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 with hereditary transthyretin-mediated amyloidosis (hATTR).

Detailed Description

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.

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 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

Eligibility Gender
All
Eligibility Age
Minimum: 18 Years

Medical Director
Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
NCT Number
Keywords
RNAi therapeutic
FAP
Familial Amyloid Polyneuropathies
TTR
Transthyretin
Amyloidosis
MeSH Terms
Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis, Familial
Amyloidosis