Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.

Inclusion Criteria:

• Received liver transplant for treatment of hATTR amyloidosis ≥12 months before study start
• Has increase in polyneuropathy disability (PND) score after liver transplant
• Has received stable immunosuppressive regimen with ≤10 mg/day of prednisone for at least 3 months before study start
• Has Karnofsky Performance Status (KPS) of ≥70%
• Has vitamin A level greater than or equal to lower limit of normal

Exclusion Criteria:

• Has previously received inotersen or patisiran
• Has clinically significant liver function test abnormalities
• Has known portal hypertension with ascites
• Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73 m^2
• Has known leptomeningeal amyloidosis
• Has infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV)
• Has New York Heart Association heart failure classification of >2
• Is wheelchair bound or bedridden
• Has received organ transplants other than liver transplant
• Will be using another tetramer stabilizer during the study