● Recruiting

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: Paroxysmal Nocturnal Hemoglobinuria (PNH) · Persistent Anemia · Sponsor: Regeneron Pharmaceuticals

PhasePhase 1/Phase 2

Planned participants24

Who can joinAll sexes, 18 Years to no upper limit

Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Clinical Trials Administrator · 844-734-6643 · clinicaltrials@regeneron.com

Always discuss trial participation with your own doctor first.

Locations (8)

Toronto General Hospital	Toronto, Ontario, Canada	Recruiting
St. Vincent Hospital - The Catholic University of Korea	Suwon, Gyeonggi-do, South Korea	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, United Kingdom	Recruiting
Kings College Hospital NHS Foundation Trust	London, United Kingdom	Recruiting

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Source record: clinicaltrials.gov/study/NCT07187401