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A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of ALN-CFB, an Investigational siRNA Drug Targeting Complement Factor B, in Adults With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on C5 Inhibitor Therapy
Led by Regeneron Pharmaceuticals · Updated on 2026-03-17
24
Participants Needed
7
Research Sites
84 weeks
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating an experimental drug called ALN-CFB in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who continue to have anemia despite treatment with a complement component C5 inhibitor. This Phase 1 and 2 study aims to assess the safety and tolerability of ALN-CFB compared to a placebo, while also examining how the drug affects levels of Complement Factor B (CFB) protein in the blood and its concentration over time. Participants will be randomly assigned to receive either ALN-CFB or a placebo following a single-ascending dose escalation design. The study includes a double-blind period where neither participants nor researchers know who receives the drug or placebo. The protocol will be updated to describe Part B after analysis of Part A data. During the study, participants will be monitored for treatment-emergent adverse events and their severity over one year. Researchers will measure ALN-CFB and its metabolites in plasma and urine, along with changes in CFB concentration from baseline. The study involves regular visits for assessments and safety monitoring, lasting up to 365 days.
CONDITIONS
Brief 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 Paroxysmal Nocturnal Hemoglobinuria confirmed by a history of high flow cytometry
- Treated with a stable dose of C5 inhibitor (eculizumab, approved biosimilar, ravulizumab, or crovalimab) for at least 24 weeks before screening
- Hemoglobin level of 10.5 g/dL or lower at screening visit 1 with evidence of anemia prior to this visit
- Peripheral blood reticulocyte count of at least 100 x 10^9/L at screening visit 1
You will not qualify if you...
- History of bone marrow transplantation or organ transplant
- History of meningococcal infection or recurrent infections by other encapsulated bacteria
- Any active infection or recent infection requiring systemic antibiotics, antivirals, or antifungals within 2 weeks of screening or during screening
- Laboratory evidence of bone marrow failure
- Recent unstable medical conditions not related to PNH or its complications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 24 weeks prior to screening
Participants continue stable C5 inhibitor treatment to establish baseline before receiving the investigational drug or placebo.
Visit schedule as per routine care; not part of trial visits
Duration - Up to 365 days
Participants receive either the investigational siRNA drug ALN-CFB or placebo as defined in the protocol to evaluate safety and efficacy.
Multiple visits during treatment as scheduled by the trial protocol
Duration - Up to 365 days post-treatment
Participants are monitored for treatment-emergent adverse events and changes in blood and urine concentrations of the drug and metabolites after dosing.
Visits for safety and laboratory assessments as scheduled
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
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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