ALN-CFB for adults with PNH who remain anemic on C5 inhibitors

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This first-in-human phase 1/2 trial gives adults with PNH and persistent anemia either ALN-CFB or placebo while they remain on a stable C5 inhibitor. The study primarily measures safety and tolerability and also tracks drug levels, changes in Complement Factor B protein, and effects on red blood cell counts and hemolysis markers. Results from Part A will guide amendments and dosing for Part B. Participants will be closely monitored for adverse events, including infection risk related to complement modulation.

Why it matters

Who can join: the full rules

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

Where it's running

• Toronto General Hospital
  Toronto, Ontario, Canada Recruiting
• St. Vincent Hospital - The Catholic University of Korea
  Suwon, Gyeonggi-do, South Korea 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

Study contacts

• Study coordinator: Clinical Trials Administrator
• Email: clinicaltrials@regeneron.com
• Phone: 844-734-6643

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.