Summary

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

Description

Summary

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

Keywords

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

Eligibility

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 < 1,000/µL at screening.

Locations

  • UC Irvine, Department of Neurology accepting new patients
    Orange California 92868 United States
  • Oregon Health & Science University (OHSU) not yet accepting patients
    Portland Oregon 97239 United States

Lead Scientist at UC Irvine

  • Ali Habib, MD
    Associate Clinical Professor, Neurology, School of Medicine. Authored (or co-authored) 4 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Cabaletta Bio
ID
NCT05451212
Phase
Phase 1 Myasthenia Gravis Research Study
Study Type
Interventional
Participants
Expecting 24 study participants
Last Updated