CD19-BCMA CAR-T cell therapy for connective tissue diseases

A Single-center Clinical Study to Evaluate the Safety and Efficacy of CD19-BCMA CAR-T Cell Therapy for Connective Tissue Diseases

This research will try a CD19-BCMA CAR-T cell treatment in people with active, autoantibody-positive connective tissue diseases who have not responded to standard therapies.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 1 Year to 70 Years
• Sex: All
• Sponsor: Guangzhou Bio-gene Technology Co., Ltd (Industry-sponsored)
• Drugs / interventions: CAR-T
• Locations: 1 site (Dongguan, Guangdong)
• Trial ID: NCT07152223 on ClinicalTrials.gov

What this trial studies

This single-arm, single-center investigator-initiated trial will give patients autologous CD19-BCMA CAR-T cells to study safety, pharmacokinetics, and preliminary signs of clinical benefit. Eligible participants have active connective tissue disease (such as SLE, RA, SSc, SS, PM/DM, or MCTD), at least one positive autoantibody, and inadequate response or intolerance to standard treatments. Treatment will follow typical CAR-T procedures including cell collection, manufacturing, and infusion with defined follow-up for adverse events and immune monitoring. The study is conducted at a single hospital in Dongguan, Guangdong and is sponsored by Guangzhou Bio-gene Technology Co., Ltd.

Who should consider this trial

Good fit: Ideal candidates are adults with active, autoantibody-positive connective tissue disease who have failed, are intolerant of, or relapsed after at least one standard therapy and who can comply with consent, contraception, and follow-up requirements.

Not a fit: Patients with inactive disease, seronegative cases, those well controlled on standard therapies, pregnant individuals, or those with contraindications to CAR-T or significant comorbidities are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, this approach could reduce or eliminate disease-causing B cells and plasma cells and produce longer-lasting remission for refractory connective tissue diseases.

How similar studies have performed: Small case series of CD19-targeted CAR-T have produced remissions in refractory autoimmune disease and BCMA-targeted CAR-T is established in plasma cell disorders, but combined CD19-BCMA CAR-T for connective tissue diseases is largely novel.

Eligibility criteria

Inclusion Criteria:

1. Good compliance: The patient or their guardian understands and voluntarily signs the informed consent form, and it is expected that all documents, procedures, follow-up examinations and treatments stipulated in the study can be completed.
2. Previously diagnosed with one of the following diseases and meeting the corresponding latest international classification/diagnostic criteria :

   1. Systemic lupus erythematosus (SLE);
   2. Rheumatoid arthritis (RA);
   3. Systemic scleroderma (SSc) ;
   4. Sjogren's Syndrome (SS);
   5. Polymyositis/dermatomyositis (PM/DM);
   6. Mixed connective tissue disease (MCTD).
3. Researchers determine that the disease is in an active stage;
4. At least one autoantibody is positive in the serum or plasma during the screening period;
5. Insufficient response, intolerance or recurrence to at least one standard treatment;
6. The pregnancy test of the female subjects was negative, and the subjects agreed to take effective contraceptive measures throughout the trial period.
7. The patient's major tissues and organs are functioning well.

Exclusion Criteria:

1. Intracranial hypertension or confusion;
2. Symptomatic heart failure or severe arrhythmia;
3. Respiratory failure;
4. Accompanied by other types of malignant tumors;
5. Diffuse vascular coagulation;
6. Serum creatinine and/or urea nitrogen ≥ 1.5 times the normal value;
7. Sepsis or other difficult-to-control infections;
8. Uncontrollable diabetes;
9. Severe mental disorder;
10. Cranial magnetic resonance imaging (MRI) examination revealed obvious lesions in the intracranial cavity.
11. WHO physical status classification ≥ level 3;
12. Organ transplant recipients;
13. Pregnant and lactating women;
14. HIV+，HBV，HCV，EBV，CMV.

Where this trial is running

Dongguan, Guangdong

• Dongguan Taixin Hospital — Dongguan, Guangdong, China (Recruiting)

Study contacts

• Principal investigator: Yuelin He, Ph.D. degree — Dongguan Taixin Hospital
• Study coordinator: Yuelin He, Ph.D. degree

Conditions Connective Tissue Diseases