CABA-201: Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
CABA-201: Single intravenous infusion of CABA-201 at escalating dose levels without preconditioning
Study 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
Eligibility
Sex
ALL
Min age
18 Years
Max age
70 Years
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, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.
* Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures
* Significant lung or cardiac impairment
* Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant
Other protocol-defined inclusion/exclusion criteria may apply.
Primary outcome measure(s)
To evaluate incidence and severity of adverse events (AEs) — Up to 28 days after CABA-201 infusion 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.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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