Investigating a new cell therapy for pemphigus vulgaris
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
This study is testing a new cell therapy for people with pemphigus vulgaris who haven't found relief with standard treatments to see if it can help them feel better.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Cabaletta Bio Industry-sponsored |
| Drugs / interventions | Rituximab, Prednisone, CART, Chimeric Antigen Receptor |
| Locations | 13 sites (Redwood City, California and 12 other locations) |
| Trial ID | NCT04422912 on ClinicalTrials.gov |
What this trial studies
This phase 1/2 open-label study evaluates the safety and dosing of autologous CART cells, specifically DSG3-CAART and CABA-201, in patients with active pemphigus vulgaris who have not responded adequately to standard treatments. The study aims to determine the maximum tolerated dose and optimal infusion schedule for DSG3-CAART, while also assessing the safety and efficacy of CABA-201 without preconditioning. Patients will be monitored for clinical responses and potential remission of their disease.
Who should consider this trial
Good fit: Ideal candidates include individuals with a confirmed diagnosis of mucosal pemphigus vulgaris who have not adequately responded to standard immunosuppressive therapies.
Not a fit: Patients with active cutaneous lesions or those currently on high doses of prednisone may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could lead to complete and durable remission for patients suffering from pemphigus vulgaris.
How similar studies have performed: While this approach is innovative, similar studies using CAR-T therapies have shown promise in other autoimmune conditions, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria for DSG3-CAART: Closed to enrollment * 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 Inclusion Criteria for CABA-201 sub-study: Open to enrollment * 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 Exclusion Criteria: * 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 Exclusion Criteria for CABA-201 sub-study * 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.
Where this trial is running
Redwood City, California and 12 other locations
- Stanford University, Dept. of Dermatology — Redwood City, California, United States (Recruiting)
- UC Davis, Dept. of Dermatology — Sacramento, California, United States (Recruiting)
- Yale University — New Haven, Connecticut, United States (Recruiting)
- Northwestern University — Chicago, Illinois, United States (Recruiting)
- University of Iowa — Iowa City, Iowa, United States (Recruiting)
- Brigham and Women's Hospital — Boston, Massachusetts, United States (Recruiting)
- Mount Sinai - Icahn School of Medicine — New York, New York, United States (Withdrawn)
- Columbia University — New York, New York, United States (Recruiting)
- University of North Carolina, Department of Dermatology — Chapel Hill, North Carolina, United States (Withdrawn)
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (Recruiting)
- UT Southwestern Medical Center, Dept. of Dermatology — Dallas, Texas, United States (Recruiting)
- MD Anderson Texas Medical Center — Houston, Texas, United States (Recruiting)
- University of Washington — Seattle, Washington, United States (Completed)
Study contacts
- Study coordinator: Cabaletta Bio
- Email: clinicaltrials@cabalettabio.com
- Phone: +1 267 759 3100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.