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Clinical Trials in the UK / NCT07187401
Recruiting Phase 1/2

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

NCT07187401 · tracked via the Priya Life Science UK tracker
Sponsor
Regeneron Pharmaceuticals
Phase
Phase 1/2
Started
2026-02-11
Last updated
2026-07-09

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)Persistent Anemia

Investigational drug(s) / intervention(s)

ALN-CFBPlacebo

ALN-CFB: Administered as defined in the protocol

Placebo: Administered as defined in the protocol

Study 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

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)

Trial sites (8)

FacilityCityRegionStatus
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 Seoul South Korea Recruiting
Seoul National University Hospital Seoul South Korea Recruiting
Severance Hospital Seoul South Korea Recruiting
Samsung Medical Center Seoul South Korea Recruiting
St. James's University Hospital Leeds West Yorkshire Recruiting
Kings College Hospital NHS Foundation Trust London United Kingdom Recruiting

More Regeneron Pharmaceuticals trials in the UK

Other trials for the same condition

Official registry record

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.

View NCT07187401 on ClinicalTrials.gov ↗ ← All trials in the UK