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

Last updated: October 25, 2024
Sponsor: Cabaletta Bio
Overall Status: Active - Recruiting

Phase

1

Condition

Neuropathy

Polymyositis (Inflammatory Muscle Disease)

Sarcopenia

Treatment

MuSK-CAART

Clinical Study ID

NCT05451212
CAB-MuSK-101
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSKantibody 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

Exclusion

Exclusion Criteria:

  • 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.

Study Design

Total Participants: 24
Treatment Group(s): 1
Primary Treatment: MuSK-CAART
Phase: 1
Study Start date:
November 23, 2022
Estimated Completion Date:
October 31, 2028

Connect with a study center

  • UC Irvine, Department of Neurology

    Orange, California 92868
    United States

    Active - Recruiting

  • UC Davis, Department of Neurology

    Sacramento, California 95817
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Active - Recruiting

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina 27599
    United States

    Site Not Available

  • Oregon Health & Science University (OHSU)

    Portland, Oregon 97239
    United States

    Active - Recruiting

  • Houston Methodist Hospital

    Houston, Texas 77030
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.