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

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Transthyretin Amyloidosis; Amyloidosis, Familial

Treatments: Drug: Patisiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT03862807

ALN-TTR02-008

2018-003519-24 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 24 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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24 participants in 1 patient group

Patisiran

Experimental group

Description:

Participants received patisiran 0.3 milligrams/kilogram (mg/kg) via intravenous (IV) infusion once every 3 weeks (q3w) for 12 months. Dosing was based on actual body weight. For participants weighing 100 kg or more, patisiran was administered at a total dose of 30 mg IV q3w.

Treatment:

Drug: Patisiran