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)
- Conditions
- Pemphigus Vulgaris
- Registration Number
- NCT04422912
- Lead Sponsor
- Cabaletta Bio
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 55
Inclusion Criteria:<br><br> - Confirmed diagnosis of mPV by prior or screening biopsy and prior positive anti-<br> DSG3 antibody ELISA<br><br> - mPV inadequately managed by at least one standard immunosuppressive therapies<br><br> - Active mPV at screening<br><br> - Anti-DSG3 antibody ELISA positive at screening<br><br>Inclusion Criteria for CABA-201 sub-study<br><br> - Confirmed diagnosis of PV by prior or screening biopsy and prior positive DSG3<br> ELISA, IIF, and/or DIF<br><br> - PV inadequately managed by at least one standard immunosuppressive therapy<br><br> - Active PV at screening<br><br> - DSG3 ELISA positive at screening<br><br>Exclusion Criteria:<br><br> - Active cutaneous lesions associated with PV that indicates mucocutaneous rather than<br> mucosal-dominant disease<br><br> - Rituximab in last 12 months unless PV symptoms have recently worsened or anti-DSG3<br> antibody titers have recently increased<br><br> - Prednisone > 0.25mg/kg/day<br><br> - Other autoimmune disorder requiring immunosuppressive therapies<br><br> - Investigational treatment in last 3 months<br><br>Exclusion Criteria for CABA-201 sub-study<br><br> - Have paraneoplastic pemphigus or active malignancy (not including non-melanoma skin<br> cancer)<br><br> - Have received rituximab or other anti-CD20 or anti-CD19 therapies in last 12 months<br> unless anti-DSG3 antibody titers have recently increased or PV symptoms have<br> recently worsened<br><br> - Prednisone > 0.25mg/kg/day<br><br> - Other autoimmune disorder requiring immunosuppressive therapies<br><br> - Investigational treatment in last 3 months
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Adverse events, including Dose Limit Toxicity;For CABA-201 Sub-study: To evaluate adverse events reported by subjects
- Secondary Outcome Measures
Name Time Method Percent of CAAR-transduced cells;Total DSG3-CAART positive cells;Cellular kinetics profile of DSG3-CAART;Change in DSG3 autoantibody titer;Serologic remission;Pemphigus Disease Area Index (PDAI);Clinical remission: complete remission off therapy and complete remission on minimal therapy;Time to clinical remission and time to serologic remission;Duration of clinical remission and duration of serologic remission;For CABA-201 Sub-study: To evaluate adverse events reported by subjects;For CABA-201 Sub-study: To characterize the pharmacodynamics (PD);For CABA-201 Sub-study: To characterize the pharmacokinetics (PK);For CABA-201 Sub-study: To evaluate autoantibody -related biomarkers;For CABA-201 Sub-study: To evaluate efficacy