Status:

RECRUITING

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

Lead Sponsor:

Regeneron Pharmaceuticals

Conditions:

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Persistent Anemia

Eligibility:

All Genders

18+ years

Phase:

PHASE1

PHASE2

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 inhibito...

Detailed Description

The protocol will be amended to describe Part B of the study after Part A data have been analyzed.

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

Key Trial Info

Start Date :

February 11 2026

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

July 15 2031

Estimated Enrollment :

24 Patients enrolled

Trial Details

Trial ID

NCT07187401

Start Date

February 11 2026

End Date

July 15 2031

Last Update

March 17 2026

Active Locations (7)

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Page 1 of 2 (7 locations)

1

Toronto General Hospital

Toronto, Ontario, Canada, M5G 2C4

2

St. Vincent Hospital - The Catholic University of Korea

Suwon, Gyeonggi-do, South Korea, 16247

3

Seoul National University Hospital

Seoul, South Korea, 03080

4

The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, South Korea, 06591