Inclusion criteria | Exclusion criteria |
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• Diagnosis of hATTR amyloidosis with documented mutation • Anticipated survival ≥2 years • Aged 18–85 years (inclusive) • NIS of 5–130 • PND score ≤IIIb • NCS sum of the sural SNAP, tibial CMAP, ulnar SNAP, ulnar CMAP, and peroneal CMAP of ≥2 points • Karnofsky performance status ≥60% • Absolute neutrophil count ≥1500 cells/mm3 • Platelet count ≥50,000 cells/mm3 • Adequate biochemical liver functiona • Serum creatinine ≤2 × ULN | • Previous liver transplantation, or liver transplantation planned during study period • Sensorimotor or autonomic neuropathy not related to hATTR amyloidosis • Primary or leptomeningeal amyloidosis • Type 1 diabetes • Type 2 diabetes for ≥5 years • Active hepatitis B or C, or HIV infection • NYHA heart failure classification >2 • Acute coronary syndrome within past 3 months • Uncontrolled cardiac arrhythmia or unstable angina • Severe reaction to a liposomal product or hypersensitivity to oligonucleotides • Unable to take premedications • Received an investigational agent within 30 days/5 half-lives (whichever is longer) of study drug administration • Currently taking tafamidis, diflunisal, doxycycline, or tauroursodeoxycholic acid |