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

Status: Recruiting
Location: See all (5) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
United States
California
UC Irvine, Department of Neurology
RECRUITING
Orange
UC Davis, Department of Neurology
RECRUITING
Sacramento
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Oregon
Oregon Health & Science University (OHSU)
RECRUITING
Portland
Texas
Houston Methodist Hospital
ACTIVE_NOT_RECRUITING
Houston
Contact Information
Primary
Cabaletta Bio
clinicaltrials@cabalettabio.com
+1 267 759 3100
Time Frame
Start Date: 2022-11-23
Estimated Completion Date: 2028-10
Participants
Target number of participants: 24
Treatments
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.
Related Therapeutic Areas
Sponsors
Leads: Cabaletta Bio

This content was sourced from clinicaltrials.gov