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 (Jun 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
Treatments studied
Other
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
Structured fields extracted by AI. May contain errors — verify against the official protocol.
Frequently asked questions
Is NCT07187401 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior bone marrow transplantation, organ transplant disqualifies patients from enrollment.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
Check if I qualify