Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Male or female greater than or equal to 18 years of age

• Have a diagnosis of hATTR

• Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements

• Have adequate complete blood counts, liver function tests and coagulation tests

Exclusion Criteria:

• Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months

• Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial

• Have inadequate cardiac function

• Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis

• Have known serious comorbidities or considered unfit for the program by the investigator

• Prior or planned liver or heart transplantation