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
NCT ID: NCT07187401
Last Updated: 2025-11-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
24 participants
INTERVENTIONAL
2025-11-28
2030-01-04
Brief Summary
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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
Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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Single-Ascending Dose Escalation
ALN-CFB
Administered as defined in the protocol
Placebo
Placebo
Administered as defined in the protocol
Interventions
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ALN-CFB
Administered as defined in the protocol
Placebo
Administered as defined in the protocol
Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
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
18 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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Toronto General Hospital
Toronto, Ontario, Canada
Seoul National University Hospital
Jongno-gu, Seoul, South Korea
Countries
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Central Contacts
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Facility Contacts
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Christopher Patriquin
Role: primary
Other Identifiers
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2024-519806-11-00
Identifier Type: CTIS
Identifier Source: secondary_id
ALN-CFB-PNH-2468
Identifier Type: -
Identifier Source: org_study_id