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

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

  • MuSK Myasthenia Gravis

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/µL 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