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

Overview

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 within the last 24 months and known to have received active hATTR amyloidosis investigational therapy
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
Jacksonville, Fla. Mayo Clinic principal investigator Elizabeth Mauricio, M.D.	Closed for enrollment

Mayo Clinic Location

Status

Jacksonville, Fla.

Mayo Clinic principal investigator

Elizabeth Mauricio, M.D.

Closed for enrollment

More information

Publications

Publications are currently not available

Clinical Trials