The purpose of this study is to evaluate the safety and effectiveness of an experimental drug called eplontersen in people with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). ATTRCM is a disease caused by change in a protein called transthyretin (TTR). These changes can cause the TTR to clump and accumulate in certain parts of the body. The build-up is referred to as amyloid deposits, and when amyloid is deposited into the heart, it can result in a condition called cardiomyopathy. Eplontersen reduces the level of TTR in the blood of animals and healthy volunteers who have been tested. Reducing the amount of TTR in the blood may reduce the amount of amyloid deposits in the body and may prevent the patient’s cardiomyopathy from getting worse over time. Participants will receive subcutaneous (under the skin) injections of eplontersen once a month for a maximum of 36 injections over the course of 3 years and will also be required to take a Vitamin A supplement for the duration of the trial. Participants in this study will have already participated in either the ION 682884-CS2 or ISIS 420915-CS101 clinical trials.
What is the full name of this clinical trial?
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy