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

• Status: Completed
• Trial end date: 2020-10-20
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

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 an other tetramer stabilizer during the study