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)
1 other identifier
interventional
40
1 country
13
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Sep 2020
Longer than P75 for phase_1
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 2, 2020
CompletedFirst Posted
Study publicly available on registry
June 9, 2020
CompletedStudy Start
First participant enrolled
September 29, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 1, 2029
December 17, 2025
December 1, 2025
8.3 years
June 2, 2020
December 11, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Adverse events, including Dose Limit Toxicity
Incidence of adverse events that are related to DSG3-CAART therapy
3 months
For CABA-201 Sub-study: To evaluate adverse events reported by subjects
Incidence and severity of AEs
Up to 28 days after CABA-201 infusion
Secondary Outcomes (14)
Percent of CAAR-transduced cells
Baseline
Total DSG3-CAART positive cells
Baseline
Cellular kinetics profile of DSG3-CAART
Up to 36 months
Change in DSG3 autoantibody titer
Up to 36 months
Serologic remission
Up to 36 months
- +9 more secondary outcomes
Study Arms (2)
DSG3-CAART
EXPERIMENTALSingle or multiple intravenous infusion(s) of DSG3-CAART at varying dose levels. This study is now closed to enrollment.
CABA-201
EXPERIMENTALInfusion of CABA-201 with or without cyclophosphamide and fludarabine preconditioning, or with or without cyclophosphamide preconditioning. This sub-study is open to enrollment.
Interventions
Intravenous infusions of DSG3-CAART alone at different doses and different fractionations, with or without intravenous immunoglobulin, cyclophosphamide, and/or fludarabine.
Single intravenous infusion of CABA-201 at escalating doses, with or without preconditioning.
Eligibility Criteria
You may qualify if:
- Confirmed diagnosis of mPV by prior or screening biopsy and prior positive anti- DSG3 antibody ELISA
- mPV inadequately managed by at least one standard immunosuppressive therapies
- Active mPV at screening
- Anti-DSG3 antibody ELISA positive at screening
- Age ≥18
- Confirmed diagnosis of PV by prior or screening biopsy and prior positive DSG3 ELISA, IIF, and/or DIF
- PV inadequately managed by at least one standard immunosuppressive therapy
- Active PV at screening
- DSG3 ELISA positive at screening
You may not qualify if:
- Active cutaneous lesions associated with PV that indicates mucocutaneous rather than mucosal-dominant disease
- Rituximab in last 12 months unless PV symptoms have recently worsened or anti-DSG3 antibody titers have recently increased
- Prednisone \> 0.25mg/kg/day
- Other autoimmune disorder requiring immunosuppressive therapies
- Investigational treatment in last 3 months
- Have paraneoplastic pemphigus or active malignancy (not including non-melanoma skin cancer) or malignancy diagnosed within the previous 5 years
- Have received rituximab or other anti-CD20 or anti-CD19 therapies in last 12 months unless anti-DSG3 antibody titers have recently increased or PV symptoms have recently worsened
- Prednisone \> 0.25mg/kg/day
- Other autoimmune disorder requiring immunosuppressive therapies
- Treatment with any investigational agent within 4 weeks or 5 half-lives, whichever is longer.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cabaletta Biolead
Study Sites (13)
Stanford University, Dept. of Dermatology
Redwood City, California, 94063, United States
UC Davis, Dept. of Dermatology
Sacramento, California, 95816, United States
Yale University
New Haven, Connecticut, 06520, United States
Northwestern University
Chicago, Illinois, 60611, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
Mount Sinai - Icahn School of Medicine
New York, New York, 10029, United States
Columbia University
New York, New York, 10032, United States
University of North Carolina, Department of Dermatology
Chapel Hill, North Carolina, 27516, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
UT Southwestern Medical Center, Dept. of Dermatology
Dallas, Texas, 75235, United States
MD Anderson Texas Medical Center
Houston, Texas, 77030, United States
University of Washington
Seattle, Washington, 98109, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Cabaletta Bio
Cabaletta Bio
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 2, 2020
First Posted
June 9, 2020
Study Start
September 29, 2020
Primary Completion (Estimated)
January 1, 2029
Study Completion (Estimated)
January 1, 2029
Last Updated
December 17, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will not share