Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR) (NCT02939820) | Clinical Trial Compass
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Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Plain-language summary
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
Who can participate
Age range18 Years
SexALL
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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