Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis

Description

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.

Conditions

MuSK Myasthenia Gravis

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Study Overview

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Study Details

Study overview

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.

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

Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis

Condition
MuSK Myasthenia Gravis
Intervention / Treatment

-

Contacts and Locations

Orange

UC Irvine, Department of Neurology, Orange, California, United States, 92868

Sacramento

UC Davis, Department of Neurology, Sacramento, California, United States, 95817

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Portland

Oregon Health & Science University (OHSU), Portland, Oregon, United States, 97239

Houston

Houston Methodist Hospital, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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
  • * 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.

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Cabaletta Bio,

Medical Director, STUDY_CHAIR, Cabaletta Bio

Study Record Dates

2028-10