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Clinical trial

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

NCT07187401 is registered in ClinicalTrials.gov with Regeneron Pharmaceuticals as lead sponsor.

NCT NCT07187401
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Regeneron Pharmaceuticals
Phase
Phase 1/2
Overall status
Recruiting
Start date
February 11, 2026
Completion date
July 15, 2031
Enrollment
24 (estimated)
Sites
8

Conditions

As the registry lists them; a condition with its own page is linked.

paroxysmal nocturnal hemoglobinuria (pnh), persistent anemia