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NCT06359041PHASE1, PHASE2Recruiting

RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis

Cabaletta Bio

Start Date

12/17/2024

Completion Date

9/1/2029

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

Detailed 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 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. In addition, escalating doses of CABA-201 will be evaluated in patients without CY and FLU preconditioning.

Eligibility Criteria

Age Range: 18 years to 70 years

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.

Interventions

BIOLOGICAL

CABA-201

BIOLOGICAL

CABA-201

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Conditions

Generalized Myasthenia Gravis (gMG)

Locations

University of California Irvine

Orange, California 92868

United States

UC Davis, Department of Neurology

Sacramento, California 95817

United States

University of California, San Francisco

San Francisco, California 94143

United States

University of Colorado

Aurora, Colorado 80045

United States

Mayo Clinic Florida

Jacksonville, Florida 32224

United States

Northwestern Memorial Hospital

Chicago, Illinois 60611

United States

University of Kansas Medical Center

Kansas City, Kansas 66160

United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts 02215

United States

University of Massachusetts Chan Medical School

Worcester, Massachusetts 01655

United States

Mayo Clinic - Rochester

Rochester, Minnesota 55905

United States

Columbia University

New York, New York 10032

United States

University of Rochester Medical Center

Rochester, New York 14642

United States

Oregon Health & Science University

Portland, Oregon 97239

United States

Baylor College of Medicine Neurology Department

Houston, Texas 77030

United States

Houston Methodist Hospital

Houston, Texas 77030

United States

University of Texas MD Anderson Cancer Center

Houston, Texas 77030

United States

Swedish Neuroscience Research

Seattle, Washington 98122

United States