An Open-label, Multi-center, Phase I/II Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Participants With Treatment-resistant Generalized Myasthenia Gravis
Novartis Pharmaceuticals
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.
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 wi…
Eligibility
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Confirmed gMG diagnosis supported by the following: * Documented report of positive serology testing for either AChR antibodies or MuSK antibodies at screening AND at least one of the following: * History of abnormal neuromuscular transmission test demonstrated by repetitive nerve stimulation or single-fiber electromyography * History of positive acetylcholinesterase inhibitor test * Improvement in MG signs on an oral acetylcholinesterase inhibitor as assessed by the treating physician 2. MGFA Class III-IVa (gMG) at screening 3. Treatment-resistant gMG as d…
Interventions
- GeneticYTB323
CAR-T cell suspension for intravenous infusion
Locations (10)
- Univ Cali Irvine ALS NeuromuscularOrange, California
- Wake Forest Univ School of MedicineWinston-Salem, North Carolina
- Houston Methodist HospitalHouston, Texas
- Novartis Investigative SiteBordeaux
- Novartis Investigative SiteBrest
- Novartis Investigative SiteLille