ION682884-CS12
Research type
Research Study
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)
IRAS ID
1007819
Contact name
Li Zhou
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
This is a multicentre, open-label extension of the ION-682884-CS2 and of the ISIS 420915-CS101 studies in participants with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM). Upon completion of the treatment phase of these two studies participants will be given the option to continue to receive a study medication called eplontersen in the ION-682884-CS12 study. Eplontersen will be administered by subcutaneous injection once every 4 weeks for 36 months or 6 months after eplontersen is health authority approved and available in the UK, whichever occurs first.
Transthyretin-mediated amyloid cardiomyopathy is a disease caused by changes in a protein called transthyretin (TTR). This change can cause the TTR to clump and build up in certain parts of body such as the nervous system, stomach, intestines, and heart. This build up is called an amyloid deposit. Amyloid deposits can sometimes cause heart disease or neuropathy, a nerve disorder. When amyloid is deposited into the heart, it can result in a condition referred to as cardiomyopathy.
Eplontersen reduces the level of TTR in the blood of animals and healthy volunteers tested to date. Reducing the
amount of TTR in participants blood may reduce the amount of amyloid deposits in participants body and may keep
participants cardiomyopathy from getting worse over time. The purpose of this study is to evaluate the safety and tolerability of eplontersen and determine if it can help people with cardiomyopathy.
Approximately 1400 participants from the N-682884-CS2 and ISIS 420915-CS101 may be eligible for this study from multiple sites worldwide. This is a an open label study, which means that both the participant and the study staff will know what medication the participants are receiving.
The length of participation is approximately 6 months: up to 36 weeks of Treatment Period, and up to 3 months for Post-Treatment Evaluation Period.
There will be 18 study visits to complete medical exams, dosing and tests.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
23/EM/0178
Date of REC Opinion
30 Aug 2023
REC opinion
Further Information Favourable Opinion