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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05070858 | A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis | PHASE3 | ACTIVE NOT_RECRUITING | 288 | — | — | Dec 14, 2021 | Nov 11, 2028 | Feb 12, 2026 | 116 | United States, Belgium +17 |
The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability
| Arm | Type | Description |
|---|---|---|
| Group 1 | EXPERIMENTAL | Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP |
| Group 2 | EXPERIMENTAL | Combination regimen throughout the study. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP. |
| Group 3 | EXPERIMENTAL | Cemdisiran throughout the study |
| Group 4 | EXPERIMENTAL | Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP. |
| Name | Type | Description |
|---|---|---|
| Pozelimab + Cemdisiran | DRUG | Subcutaneous administration as described in the protocol |
| Cemdisiran | DRUG | SC administration as described in the protocol |
| Placebo | OTHER | SC administration as described in the protocol |
| Pozelimab | DRUG | SC administration as described in the protocol |
Key Inclusion Criteria: 1. Male or female participants ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older) 2. Participant with documented diagnosis of Myasthenia Gravis (MG) based on medical history and supported by previous evaluations as desc...