NCT07187401

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

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: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

The protocol will be amended to describe Part B of the study after Part A data have been analyzed.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Persistent Anemia

Interventions

Type	Name	Description
DRUG	ALN-CFB	Administered as defined in the protocol
DRUG	Placebo	Administered as defined in the protocol

Timeline

• Start date: 2026-02-11
• Primary completion: 2029-11-29
• Completion: 2031-07-15
• First posted: 2025-09-23
• Last updated: 2026-03-17

Locations

7 sites across 3 countries: Canada, South Korea, United Kingdom

Regulatory

• FDA-regulated drug study