A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
Recruiting · NCT07187401 · Registry updated 2026-07-09Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor | Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome | Recruiting | Phase 1 / Phase 2 | 2026-07-09 | NCT07187401 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Regeneron Pharmaceuticals | 238 |
8 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Kings College Hospital NHS Foundation Trust | London | United Kingdom | 6 |
| Samsung Medical Center | Seoul | South Korea | 30 |
| Seoul National University Hospital | Seoul | South Korea | 40 |
| Severance Hospital | Seoul | South Korea | 5 |
| St. James's University Hospital | Leeds | United Kingdom | 1 |
| St. Vincent Hospital - The Catholic University of Korea | Suwon | South Korea | 3 |
| The Catholic University of Korea, Seoul St. Mary's Hospital | Seoul | South Korea | 47 |
| Toronto General Hospital | Toronto | Canada | 42 |