Summary

Eligibility
for people ages 18-70 (full criteria)
Location
at Orange, California and other locations
Dates
study started
completion around
Principal Investigator
by Ali Habib, MD

Description

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

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

Details

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.

Keywords

Generalized Myasthenia Gravis (gMG), CABA-201, autoimmune disease, anti-CD19 CAR-T therapy, cellular therapy, Myasthenia Gravis, Neuromuscular, Anti-AChR, Anti-LRP4, Anti-MuSK, Muscle Weakness

Eligibility

You can join if…

Open to people ages 18-70

  • 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

You CAN'T join if...

  • 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.

Locations

  • University of California Irvine accepting new patients
    Orange California 92868 United States
  • UC Davis, Department of Neurology accepting new patients
    Sacramento California 95817 United States
  • Houston Methodist Hospital accepting new patients
    Houston Texas 77030 United States

Lead Scientist at UC Irvine

  • Ali Habib, MD
    Health Sciences Professor, Neurology, School of Medicine. Authored (or co-authored) 30 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Cabaletta Bio
ID
NCT06359041
Phase
Phase 1/2 Myasthenia Gravis Research Study
Study Type
Interventional
Participants
Expecting 12 study participants
Last Updated