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ALN-CFB

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

Ensayo de REGENERON PHARMACEUTICALS, INC. en Paroxysmal Nocturnal Hemoglobinuria (PNH) · Persistent Anemia (del registro, en inglés).

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

24

previstos

Centros

8

Fin del objetivo primario

nov 2029

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en feb 2026.

Qué mide

Occurrence of Treatment-Emergent Adverse Events (TEAEs) (Through 365 Days) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis 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

Más ensayos de REGN

Todos los de REGN, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT07187401), actualizada en jul 2026.