FIH Study of ALN-CFB in patients with PNH and persistent anemia on SOC
Research type
Research Study
Full title
A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, FIRST IN HUMAN STUDY OF THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY EFFICACY OF ALN-CFB, AN INVESTIGATIONAL SIRNA THERAPEUTIC AGAINST COMPLEMENT FACTOR B, IN PARTICIPANTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND PERSISTENT ANEMIA ON APPROVED C5-INHIBITOR THERAPY
IRAS ID
1012023
Contact name
Medical Affairs NAP
Contact email
Sponsor organisation
Regnenron Pharmaceuticals Inc
Research summary
This study aims to evaluate the safety, and how well tolerated ALN-CFB is, a new investigational drug, in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have persistent anaemia despite being on inhibitor therapy. This study will be first time ALN-CFB is given to humans.
PNH is a rare blood disorder that causes red blood cells to break down, leading to anaemia and other complications. While existing treatments help many patients, some still experience ongoing anaemia. This study is important because it explores a potential new treatment option for these patients.
The study will test ALN-CFB, which targets a protein called complement factor B, in combination with patients' current PNH medication.
This is a Phase 1/2 clinical trial that will take place at qualified stud sites. The study has two parts:
• Part A: Participants receive either a single dose of ALN-CFB or a dose of ALN-CFB or placebo and after 30 days a dose of placebo or ALN-CFB, with follow-up for about 57 weeks.
• Part B: Details to be determined based on Part A results.
In Part A, participants will:
• Undergo screening tests
• Have regular follow-up visits for blood tests and monitoring
• Continue their current PNH medication throughout the study
Benefits are not guaranteed. The study will provide important information about ALN-CFB's safety and effectiveness, which could lead to new treatment options for PNH patients in the future. Risks are unknown but based on medications similar to ALN-CFB, possible risks include infections, changes in liver function, allergic reactions.
This research is crucial for developing better treatments for PNH patients who continue to struggle with anaemia despite current therapies.REC name
South Central - Berkshire Research Ethics Committee
REC reference
25/SC/0203
Date of REC Opinion
1 Aug 2025
REC opinion
Further Information Favourable Opinion