Purpose of this Study
We are doing this study to find out if an experimental drug called ALXN2220 (the study drug) is a safe and effective option for people with transthyretin amyloid cardiomyopathy (ATTR-CM). The study drug is designed to act as a "depleter," which means its desired function is to target the active removal of pre-existing amyloids and restore the heart's function and elasticity.
Who Can Participate?
Eligibility
Adults ages 18-90 who:
- Are diagnosed with wild-type ATTR-CM or variant ATTR-CM
- Are willing to undergo genetic testing for mutations to the TTR gene
- Have never received any previous treatment with an ATTR amyloid depleter
Age Range
18-90
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
If you choose to join this study, you will get a random assignment (like a coin flip) to either get the study drug or a placebo (inactive substance with no drug in it). Regardless of assignment, everyone in the study will:
- Visit our clinic about 56 times over the course of 4 years
- Get an intravenous (IV) infusion of the study drug or placebo once every 4 weeks
- Fill out questionnaires
- Have physical exams and various fitness tests done throughout the study to assess heart health and function
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
Yes
Study Details
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)
Principal Investigator
Michel
Khouri
Protocol Number
PRO00114661
Phase
III
Enrollment Status
Open to Enrollment