Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis
Start Date
4/22/2025
Completion Date
10/26/2029
Summary
This is a phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis. YTB323 is a Biological CAR-T cell therapy.
Detailed Description
This is an open-label, multi-center, non-confirmatory study intended to assess safety, efficacy, and cellular kinetics of YTB323 treatment in participants with treatment-resistant generalized myasthenia gravis in order to enable a benefit to risk assessment for further development in generalized myasthenia gravis (gMG). The study plans to enroll approximately 15 participants with treatment-resistant gMG. The study utilizes a single dose design across 2 cohorts, consisting of a sentinel cohort of 3 patients followed by an expansion cohort of an additional 12 patients. All participants dosed with YTB323 will be followed until 15 years after YTB323 administration in the Long-Term Follow-up (LTFU).
Eligibility Criteria
Age Range: 18 years to 65 years
Interventions
YTB323
Conditions
Locations
Univ Cali Irvine ALS Neuromuscular
Orange, California 92868
United States
Wake Forest Univ School of Medicine
Winston-Salem, North Carolina 27157-1052
United States
Thomas Jefferson University
Philadelphia, Pennsylvania 19107
United States
Houston Methodist Hospital
Houston, Texas 77030
United States
Novartis Investigative Site
Bordeaux, 33076
France
Novartis Investigative Site
Brest, 29200
France
Novartis Investigative Site
Lille, 59037
France
Novartis Investigative Site
Chiba, Chiba 2608677
Japan
Novartis Investigative Site
Kyoto, 6068507
Japan
Novartis Investigative Site
Sheffield, South Yorkshire S10 2JF
United Kingdom
Novartis Investigative Site
London, SE5 9RS
United Kingdom