Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Read our disclaimer for details.
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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
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
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