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
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
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria:
1. Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing
2. 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
3. Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol
4. Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1
Key Exclusion Criteria:
1. Has history of bone marrow transplantation or receipt of an organ transplant
2. Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms
3. 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
4. Has laboratory evidence of bone marrow failure, as described in the protocol
5. 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
Primary outcome measure(s)
Occurrence of Treatment-Emergent Adverse Events (TEAEs) — Through 365 Days
Severity of TEAEs — Through 365 Days
Trial sites (8)
Facility
City
Region
Status
Toronto General Hospital
Toronto
Ontario
Recruiting
St. Vincent Hospital - The Catholic University of Korea
Suwon
Gyeonggi-do
Recruiting
The Catholic University of Korea, Seoul St. Mary's Hospital
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.