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

N/AApproved For MarketingNCT02939820

Alnylam Pharmaceuticals

Overview

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

Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.

Study Type

EXPANDED_ACCESS

Conditions

TTR-mediated Amyloidosis Amyloidosis, Hereditary Amyloid Neuropathies, Familial Familial Amyloid Polyneuropathies Amyloid Neuropathies Amyloidosis, Hereditary, Transthyretin-Related

Interventions

patisiran (ALN-TTR02)DRUG

patisiran (ALN-TTR02) administered by intravenous (IV) infusion

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Data from ClinicalTrials.gov

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