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
Condition(s) studied
Investigational drug(s) / intervention(s)
ALN-CFB: Administered as defined in the protocol
Placebo: Administered as defined in the protocol
Study summary
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
Eligibility
Primary outcome measure(s)
- Occurrence of Treatment-Emergent Adverse Events (TEAEs) — Through 365 Days
- Severity of TEAEs — Through 365 Days
Trial sites (8)
| Facility | City | Region | Status |
|---|---|---|---|
| Toronto General Hospital | Toronto | Ontario | Recruiting |
| St. Vincent Hospital - The Catholic University of Korea | Suwon | Gyeonggi-do | Recruiting |
| The Catholic University of Korea, Seoul St. Mary's Hospital | Seoul | South Korea | Recruiting |
| Seoul National University Hospital | Seoul | South Korea | Recruiting |
| Severance Hospital | Seoul | South Korea | Recruiting |
| Samsung Medical Center | Seoul | South Korea | Recruiting |
| St. James's University Hospital | Leeds | West Yorkshire | Recruiting |
| Kings College Hospital NHS Foundation Trust | London | United Kingdom | Recruiting |
More Regeneron Pharmaceuticals trials in South Korea
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07187401 on ClinicalTrials.gov ↗ ← All trials in South Korea