RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis
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 the safety and efficacy of an investigational cell therapy, CABA-201, that can be given to patients with gMG. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
University of California Irvine
Orange, California, United States
RECRUITINGTo 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.
Time frame: Up to 28 days after CABA-201 infusion
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.
Time frame: Up to 156 weeks
To characterize the pharmacodynamics (PD)
Levels of B cells in the blood
Time frame: Up to 156 weeks
To characterize the pharmacokinetics (PK)
Levels of CABA-201-positive T cells in the blood
Time frame: Up to 156 weeks
To evaluate disease-related biomarkers
Levels of MG-specific autoantibodies in the serum
Time frame: Up to 156 weeks
To evaluate efficacy by change in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score over time.
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UC Davis, Department of Neurology
Sacramento, California, United States
RECRUITINGUniversity of California, San Francisco
San Francisco, California, United States
RECRUITINGUniversity of Colorado
Aurora, Colorado, United States
RECRUITINGMayo Clinic Florida
Jacksonville, Florida, United States
RECRUITINGNorthwestern Memorial Hospital
Chicago, Illinois, United States
RECRUITINGUniversity of Kansas Medical Center
Kansas City, Kansas, United States
RECRUITINGBeth Israel Deaconess Medical Center
Boston, Massachusetts, United States
RECRUITINGUniversity of Massachusetts Chan Medical School
Worcester, Massachusetts, United States
RECRUITINGMayo Clinic - Rochester
Rochester, Minnesota, United States
RECRUITING...and 7 more locations
The MG-ADL scale is a validated instrument administered by a qualified assessor with 8 areas of activities of daily living as reported by patients: talking, chewing, swallowing, breathing, ability to brush teeth or comb hair, ability to rise from a chair, double vision, and eyelid droop. In each area, the score ranges from 0 (normal) to 3 (most severe). The total score is the sum of all subscores.
Time frame: Up to 156 weeks
To evaluate efficacy by change in Quantitative Myasthenia Gravis (QMG) score over time.
The QMG score is a 13-item scale conducted by a qualified assessor to evaluate disease severity in patients with MG. The total score ranges from a minimum of 0 (no myasthenic findings) to 39 (maximum myasthenic deficits).
Time frame: Up to 156 weeks
To evaluate efficacy by change in Myasthenia Gravis Composite (MGC) score over time.
The MGC scale is a physician-reported instrument for the assessment of MG patients' symptoms and impairments. It includes 10 domains, and the total score ranges from 0 (normal) to 50 (most severe).
Time frame: Up to 156 weeks