Actively Recruiting
A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CART Cells (Desmoglein 3 Chimeric Autoantibody Receptor T Cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T Cells [CABA-201]) in Subjects With Active, Pemphigus Vulgaris
Led by Cabaletta Bio · Updated on 2026-05-29
40
Participants Needed
13
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating autologous Chimeric Antigen Receptor T cells (CART cells) in people with active pemphigus vulgaris (PV), a condition causing painful blisters on skin and mucous membranes. This phase 1/2 open-label study includes two parts: the main study focused on DSG3-CAART therapy for mucosal PV patients not well controlled by standard treatments, and a sub-study assessing CABA-201 therapy in mucosal-dominant and mucocutaneous PV patients. The study aims to find safe dosing and to understand if these therapies can control disease activity. The main study (now closed to enrollment) tested different doses and infusion schedules of DSG3-CAART, sometimes combined with other treatments. The open sub-study involves a single infusion of CABA-201 with varying preconditioning regimens using cyclophosphamide and fludarabine. Treatments are given intravenously, and the sub-study is currently enrolling participants. Participants will be monitored for adverse events up to 3 months for DSG3-CAART and up to 156 weeks for CABA-201. Researchers will measure safety, immune cell levels, antibody changes, disease activity scores, and remission status over up to 36 months. The study includes regular clinical assessments, laboratory tests, and ongoing safety follow-up to evaluate treatment effects and tolerability.
CONDITIONS
Brief Title
A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CART Cells (Desmoglein 3 Chimeric Autoantibody Receptor T Cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T Cells [CABA-201]) in Subjects With Active, Pemphigus Vulgaris (RESET-PV)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- Confirmed diagnosis of pemphigus vulgaris by biopsy and positive DSG3 antibody tests
- Active pemphigus vulgaris symptoms at screening
- Disease inadequately managed by at least one standard immunosuppressive therapy
- Positive anti-DSG3 antibody test at screening
You will not qualify if you...
- Active cutaneous lesions indicating mucocutaneous rather than mucosal-dominant disease
- Use of rituximab or other anti-CD20 or anti-CD19 therapies within the last 12 months unless recent worsening of symptoms or antibody levels
- Prednisone dose greater than 0.25 mg/kg/day
- Other autoimmune disorders requiring immunosuppressive therapy
- Treatment with investigational agents within the last 3 months for DSG3-CAART or within 4 weeks or 5 half-lives for CABA-201
- Diagnosis of paraneoplastic pemphigus or active malignancy within 5 years (excluding non-melanoma skin cancer) for CABA-201 sub-study participants
- Recent rituximab or anti-CD20/anti-CD19 therapies within 12 months unless symptoms or antibody levels have worsened for CABA-201 sub-study participants
- Prednisone dose greater than 0.25 mg/kg/day for CABA-201 sub-study participants
- Other autoimmune disorders requiring immunosuppressive therapies for CABA-201 sub-study participants
- Recent treatment with investigational agents as specified for CABA-201 sub-study participants
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 3 months
Participants receive single or multiple intravenous infusions of DSG3-CAART or a single intravenous infusion of CABA-201 at varying dose levels, with or without preconditioning medications.
1 infusion visit and multiple follow-up visits during treatment period
Duration - Up to 36 months for DSG3-CAART and up to 156 weeks for CABA-201
Participants are monitored for safety, clinical response, and biomarker changes after treatment infusion.
Regular follow-up visits over several months to years
Trial Site Locations
Total: 13 locations
1
Stanford University, Dept. of Dermatology
Redwood City, California, United States, 94063
Actively Recruiting
2
UC Davis, Dept. of Dermatology
Sacramento, California, United States, 95816
Actively Recruiting
3
Yale University
New Haven, Connecticut, United States, 06520
Actively Recruiting
4
Northwestern University
Chicago, Illinois, United States, 60611
Actively Recruiting
5
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
6
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
7
Mount Sinai - Icahn School of Medicine
New York, New York, United States, 10029
Withdrawn
8
Columbia University
New York, New York, United States, 10032
Actively Recruiting
9
University of North Carolina, Department of Dermatology
Chapel Hill, North Carolina, United States, 27516
Withdrawn
10
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
11
UT Southwestern Medical Center, Dept. of Dermatology
Dallas, Texas, United States, 75235
Actively Recruiting
12
MD Anderson Texas Medical Center
Houston, Texas, United States, 77030
Actively Recruiting
13
University of Washington
Seattle, Washington, United States, 98109
Completed
Research Team
C
Cabaletta Bio
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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