NCT07187401View on ClinicalTrials.gov

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

PHASE1/PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

October 13, 2025

Primary Completion Date

September 14, 2029

Study Completion Date

December 7, 2029

Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)Persistent Anemia
Interventions
DRUG

ALN-CFB

Administered as defined in the protocol

DRUG

Placebo

Administered as defined in the protocol

All Listed Sponsors
lead

Regeneron Pharmaceuticals

INDUSTRY

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 | Biotech Hunter | Biotech Hunter