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


About this study

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

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Martha Grogan, M.D.

Open for enrollment

Contact information:

Martha Grogan M.D.



More information


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