RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis
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
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, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.
Single group
CABA-201
Biological/VaccineSingle intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
CABA-201
Biological/VaccineSingle intravenous infusion of CABA-201 at escalating dose levels without preconditioning
To evaluate incidence and severity of adverse events (AEs)
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal result of an investigation), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The term AE is used to include both serious and non-serious AEs.
To evaluate the incidence and severity of adverse events (AEs)
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal result of an investigation), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The term AE is used to include both serious and non-serious AEs.
To characterize the pharmacodynamics (PD)
Levels of B cells in the blood
To characterize the pharmacokinetics (PK)
Levels of CABA-201-positive T cells in the blood
To evaluate disease-related biomarkers
Levels of MG-specific autoantibodies in the serum
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