Trial Profile

A 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

Status: Not yet recruiting

Phase of Trial: Phase I/II

Latest Information Update: 10 Nov 2025

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At a glance

06 Nov 2025 Planned End Date changed from 7 Dec 2029 to 4 Jan 2030.
06 Nov 2025 Planned primary completion date changed from 14 Sep 2029 to 11 Oct 2029.
06 Nov 2025 Planned initiation date changed from 13 Oct 2025 to 28 Nov 2025.

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