APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Purpose
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
Condition
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Eligibility
- Eligible Ages
- Between 18 Years and 85 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with cardiomyopathy - Medical history of heart failure with at least 1 prior hospitalization for heart failure, or current clinical evidence (signs and symptoms of heart failure) - Clinically stable with no cardiovascular related hospitalizations within 6 weeks of study start - Has never taken tafamidis before (tafamidis naïve) or currently on tafamidis for ≥6 months with evidence of disease progression while on tafamidis treatment - Able to complete ≥150 m on the 6-minute walk test - Screening N-terminal pro B-type natriuretic peptide (NT-proBNP), a blood marker of heart failure severity, >300 ng/L and <8500 ng/L; in participants with permanent or persistent atrial fibrillation, screening NT-proBNP> 600 ng/L and <8500 ng/L
Exclusion Criteria
- Known primary amyloidosis (AL) or leptomeningeal amyloidosis. - Received prior TTR lowering treatment - New York Heart Association heart failure classification of III and at high risk - New York Heart Association heart failure classification of IV - Neuropathy requiring cane or stick to walk, or is wheelchair bound - Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2 - Abnormal liver function - Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection - Has non-amyloid disease that significantly affects ability to walk (e.g., severe chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular disease affecting ambulation) - Prior or planned heart, liver, or other organ transplant - Other cardiomyopathy not related to ATTR amyloidosis
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Patisiran |
Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period. |
|
Placebo Comparator Placebo |
Participants will be administered multiple doses of placebo in the double-blind period. In the open-label extension period, participants will be administered multiple doses of patisiran. |
|
More Details
- Status
- Active, not recruiting
- Sponsor
- Alnylam Pharmaceuticals