beijing for screening, treatment, and the inpatient observation period.
Eligibility Criteria
These criteria are summarized from the public trial protocol. Meeting some of them does not mean enrollment is guaranteed. The trial has four cohorts with different specific requirements, and final eligibility is determined only by the trial investigators after complete record review.
check_circleInclusion Criteria — May Be Eligible
- ✓Refractory Lupus Nephritis (LN): Failure to achieve induction remission after 3 to 6 months of at least one immunosuppressive agent (glucocorticoids, cyclophosphamide, tacrolimus, mycophenolate mofetil, or cyclosporine), with no reduction or worsening of proteinuria or persistent positive autoantibodies
- ✓LN diagnosis per 2019 ACR criteria, confirmed by renal biopsy per 2018 ISN/RPS as active proliferative Class III or IV (excluding pure chronic lesions), or combined Class III/IV with Class V
- ✓LN cohort: Age 10–65, meets 2019 ACR/EULAR SLE classification, ANA ≥1:80 and/or anti-dsDNA positive (≥30 IU/mL by ELISA)
- ✓Systemic Sclerosis (SSc): Age 18–65, ACR/EULAR SSc classification score ≥9, skin thickening of the fingers extending proximal to the metacarpophalangeal joints
- ✓Primary Sjögren Syndrome with PAH (pSS-PAH): Age 18–65, meets 2002 AECG or 2016 ACR/EULAR pSS criteria, right-heart-catheterization-confirmed PAH (mPAP ≥20 mmHg, PAWP ≤15 mmHg, PVR >2 WU), WHO functional class I–II, 6MWD >440 m, BNP <50 ng/L or NT-proBNP <300 ng/L
- ✓Broader AID cohort: Age 10–65, refractory autoimmune disease with serologic confirmation, intolerance to or organ damage from long-term immunosuppression
- ✓Across all cohorts: B-cell CD19+ expression confirmed, immunosuppressant washout ≥1 week, lymphocyte count >0.5×10⁹/L, no cell collection contraindications, no serious allergic constitution
- ✓Adequate organ function: AST/ALT ≤3× ULN, T-Bil ≤2× ULN (≤3× for Gilbert syndrome), SpO₂ >95% on room air, ECOG 0–2 (LN), expected survival ≥90 days
- ✓Signed informed consent by patient and/or legal guardian
cancelExclusion Criteria — May Not Be Eligible
- ×Intracranial hypertension, disorder of consciousness, or active intracranial lesions on MRI
- ×Symptomatic heart failure or severe arrhythmia
- ×Severe respiratory failure
- ×Other malignancies
- ×Diffuse intravascular coagulation
- ×Septicemia or other uncontrollable infections, including active tuberculosis
- ×Uncontrolled diabetes or other endocrine diseases
- ×Severe mental disorders
- ×Prior organ transplantation (bone marrow transplant is not exclusionary)
- ×Pregnancy, breastfeeding, or refusal to use effective contraception
- ×Positive screening for hepatitis B, hepatitis C, HIV, or syphilis
- ×Inability to undergo PBMC collection and no cryopreserved PBMCs available
- ×eGFR (CKD-EPI) <30 ml/min/1.73m²
- ×Prior CAR-T therapy other than CD19-CAR-T
- ×For SSc cohort: overlapping rheumatoid arthritis, SLE, or inflammatory myopathy; SSc-mimicking conditions; severe active CNS lupus within 60 days of baseline; dialysis; recent major surgery within 4 weeks
- ×For pSS-PAH cohort: PH from other causes (portal hypertension, congenital heart disease, drugs/toxicants, chronic hypoxic lung disease with FEV1 <55% or TLC <60%, chronic thromboembolic PH)
Criteria here are illustrative. The trial protocol has its own detailed list. CancerFax can help organize records for review, but only the trial center can confirm participation.
