OncoMatch/Clinical Trials/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
Is NCT07187401 recruiting? Yes, currently enrolling (May 2026). This Phase 1/2 trial studies ALN-CFB for paroxysmal nocturnal hemoglobinuria (pnh).
Treatment: ALN-CFB — 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
Check if I qualifyExtracted eligibility criteria
Prior therapy
Must have received: C5 inhibitor (eculizumab, eculizumab biosimilar, ravulizumab, crovalimab)
Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit
Cannot have received: bone marrow transplantation
Has history of bone marrow transplantation
Cannot have received: organ transplant
receipt of an organ transplant
Lab requirements
Blood counts
hemoglobin ≤10.5 g/dl at screening visit 1, with evidence of anemia prior to this visit; peripheral blood reticulocyte count of ≥100 x 10^9/l at screening visit 1
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