Study of ALXN2220 versus Placebo in Adults with ATTR-CM
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
IRAS ID
1009301
Contact name
Cristina Quarta
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2023-506669-70
Research summary
Amyloidosis is a rare, progressive, fatal disease caused by the deposition of misfolded "amyloid” protein in tissues and organs. This is a faulty form of transthyretin (TTR), which is normally used for transporting thyroid hormone and vitamin A in the blood. ATTR-CM is a type of amyloidosis where misfolded TTR is predominantly deposited in the heart, causing damage to the heart muscle. ATTR-CM is an increasingly recognized cause of heart failure with poor prognosis and limited therapeutic options.
Current treatment approaches focus on eliminating or stabilising the production of the amyloid protein. There are no approved therapies that remove deposited amyloid protein from organs, which can continue to cause worsening heart failure despite treatment with TTR stabilizers or gene silencers.
The purpose of this study is to evaluate the efficacy and safety of ALXN2220 in adult participants with ATTR-CM. ALXN2220 is a human anti-ATTR monoclonal antibody. ALXN2220 selectively binds misfolded, deposited ATTR, with no binding to the normal TTR transport protein. This leads to immune-mediated clearance of ATTR deposits from tissues/organs. By depleting TTR amyloid deposits in the heart, ALXN2220 is expected to halt or reverse disease progression in ATTR-CM. The primary purpose of this study is to measure if ALXN2220 improves mortality and cardiovascular morbidity in participants with ATTR-CM.
Prior to enrolment, participants will be assessed to ensure they are safe to dose. Approximately 1000 participants are expected to be enrolled. Participants will have a 2:1 chance to receive ALXN2220 or placebo every 4 weeks via an intravenous (IV) drip. In this double-blind study, neither the study doctor nor participants know which treatment they receive and the treatments will be randomly assigned. The total duration of the study from the first day of screening to the last visit is approximately 2.5 years for the last participant to enter the study.
REC name
London - London Bridge Research Ethics Committee
REC reference
24/LO/0013
Date of REC Opinion
19 Feb 2024
REC opinion
Further Information Favourable Opinion