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 now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia
In brief
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and continue to have anemia (low red blood cell count). The aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug. The study is looking at several other research questions, including: * What side effects may happen from taking ALN-CFB * How much ALN-CFB is in the blood at different times * How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB
Key facts
- Study ID
- NCT07187401
- Run by
- Regeneron Pharmaceuticals
- People needed
- 24
- Starts
- 2026-02-11
- Expected to finish
- 2031-07-15
- Last updated by the study team
- 2026-07-09
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Toronto General Hospital — Toronto, Ontario, Canada (enrolling)
- St. Vincent Hospital - The Catholic University of Korea — Suwon, Gyeonggi-do, South Korea (enrolling)
- The Catholic University of Korea, Seoul St. Mary's Hospital — Seoul, South Korea (enrolling)
- Seoul National University Hospital — Seoul, South Korea (enrolling)
- Severance Hospital — Seoul, South Korea (enrolling)
- Samsung Medical Center — Seoul, South Korea (enrolling)
- St. James's University Hospital — Leeds, West Yorkshire, United Kingdom (enrolling)
- Kings College Hospital NHS Foundation Trust — London, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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