CAR-T cell therapy targeting specific markers in autoimmune diseases

Sequential CAR-T Cells Targeting BCMA/CD19 in Patients With Relapsed/ Refractory Autoimmune Diseases

This study is testing a new CAR-T cell therapy to see if it can help people with tough autoimmune diseases like lupus and Sjogren's syndrome feel better.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: 21 Years to 90 Years
• Sex: All
• Sponsor: Essen Biotech (Academic / other)
• Drugs / interventions: CAR-T, chimeric antigen receptor, immunotherapy, CAR T
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06428188 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of chimeric antigen receptor T cell (CAR-T) immunotherapy targeting BCMA and CD19 in patients with relapsed or refractory autoimmune diseases, including systemic lupus erythematosus and Sjogren's syndrome. The study is open-label and single-arm, focusing on assessing the safety profile, including potential cytokine storms, and the treatment's impact on disease status. Participants will receive CAR-T cells designed to target specific B cell markers associated with their autoimmune conditions. The trial aims to enhance understanding of CAR-T cell functionality and persistence in this patient population.

Who should consider this trial

Good fit: Ideal candidates are individuals with recurrent or refractory autoimmune diseases who have not responded to standard treatments.

Not a fit: Patients with stable autoimmune diseases that are well-managed with existing therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a novel treatment option for patients with difficult-to-treat autoimmune diseases.

How similar studies have performed: While CAR-T therapies have shown success in hematological malignancies, their application in autoimmune diseases is still novel and largely untested.

Eligibility criteria

Inclusion Criteria:

* Expected survival time ≥3 months;
* Subjects with recurrent/refractory autoimmune diseases who have failed standard treatment or lack effective treatment, Including but not limited to systemic lupus erythematosus, idiopathic inflammatory myopathy, systemic sclerosis, IGG4-associated diseases, primary Sjogren's syndrome, rheumatoid arthritis, connective tissue disease-associated interstitial lung disease, immune thrombocytopenia, primary biliary cholangitis, etc.
* Histological evidence of non-suppurative destructive cholangitis and small bile duct destruction.
* Liver and kidney function, cardiopulmonary function meet the following requirements:
* Creatinine ≤1.5×ULN; (2) Electrocardiogram showed no clinically significant abnormal bands;
* Blood oxygen saturation \>91% in non-oxygen state;
* Total bilirubin ≤2×ULN; ALT and AST≤2.5 x ULN; ALT and AST abnormalities due to disease, such as liver infiltration or bile duct obstruction, were determined to be less than 5×ULN. If Gilbert syndrome is diagnosed, the total bilirubin index can be relaxed to ≤3.0×ULN and the direct bilirubin ≤1.5×ULN.
* No serious mental disorders;
* Can understand this test and have signed the informed consent.

Exclusion Criteria:

* Malignant tumors other than R/R AID disease in the 5 years prior to screening, except for adequately treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, local prostate cancer after radical surgery, and breast ductal carcinoma in situ after radical surgery;
* Hepatitis B surface antigen (HBsAg) positive; Hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer detection is not within the normal reference value range; Hepatitis C virus (HCV) Antibody positive and peripheral blood hepatitis C virus (HCV) RNA positive; Human immunodeficiency virus (HIV) Antibody positive; Syphilis positive;
* Serious heart disease, including but not limited to unstable angina, myocardial infarction or bypass or stent surgery (within 6 months prior to screening), congestive heart failure (NYHA classification ≥III), and severe arrhythmia;
* Systemic diseases that are deemed unstable by researchers: including but not limited to severe liver, kidney, or metabolic diseases that require drug treatment;
* Active or uncontrollable infections (except mild genitourinary and upper respiratory tract infections) that require systemic treatment within 7 days prior to administration;
* Pregnant or lactating women, and female subjects who plan pregnancy within 2 years after cell transfusion or male subjects whose partners plan pregnancy within 2 years after cell transfusion;
* Patients who received CAR-T therapy or other gene-modified cell therapy before screening;
* Participated in other clinical studies 1 month before screening;
* Evidence of central nervous system invasion during subject screening;
* Mental patients with depression or suicidal thoughts;
* Situations considered unsuitable for inclusion by other researchers.

Where this trial is running

Beijing, Beijing Municipality

• District One Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Rhoda M Smith, Phd

Conditions Autoimmune Diseases; Systemic Lupus Erythematosus; Systemic Lupus Erythematosus Acute; Sjogren's Syndrome; CAR-T