Condition: Transthyretin amyloidosis with cardiomyopathy
Key Inclusion Criteria:
- 18-85 years old
- Documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy
- Medical history of heart failure
- Clinically stable, with no cardiovascular-related hospitalizations within 6 weeks prior to research group assignment
Key Exclusion Criteria:
- Has known primary amyloidosis or leptomeningeal amyloidosis
- New York Heart Association Class III AND ATTR amyloidosis disease Stage 3
- New York Heart Association Class IV at the screening visit
APOLLO-B: A Phase III, Randomized, Double-Blind, Placebo-Controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) (IRB no. 0000303)
The purpose of this study is to evaluate the safety and investigational use of a drug called Patisiran and whether it may improve symptoms and quality of life in patients with transthyretin (ATTR) amyloidosis with cardiomyopathy. The study focuses on individuals with this condition who are not taking tafamidis or whose condition has deteriorated while taking tafamidis as part their standard-of-care-treatment.
ATTR amyloidosis is caused by certain differences in the protein called transthyretin (TTR) and results in collections of proteins called amyloid fibrils, which can affect organ function. Patisiran (the study drug) blocks the ability of ribonucleic acid to produce TTR, thereby lowering TTR levels in the blood and reducing the amount of amyloid fibrils in the organs.
Participants will be randomly assigned to receive either patisiran or a placebo (inactive substance). Researchers will compare the effects of patisiran to the effects of placebo to determine which is more effective.