CS2 - Phase 3 study of ION-682884 in Patients with ATTR CM
Research type
Research Study
Full title
A Phase 3 Global, Double-Blind, Randomized, Placebo‑Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR CM)
IRAS ID
274815
Contact name
Marianna Fontana
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2019-002835-27
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 5 months, 30 days
Research summary
This is a randomised, double-blind, placebo-controlled study of ION-682884 vs. placebo administered by subcutaneous injection once every 4 weeks for 120 weeks in participants with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) receiving available background standard of care.
Transthyretin-mediated amyloid cardiomyopathy is a disease caused by change in a protein called transthyretin (TTR). This change can either be sporadic which is known as wild-type (wtATTR-CM) or due to a mutation of the TTR gene which is hereditary (hATTR-CM). In both cases, these changes can cause the TTR to clump and build up in certain parts of body such as 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.
ION-682884 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. However, it is not known if ION-682884 will help participants cardiomyopathy. The purpose of this study is to evaluate the safety of ION-682884 and determine if it can help people with cardiomyopathy.Approximately 750 participants between 18 and 90 years of age, will be randomised 1:1 to either ION-682884 or placebo from multiple sites worldwide. This is a double-blind study, which means that neither participant nor the study staff will know whether participants are taking ION-682884 or placebo.
The length of participation is approximately 146 weeks: up to 6 weeks of Screening Period (10 weeks if biopsy and/or genetic testing is needed), 120 weeks of Treatment Period, and 20 weeks for Post-Treatment Evaluation Period.
There will be 63 study visits to complete medical exams, dosing and tests. Of these 63 visits, 18 must be completed at study site and 45 can be conducted outside of the clinic.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
20/SC/0028
Date of REC Opinion
3 Apr 2020
REC opinion
Further Information Favourable Opinion