Purpose

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.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion 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

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/┬ÁL at screening.

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
MuSK-CAART
Cohort A: Infusion of MuSK-CAART at various dose levels with or without pre-treatment (6 groups planned). Cohort B: Infusion of MuSK-CAART at the dose regimen selected from Part A.
  • Biological: MuSK-CAART
    Intravenous infusion of MuSK-CAART at different doses. Subjects may also receive MuSK-CAART following pre-treatment with CY, or CY plus FLU.

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66160
Contact:
Andrew J Heim
913-945-9926
aheim2@kumc.edu

More Details

Status
Recruiting
Sponsor
Cabaletta Bio

Study Contact

Cabaletta Bio
+1 267 759 3100
clinicaltrials@cabalettabio.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.