NCT07187401 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 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Regeneron Pharmaceuticals |
| Condition | Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2026-02-11 |
| Primary Completion | 2029-11-29 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 24 participants in total. It began in 2026-02-11 with a primary completion date of 2029-11-29.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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 Criteria
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
Contact & Investigator
Clinical Trial Management
STUDY DIRECTOR
Regeneron Pharmaceuticals
Frequently Asked Questions
Who can join the NCT07187401 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Paroxysmal Nocturnal Hemoglobinuria (PNH). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07187401 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07187401 currently recruiting?
Yes, NCT07187401 is actively recruiting participants. Contact the research team at clinicaltrials@regeneron.com for enrollment information.
Where is the NCT07187401 trial being conducted?
This trial is being conducted at Toronto, Canada, Suwon, South Korea, Seoul, South Korea, Seoul, South Korea and 4 additional locations.
Who is sponsoring the NCT07187401 clinical trial?
NCT07187401 is sponsored by Regeneron Pharmaceuticals. The principal investigator is Clinical Trial Management at Regeneron Pharmaceuticals. The trial plans to enroll 24 participants.