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)

Launched by CABALETTA BIO · Jun 5, 2020

Keywords

ClinConnect Summary

This clinical trial, called RESET-PV, is looking at a new treatment for people with pemphigus vulgaris (PV), a rare autoimmune disease that causes painful blisters and sores on the skin and mucous membranes. The study is testing two types of specialized immune cells, known as CART cells, to see if they can help manage the symptoms in patients whose condition has not improved with standard treatments. It is currently open for participants aged 65 to 74 who have been diagnosed with active pemphigus vulgaris and have tested positive for a specific antibody linked to the disease.

To join the trial, participants must have had their diagnosis confirmed through a biopsy and must have tried at least one standard treatment without satisfactory results. Those who have certain skin lesions or have received specific treatments recently may not qualify. If eligible, participants can expect to receive the CART cell therapy and will be closely monitored for safety and effectiveness throughout the study. This trial is crucial for exploring new options for managing pemphigus vulgaris and improving patients' quality of life.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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
• • 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)
• • 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
• • Investigational treatment in last 3 months