Actively Recruiting
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
Led by Regeneron Pharmaceuticals · Updated on 2026-03-17
24
Participants Needed
7
Research Sites
282 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with PNH confirmed by high flow cytometry from prior testing
- Treated with a stable dose of C5 inhibitor (eculizumab, eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks before screening
- Hemoglobin level at or below 10.5 g/dL at screening with evidence of anemia prior to screening
- Peripheral blood reticulocyte count of at least 100 x 10^9/L at screening
You will not qualify if you...
- History of bone marrow transplantation or organ transplant
- History of meningococcal infection or similar recurrent infections by encapsulated bacteria
- Active ongoing infection or recent infection needing systemic treatment within 2 weeks of screening
- Laboratory evidence of bone marrow failure
- Recent unstable medical conditions not related to PNH or its complications
- Other protocol defined exclusion criteria apply
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 7 locations
1
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
Actively Recruiting
2
St. Vincent Hospital - The Catholic University of Korea
Suwon, Gyeonggi-do, South Korea, 16247
Actively Recruiting
3
Seoul National University Hospital
Seoul, South Korea, 03080
Actively Recruiting
4
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, South Korea, 06591
Actively Recruiting
5
Severance Hospital
Seoul, South Korea, 3722
Actively Recruiting
6
Samsung Medical Center
Seoul, South Korea, 6351
Actively Recruiting
7
St. James's University Hospital
Leeds, West Yorkshire, United Kingdom, LS97TF
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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