for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
completion around
Principal Investigator
by Ali Habib, MD



Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction. This phase 1 study is being conducted to evaluate the safety of various dosing regimens of an investigational cell therapy, MuSK-CAART, that can be given to patients with anti-MuSK antibody positive Myasthenia Gravis (MuSK MG), who have active disease. Various dosing regimens of MuSK-CAART alone, in combination with cyclophosphamide (CY), and in combination with CY and fludarabine (FLU) will be evaluated. Treatment with MuSK-CAART may potentially lead to complete and durable remission of disease.

Official Title

A Phase 1, Open-label, Safety and Dose-finding Study of Autologous Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART) in Subjects With Anti-MuSK-antibody-positive Myasthenia Gravis


MuSK Myasthenia Gravis, CAAR-T (Chimeric Autoantibody Receptor T Cells) Therapy, CAR-T (Chimeric Antigen Receptor T Cells) Therapy, Cell Therapy, Autoimmune Disease, Autoimmunity, Immunotherapy, Adoptive, Immune System Diseases, Myasthenia Gravis (MG), Muscle-specific tyrosine kinase (MuSK), Muscle Weakness, Neuromuscular Diseases, Musculoskeletal Diseases, Myasthenia Gravis, MuSK-CAART


You can join if…

Open to people ages 18 years and up

  • Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSK antibody test.
  • History of a negative anti-AChR (acetylcholine receptor) antibody test.
  • Positive anti-MuSK antibody test at screening
  • MG severity Class I to IVa on the MGFA (Myasthenia Gravis Foundation of America) Clinical Classification

You CAN'T join if...

  • Rituximab in the last 12 months.
  • Prednisone > 0.25mg/kg/day [in Part A]
  • Other autoimmune disorder requiring immunosuppressive therapies.
  • Investigational treatment for MG in the past 12 weeks.
  • Absolute lymphocyte count < 500/µL at screening.


  • UC Irvine, Department of Neurology accepting new patients
    Orange California 92868 United States
  • UC Davis, Department of Neurology accepting new patients
    Sacramento California 95817 United States

Lead Scientist at UC Irvine

  • Ali Habib, MD
    Health Sciences Professor, Neurology, School of Medicine. Authored (or co-authored) 19 research publications


accepting new patients
Start Date
Completion Date
Cabaletta Bio
Phase 1 Myasthenia Gravis Research Study
Study Type
Expecting 24 study participants
Last Updated