RESET-MG: A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Participants With Generalized Myasthenia Gravis
Cabaletta Bio
Summary
RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis
Description
Myasthenia gravis (MG) is a rare autoimmune disorder characterized by autoantibody responses that cause defective transmission of signals at the neuromuscular junction, resulting in a distinctive pattern of weakness. Patients with generalized MG (gMG) typically experience symptoms associated with ocular disease in addition to weakness of many other voluntary muscle groups, including extremity, bulbar, and respiratory muscles. MG is considered a classic example of a B-cell mediated autoimmune disease. Currently, there are no curative treatments for MG. This study is being conducted to evaluate…
Eligibility
- Age range
- 18–70 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Age ≥18 and ≤70 years of age * Diagnosis of MG with generalized muscle weakness meeting criteria as defined by the MGFA class II, III , IVa, and IVb. * Diagnosis of seropositive (autoantibodies AChR, MuSK and/or LRP4) or seronegative MG Exclusion Criteria: * Contraindication to leukapheresis * History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites * Active infection requiring medical intervention at screening * Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestin…
Interventions
- BiologicalCABA-201
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
- BiologicalCABA-201
Single intravenous infusion of CABA-201 at escalating dose levels without preconditioning
Locations (17)
- University of California IrvineOrange, California
- UC Davis, Department of NeurologySacramento, California
- University of California, San FranciscoSan Francisco, California
- University of ColoradoAurora, Colorado
- Mayo Clinic FloridaJacksonville, Florida
- Northwestern Memorial HospitalChicago, Illinois