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

ALN-TTR02-008

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.

2019-03-27

2020-10-06

2020-10-20

Completed

Early phase I

24

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 24, 'type': 'ACTUAL'}}

2024-04-22