Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Amyloidosis, Hereditary, Transthyretin-Related, Familial Amyloid Polyneuropathies, TTR-mediated Amyloidosis
Conditions
Keywords
RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis
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.
Interventions
patisiran (ALN-TTR02) administered by intravenous (IV) infusion
Sponsors
Alnylam Pharmaceuticals
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Outcome results
None listed