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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07154745 | A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently | PHASE3 | RECRUITING | 35 | — | — | May 30, 2026 | Nov 22, 2031 | May 26, 2026 | 13 | Brazil, Italy +4 |
| NCT05744921 | A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works | PHASE3 | RECRUITING | 202 | — | — | Mar 7, 2023 | Feb 26, 2029 | Mar 18, 2026 | 43 | Canada, Colombia +17 |
| NCT04811716 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | PHASE2 | COMPLETED | 24 | — | — | Jul 29, 2021 | Oct 18, 2023 | Apr 8, 2025 | 13 | Hong Kong, Hungary +4 |
| NCT04888507 | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | PHASE2 | COMPLETED | 6 | — | — | Jul 8, 2021 | May 4, 2023 | Aug 29, 2025 | 1 | United Kingdom |
An SAE is any untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect. * Is an important medical event
An AESI (serious or non-serious) is one of scientific and medical concern specific to the sponsor's product or program, for which ongoing monitoring and rapid communication by the Investigator to the sponsor can be appropriate. Such an event might warrant further investigation in order to characterize and understand it
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug.
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug.
Open Label Treatment Period (OLTP)
| Arm | Type | Description |
|---|---|---|
| Pozelimab + Cemdisiran Combo | EXPERIMENTAL | - |
| PNH Transition Patients | EXPERIMENTAL | Patients with PNH who completed treatment/ protocol requirements (as applicable) in the parent study (R3918-PNH-2021 \[NCT05133531\]) |
| C5 Polymorphism Patients | EXPERIMENTAL | Patients who have not been treated in either parent study but who have a documented complement component 5 (C5) variation rendering them refractory to eculizumab/ravulizumab. Note: Loading dose of pozelimab administered IV on Day 1. |
| Pozelimab Q4W + Cemdisiran | EXPERIMENTAL | - |
| Pozelimab Q2W + Cemdisiran | EXPERIMENTAL | - |
| Pozelimab+Cemdisiran | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Pozelimab | DRUG | Administered per the protocol |
| Cemdisiran | DRUG | Administered per the protocol |
Key Inclusion Criteria: 1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing 2. Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months 3. LDH persistently \> 1.5 × Upper Limit of Normal (ULN) in th...