Status:
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
Lead Sponsor:
Regeneron Pharmaceuticals
Conditions:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Persistent Anemia
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
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 inhibito...
Detailed Description
The protocol will be amended to describe Part B of the study after Part A data have been analyzed.
Eligibility Criteria
Inclusion
- Key
- Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing
- 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, as described in the protocol
- Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol
- Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1
- Key
Exclusion
- Has history of bone marrow transplantation or receipt of an organ transplant
- Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms
- Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
- Has laboratory evidence of bone marrow failure, as described in the protocol
- Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol
- NOTE: Other Protocol Defined Inclusion / Exclusion Criteria Apply
Key Trial Info
Start Date :
November 28 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 4 2030
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT07187401
Start Date
November 28 2025
End Date
January 4 2030
Last Update
November 6 2025
Active Locations (2)
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1
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
2
Seoul National University Hospital
Jongno-gu, Seoul, South Korea, 03080