Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05451212 | Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis | PHASE1 | COMPLETED | 7 | — | — | Nov 23, 2022 | Jul 24, 2025 | Nov 10, 2025 | 5 | United States |
Incidence of adverse events (AEs), including dose-limiting toxicities (DLTs) and AEs that are related to MuSK-CAART.
| Arm | Type | Description |
|---|---|---|
| MuSK-CAART | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| MuSK-CAART | BIOLOGICAL | Intravenous infusion of MuSK-CAART at different doses. Subjects may also receive MuSK-CAART following pre-treatment with CY, or CY plus FLU. |
Inclusion Criteria: * 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 Fou...