Treatment-resistant generalized Myasthenia Gravis
Conditions
Brief summary
Occurrence, severity, and frequency of Adverse Events (AEs) (including CRS and ICANs) and change from baseline in safety parameters including, but not limited to: Vital signs, laboratory parameters, ECG, and neurological status
Detailed description
YTB323 transgene levels by qPCR over time in peripheral blood; cellular kinetics parameters (Cmax, AUC, Tmax, Clast, Tlast), Pre-existing and treatment induced immunogenicity (cellular, humoral, neutralizing antibodies) of YTB323, At various timepoints: • Change from BL of MG-ADL score • Change from BL of QMG total score • Whether or not patient achieves a ≥3-point reduction of QMG total score sustained for 6 months post BL • Whether or not patient achieves a ≥2-point reduction of MG-ADL score sustained for 6 months post BL • Whether or not a patient achieves MGFA Post intervention Status (PIS) of minimal manifestations (MM) or better and sustained for 6 months post BL
Interventions
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence, severity, and frequency of Adverse Events (AEs) (including CRS and ICANs) and change from baseline in safety parameters including, but not limited to: Vital signs, laboratory parameters, ECG, and neurological status | — |
Secondary
| Measure | Time frame |
|---|---|
| YTB323 transgene levels by qPCR over time in peripheral blood; cellular kinetics parameters (Cmax, AUC, Tmax, Clast, Tlast), Pre-existing and treatment induced immunogenicity (cellular, humoral, neutralizing antibodies) of YTB323, At various timepoints: • Change from BL of MG-ADL score • Change from BL of QMG total score • Whether or not patient achieves a ≥3-point reduction of QMG total score sustained for 6 months post BL • Whether or not patient achieves a ≥2-point reduction of MG-ADL score sustained for 6 months post BL • Whether or not a patient achieves MGFA Post intervention Status (PIS) of minimal manifestations (MM) or better and sustained for 6 months post BL | — |
Countries
France
Outcome results
None listed