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

Alnylam Pharmaceuticals

Status

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

Treatments: Drug: patisiran (ALN-TTR02)

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT02939820

ALN-TTR02-007

Details and patient eligibility

About

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

Full 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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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