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Clinical Trials associated with ALN-CFBA Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy
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
100 Clinical Results associated with ALN-CFB
100 Translational Medicine associated with ALN-CFB
100 Patents (Medical) associated with ALN-CFB
100 Deals associated with ALN-CFB