RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2022-11-23
Study Completion:2028-10
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05451212

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.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSK antibody test.
  2. * History of a negative anti-AChR (acetylcholine receptor) antibody test.
  3. * Positive anti-MuSK antibody test at screening
  4. * MG severity Class I to IVa on the MGFA (Myasthenia Gravis Foundation of America) Clinical Classification
  1. * Rituximab in the last 12 months.
  2. * Prednisone \> 0.25mg/kg/day \[in Part A\]
  3. * Other autoimmune disorder requiring immunosuppressive therapies.
  4. * Investigational treatment for MG in the past 12 weeks.
  5. * Absolute lymphocyte count \< 500/µL at screening.

Contacts and Locations

Study Contact

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

Principal Investigator

Medical Director
STUDY_CHAIR
Cabaletta Bio

Study Locations (Sites)

UC Irvine, Department of Neurology
Orange, California, 92868
United States
UC Davis, Department of Neurology
Sacramento, California, 95817
United States
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
Oregon Health & Science University (OHSU)
Portland, Oregon, 97239
United States
Houston Methodist Hospital
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Cabaletta Bio

  • Medical Director, STUDY_CHAIR, Cabaletta Bio

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-11-23
Study Completion Date2028-10

Study Record Updates

Study Start Date2022-11-23
Study Completion Date2028-10

Terms related to this study

Keywords Provided by Researchers

  • CAAR-T (Chimeric Autoantibody Receptor T Cells) Therapy
  • CAR-T (Chimeric Antigen Receptor T Cells) Therapy
  • Cell Therapy
  • Autoimmune Disease
  • Autoimmunity
  • Immunotherapy, Adoptive
  • Immune System Diseases
  • Myasthenia Gravis (MG)
  • Muscle-specific tyrosine kinase (MuSK)
  • Muscle Weakness
  • Neuromuscular Diseases
  • Musculoskeletal Diseases

Additional Relevant MeSH Terms

  • MuSK Myasthenia Gravis