Medical Records and Tests Needed for Review
Complete, well-organized records are essential. The trial team at Beijing GoBroad Hospital needs to verify diagnosis, refractory status, organ function, and screening results before any case can move forward. CancerFax helps families organize, translate, and submit these documents for preliminary review.
How the Trial Process May Work
CAR-T cell therapy trials involve significantly more steps than most clinical trials. From first inquiry to final follow-up, the process typically spans 6 to 12 months of active engagement, with safety follow-up extending to 2 years.
Patient or family contacts CancerFax. We collect medical records, organize a structured medical summary, and identify whether this trial or other advanced cell therapy options may be relevant.
Translated and organized records are submitted to the Beijing GoBroad Hospital trial team for preliminary review against the inclusion and exclusion criteria. This step does not guarantee enrollment.
If preliminary review is favorable, the patient travels to Beijing for in-person evaluation: physical examination, repeat imaging, blood work, biomarker confirmation, and any disease-specific tests required by the protocol.
The full trial protocol — risks, benefits, alternatives, study procedures, and rights — is reviewed with the patient (and guardian, for minors) before any study-related procedure begins.
T-cell collection, ~2–4 weeks of CAR-T manufacturing, lymphodepleting chemotherapy, and the single CAR-T infusion. The patient remains in or near Beijing for this entire window.
Inpatient monitoring through the 28-day primary safety window, then outpatient follow-up at 90 and 120 days for response assessment, with serious adverse event tracking continuing for 2 years.
Potential Benefits
For patients with severe refractory autoimmune disease, this trial may offer access to a therapy that is not commercially available anywhere in the world. The potential benefits are meaningful, but they are not guaranteed and must be weighed against real risks.
CD19-BCMA dual-target CAR-T is not yet approved for autoimmune disease in any country. This trial is one of the few structured pathways to access it.
Treatment at Beijing GoBroad Hospital involves rheumatology, nephrology, pulmonology, immunology, and cell therapy specialists working together on each case.
Trial protocols mandate frequent visits, detailed lab work, imaging, and structured response assessment that is more intensive than typical outpatient follow-up.
Early data from anti-CD19 CAR-T in autoimmune cohorts has shown durable remission off immunosuppression in some patients. The dual-target approach is designed to deepen and extend that response. Outcomes for this specific trial are not yet available.
Each enrolled patient contributes to the scientific understanding of CAR-T cell therapy in autoimmune disease, helping shape how this class of therapy is used for future patients.
A trial provides a clearly defined schedule, defined response criteria, defined safety monitoring, and a clear team — which can be especially valuable for refractory patients who have cycled through many treatments.
This is a Phase 1/2 trial. Its primary objective is safety, not proven efficacy. A trial may or may not directly benefit the individual patient. Realistic expectations are essential.
Risks and Side Effects
Every CAR-T cell therapy trial carries risks specific to this therapy class. Some are common and manageable with experienced cell therapy teams. Others are serious and require intensive in-hospital monitoring.
Fever, low blood pressure, hypoxia, and systemic inflammation caused by CAR-T cell activation. Most cases are manageable; severe cases may require ICU care, tocilizumab, or steroids.
Confusion, tremor, headache, language disturbance, or seizures can occur in the days to weeks after infusion. Frequency and severity vary by product and patient.
Prolonged neutropenia, thrombocytopenia, and anemia are common after lymphodepleting chemotherapy and CAR-T infusion, requiring transfusion and growth-factor support in some patients.
Because the therapy depletes both CD19+ B cells and BCMA+ plasma cells, antibody production drops. This raises infection risk and may require IVIG replacement and vaccination strategy review.
Fatigue, gastrointestinal symptoms, transient liver enzyme elevation, and infusion-related reactions are commonly observed and usually manageable.
Long-term effects of dual-target CAR-T in autoimmune disease — including secondary malignancy risk, immune reconstitution patterns, and durability of response — are not yet fully characterized.
Time and travel burden, no guaranteed benefit, possible trial discontinuation, and the need for prolonged stay in Beijing during treatment — these factors should be weighed carefully with the family and treating team.
