Purpose of this Study
We are doing this study to find out if an experimental drug called eplontersen (the study drug) is safe and helpful for people with cardiac amyloidosis. We are enrolling people who previously participated in a clinical trial for this drug called the CardioTTRansform study (ION-682884-CS2).
Who Can Participate?
Eligibility
Adults ages 18-90 who:
- Previously participated in the CardioTTRansform study (ION-682884-CS2); AND
- Completed the study regimen in the CardioTTransform study and did not miss their final study visit
For more information about who can join the study, please contact the study team at 919-681-2723.
- Previously participated in the CardioTTRansform study (ION-682884-CS2); AND
- Completed the study regimen in the CardioTTransform study and did not miss their final study visit
For more information about who can join the study, please contact the study team at 919-681-2723.
What is Involved?
The study is divided into three parts: screening, the study drug period, and follow-up.
If you choose to join the study, we will review the results from your last visit for the CardioTTRansform study to determine if you are eligible to participate in this study.
If you are eligible, you will enter the study drug period. During this period, you will:
- Get a dose of the study drug once per month
- Visit our clinic for a checkup every 3 months
- Have blood draws, give urine samples, fill out questionnaires, do walking tests, and have ECGs done during the clinic visits
- Receive compensation for your time
The study drug period will last up to 36 months.
When you stop taking the study drug, we will schedule you to come to our clinic for 2 follow-up visits.
If you choose to join the study, we will review the results from your last visit for the CardioTTRansform study to determine if you are eligible to participate in this study.
If you are eligible, you will enter the study drug period. During this period, you will:
- Get a dose of the study drug once per month
- Visit our clinic for a checkup every 3 months
- Have blood draws, give urine samples, fill out questionnaires, do walking tests, and have ECGs done during the clinic visits
- Receive compensation for your time
The study drug period will last up to 36 months.
When you stop taking the study drug, we will schedule you to come to our clinic for 2 follow-up visits.
Study Details
Full Title
An Open-Label Extension Study to Assess the Long-Term Safety of
Eplontersen (ION-682884) in Patients with Transthyretin-Mediated
Amyloid Cardiomyopathy (ATTR-CM)
Eplontersen (ION-682884) in Patients with Transthyretin-Mediated
Amyloid Cardiomyopathy (ATTR-CM)
Principal Investigator
Michel
Khouri
Protocol Number
PRO00112244
NCT ID
NCT05667493
Phase
III
Enrollment Status
OPEN TO ACCRUAL