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NCT06359041 | Not yet recruiting | Generalized Myasthenia Gravis (gMG)

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

Cabaletta Bio

Brief 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

Condition or disease

Generalized Myasthenia Gravis (gMG)

Intervention/treatment

CABA-201

Phase

Phase 1

Phase 2

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.

Study Type : Interventional
Estimated Enrollment : 12 participants
Masking : None (Open Label)
Primary Purpose : Treatment
Official Title : 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
Actual Study Start Date : August 2024
Estimated Primary Completion Date : September 2029
Estimated Study Completion Date : September 2029
Arm Intervention/treatment

Experimental: CABA-201

AChR Antibody-Positive Cohort AChR Antibody-Negative Cohort

Biological: CABA-201
Ages Eligible for Study: 18 Years to 70 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
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.
RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis

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

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