CD19 CAR-T Therapy for Refractory Pemphigus Vulgaris
China3 participantsStarted 2026-01-15
Plain-language summary
This is an investigator-initiated, single-center, single-arm, open-label exploratory clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of autologous CD19 CAR-T cell therapy in patients with refractory pemphigus vulgaris.
Who can participate
Age range
18 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Ability to provide written informed consent.
. Age 18 to 70 years at screening, male or female.
. Diagnosis of pemphigus vulgaris confirmed by clinical presentation, histopathology, direct immunofluorescence (DIF), and positive anti-desmoglein 3 and/or anti-desmoglein 1 antibodies.
. Moderate-to-severe disease activity defined as Pemphigus Disease Area Index (PDAI) ≥ 15 at screening.
. Refractory pemphigus vulgaris is defined as inadequate response, disease relapse, or treatment dependence following systemic corticosteroids and rituximab-based therapy for at least 6 months, with persistent disease activity meeting at least one of the following criteria:
. Ongoing active disease with the appearance of new erythema, blisters, or erosions;
. Inability to taper systemic corticosteroids to \< 20 mg/day prednisone equivalent (i.e., ≥ 4 tablets/day of standard prednisone dosing).
Exclusion criteria
. Active uncontrolled infection at screening, requiring systemic antimicrobial therapy. Participants with active tuberculosis, hepatitis C virus (HCV), human immunodeficiency virus (HIV), or syphilis infection will be excluded. Participants with hepatitis B surface antigen and/or hepatitis B core antibody positivity may be eligible only if HBV DNA is below the lower limit of quantification and appropriate antiviral prophylaxis is provided at the investigator's discretion.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence and Severity of Treatment-Emergent Adverse Events
Timeframe: From initiation of lymphodepleting chemotherapy through Day 90 after CAR-T cell infusion
2
Change From Baseline in PDAI Score at Week 12
Timeframe: Baseline and Week 12
3
Proportion of participants achieving disease control at Week 4 after CAR-T infusion
. History of other active autoimmune disease requiring systemic immunosuppression.
. Previous treatment with any gene-modified cellular therapy, including CAR-T or CAR-NK therapy.
. Prior allogeneic stem cell or solid organ transplantation.
. Severe or uncontrolled cardiovascular, pulmonary, hepatic, or renal disease that would increase risk associated with lymphodepleting chemotherapy or CAR-T cell infusion.
. Use of high-dose systemic corticosteroids (\>1 mg/kg/day prednisone equivalent) within 7 days prior to leukapheresis.
. Use of rituximab or other B-cell-targeted biologics within protocol-defined washout period.
. Use of intravenous immunoglobulin, plasma exchange, or other intensive immunomodulatory therapy within 2 weeks prior to leukapheresis.