What is autologous CD19 CAR-T cell therapy studying for refractory pemphigus vulgaris?

This trial is studying a personalized cellular therapy that uses a participant's own immune cells to target B cells involved in pemphigus vulgaris. Researchers aim to evaluate the safety, tolerability, and early effectiveness of this treatment in adults whose disease has not improved with standard therapies.

Who may be eligible for this pemphigus vulgaris clinical trial?

Adults aged 18 to 70 with moderate-to-severe refractory pemphigus vulgaris confirmed by specific tests may be eligible. Participants must have had prior treatment with corticosteroids and rituximab and still have active disease. Both men and women can join. Please review eligibility criteria for more details.

Is there any cost to participate in this pemphigus vulgaris clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and other expenses may vary and are not guaranteed to be covered.

Actively Recruiting

Phase Not Applicable

Age: 18Years - 70Years

All Genders

ID07681388

Evaluating Safety and Early Effectiveness of Personalized CD19 CAR-T Cell Therapy in Adults With Moderate-to-Severe Refractory Pemphigus Vulgaris

Led by Jinbo Chen · Updated on 2026-07-02

Participants Needed

Research Sites

104 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating autologous CD19 CAR-T cell therapy in adults with moderate-to-severe refractory pemphigus vulgaris, a condition that has not responded well to standard treatments. This exploratory, single-center study aims to assess the safety, tolerability, and early effectiveness of this patient-specific cellular therapy. The study also investigates how the therapy affects immune response and disease activity over time. Participants will first undergo leukapheresis to collect their own immune cells, which are then engineered outside the body to target CD19-positive B cells. Before receiving the CAR-T cells, participants will have lymphodepleting chemotherapy with fludarabine and cyclophosphamide to prepare their body for the infusion. They will then receive a single intravenous infusion of the manufactured CAR-T cells. Premedication may be given before infusion based on the investigators judgment. Throughout the study, participants will be monitored for safety concerns such as inflammatory reactions, infections, and organ function. Effectiveness will be measured through changes in disease severity scores and antibody levels. Additional tests will track immune cell changes and CAR-T cell persistence. Follow-up will continue for up to 96 weeks after infusion to assess long-term outcomes and safety.

CONDITIONS

Brief Title

CD19 CAR-T Therapy for Refractory Pemphigus Vulgaris

Who Can Participate

Age: 18Years - 70Years

All Genders

Eligibility Criteria

You may qualify if you...

Ability to provide written informed consent.
Age between 18 and 70 years, male or female.
Diagnosis of pemphigus vulgaris confirmed by clinical presentation, histopathology, direct immunofluorescence, and positive anti-desmoglein 1 and/or 3 antibodies.
Moderate-to-severe disease activity with Pemphigus Disease Area Index (PDAI) score of 15 or higher at screening.
Refractory pemphigus vulgaris defined by inadequate response, relapse, or treatment dependence after at least 6 months of systemic corticosteroids and rituximab-based therapy, with persistent active disease signs or elevated antibody titers above 100 U/mL, or inability to reduce corticosteroid dose below 20 mg/day prednisone equivalent.
Requirement for systemic therapy at screening due to active disease.
Adequate vascular access for leukapheresis.
Life expectancy greater than 6 months.
Adequate organ function including specified hematologic, renal, hepatic, cardiac, pulmonary, and coagulation parameters.
No clinically significant active infection at baseline.
Women of childbearing potential must have a negative pregnancy test within 48 hours before lymphodepleting chemotherapy and agree to use effective contraception during the study and for 12 months after CAR-T infusion.
Male participants must agree to use effective contraception and avoid sperm donation during the study and for 12 months after infusion.

You will not qualify if you...

Active uncontrolled infection requiring systemic antimicrobial therapy, including active tuberculosis, hepatitis C, HIV, or syphilis.
History of other active autoimmune diseases needing systemic immunosuppression.
Previous treatment with any gene-modified cellular therapy such as CAR-T or CAR-NK.
Prior allogeneic stem cell or solid organ transplantation.
Severe or uncontrolled cardiovascular, pulmonary, hepatic, or renal disease increasing risk with chemotherapy or CAR-T infusion.
Use of high-dose systemic corticosteroids (>1 mg/kg/day prednisone equivalent) within 7 days before leukapheresis.
Use of rituximab or other B-cell-targeted biologics within a protocol-defined washout period.
Use of intravenous immunoglobulin, plasma exchange, or intensive immunomodulatory therapy within 2 weeks before leukapheresis.
Receipt of live vaccine within 8 weeks prior to screening.
History of malignancy within 5 years except treated non-melanoma skin cancer or in situ carcinoma.
Pregnancy or breastfeeding.
Known allergy to any components of lymphodepleting chemotherapy or CAR-T product.
Any condition judged by the investigator to compromise safety or study integrity.

Research Team

Jinbo Chen, MD, PhD

Xiaoya Du

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Evaluating Safety and Early Effectiveness of Personalized CD19 CAR-T Cell Therapy in Adults With Moderate-to-Severe Refractory Pemphigus Vulgaris

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria