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CD19 CAR-T Therapy for Refractory Pemphigus Vulgaris

ConditionPemphigus Vulgaris
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-70
SponsorJinbo Chen

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Autologous CD19 CAR-T cells

    Biological/Vaccine

    Autologous CD19 CAR-T cells are manufactured ex vivo using the participant's own T cells collected by leukapheresis. The CAR construct targets CD19-expressing B cells and consists of a single-chain variable fragment directed against CD19. Participants will receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide prior to CAR-T cell infusion according to the protocol-defined schedule. Following completion of lymphodepletion, each participant will receive a single intravenous infusion of autologous CD19 CAR-T cells under inpatient monitoring. Premedication, including acetaminophen and diphenhydramine, may be administered prior to infusion at the investigator's discretion in accordance with institutional practice.

Treatment groups

3 Participants
are divided into 1 treatment group
Group A: CD19 CAR-T Therapy for Refractory Pemphigus VulgarisExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and Severity of Treatment-Emergent Adverse Events

Treatment-emergent adverse events, serious adverse events, dose-limiting toxicities, cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, infections, cytopenias, organ toxicities, infusion-related reactions, and clinically significant laboratory abnormalities will be assessed. Adverse events will be graded according to CTCAE v5.0, and immune effector cell-related toxicities will be assessed according to applicable consensus grading criteria.

Time frame
From initiation of lymphodepleting chemotherapy through Day 90 after CAR-T cell infusion
2

Change From Baseline in PDAI Score at Week 12

The change from baseline in Pemphigus Disease Area Index (PDAI) score at Week 12 will be assessed to evaluate preliminary clinical efficacy of autologous CD19 CAR-T cell therapy in patients with refractory pemphigus vulgaris.

Time frame
Baseline and Week 12
3

Proportion of participants achieving disease control at Week 4 after CAR-T infusion

Disease control is defined as cessation of new active cutaneous or mucosal lesions with established lesion healing or no further progression of existing lesions, as assessed by the investigator. This endpoint evaluates the early clinical response following CD19 CAR-T cell therapy and reflects initial disease stabilization after B-cell depletion.

Time frame
Week 4

Secondary outcomes

1

Change From Baseline in Pemphigus Disease Area Index （PDAI） Score Over Time (Range 0-263)

Changes from baseline in Pemphigus Disease Area Index (PDAI) score will be assessed at scheduled follow-up visits to evaluate the durability of clinical response. The total PDAI score ranges from 0 to 263, with higher scores indicating more severe disease.

Time frame
Baseline through Week 96 after CAR-T cell infusion
2

Change From Baseline in Physician Global Assessment Score (PGA) (Range 0-10)

Change from baseline in PGA score will be assessed at scheduled follow-up visits. PGA is a physician-rated global assessment of pemphigus disease severity, with higher scores indicating more severe disease activity.

Time frame
Baseline through Week 96 after CAR-T cell infusion
3

Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) (Range 0-206)

Change from baseline in ABSIS will be assessed at scheduled follow-up visits. ABSIS is used to evaluate autoimmune bullous disease activity, with higher scores indicating more severe disease activity.

Time frame
Baseline through Week 96 after CAR-T cell infusion
4

Proportion of participants achieving disease control at Week 12

Disease control is defined as cessation of new active cutaneous or mucosal lesions with healing or stabilization of existing lesions, as assessed by the investigator. This endpoint evaluates the durability and consolidation of clinical response following CD19 CAR-T cell therapy in patients with refractory pemphigus vulgaris.

Time frame
Week 12

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Jinbo Chen

Lead sponsor

Wuhan Integrated Traditional Chinese and Western Medicine Hospital

Sponsor institution

Source ClinicalTrials.gov
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