Official Title
Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
Brief Summary

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

Detailed Description

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More Information.

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

Drug: patisiran (ALN-TTR02)

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

Eligibility 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

- 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

- Prior or planned liver or heart transplantation

Eligibility Gender
Eligibility Age
Minimum: 18 Years ~ Maximum: N/A

Medical Director, Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
NCT Number
RNAi therapeutic
Familial Amyloid Polyneuropathies
MeSH Terms
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis, Familial