Trial Location and Hospital Information
This trial is conducted at a single site: Beijing GoBroad Hospital (北京高博医院) in Beijing, China. Beijing GoBroad Hospital is one of GoBroad Healthcare Group's flagship centers and a recognized center for hematology and advanced cell therapy in China. The trial does not have additional locations in other countries.
International patients exploring this trial typically need: organized and translated medical records, a Chinese medical visa (M visa), accommodation near the hospital for the duration of treatment and monitoring, and on-site translation support. CancerFax helps families coordinate document review, hospital communication, visa documentation guidance, travel logistics, and on-site interpretation support. CancerFax does not handle the medical decision or guarantee enrollment — those remain with the Beijing GoBroad Hospital trial team.
Costs, Trial Coverage, and Patient Expenses
Costs for clinical trials vary significantly by trial, sponsor, country, and patient nationality. For international patients, the financial picture is rarely a single number and should be confirmed in writing by the hospital before any travel.
CancerFax helps families understand expected cost categories and what is covered. Final confirmation must come from Beijing GoBroad Hospital in writing. ⚠ VERIFY: specific sponsor coverage policy for international patients on this trial.
Standard Treatment vs Clinical Trial
For severe refractory autoimmune disease, the comparison is usually between continuing or escalating standard immunosuppression versus pursuing an advanced cell therapy trial. Both paths have trade-offs worth weighing carefully.
How CancerFax Helps Patients Explore This Trial
CancerFax is a specialist patient-navigation platform with deep working relationships with leading Chinese cancer and cell therapy hospitals, including Beijing GoBroad Hospital. While CancerFax's primary focus is oncology, the same cell therapy infrastructure, hospital partnerships, and international coordination capability supports patients exploring advanced CAR-T trials in autoimmune disease.
Structured organization, translation, and presentation of complete medical records, treatment history, and biomarker results in a format the trial team can review efficiently.
Mapping the patient's history against the published inclusion and exclusion criteria, identifying gaps, and helping families obtain missing reports before submission.
Direct coordination with the Beijing GoBroad Hospital trial team — submitting case summaries, relaying medical questions, and tracking review status.
Guidance on Chinese medical visas, accommodation near the hospital, airport transfer, and the practical logistics of a multi-month treatment stay.
If this specific trial is not a fit, CancerFax helps explore other advanced cell therapy options, including single-target CAR-T trials and adjacent autoimmune CAR-T programs in China.
On-site interpretation, family communication during the inpatient window, follow-up coordination after discharge, and ongoing support through long-term monitoring.
CancerFax does not guarantee trial enrollment, treatment response, or outcome. Our role is to help patients access accurate information and appropriate pathways.
Questions to Ask Before Considering This Trial
These are the kinds of questions worth discussing in detail with your treating physician and, if a referral is made, with the trial team at Beijing GoBroad Hospital before any decision is made.
Frequently Asked Questions
Want to Know Whether This Trial May Be Relevant?
CancerFax can help review the case, organize medical records, coordinate translation, and request a preliminary review by the Beijing GoBroad Hospital trial team. There is no obligation, and the review process itself does not commit the patient to anything.
The information on this page is for educational and patient-navigation purposes only. It does not replace medical advice, diagnosis, or treatment from a qualified physician. Clinical trial eligibility, enrollment, treatment decisions, and costs are determined only by the trial investigators, hospital, sponsor, and applicable regulations. CancerFax helps patients and families understand options and coordinate case review where appropriate, but does not guarantee trial acceptance, treatment response, or clinical outcome. All clinical decisions must be made in consultation with a qualified, licensed physician with access to the patient's complete medical information.
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© CancerFax · Specialist cancer access and patient-navigation platform. CancerFax is not a medical institution, hospital, or clinical trial sponsor. Trial details may change; always confirm current eligibility, status, and costs directly with the trial center.