Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Overview
Tab Title Description
Study type
InterventionalDescribes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
Study IDs
Site IRB
- Jacksonville, Florida: 17-000983
NCT ID: NCT02939820
Sponsor Protocol Number: ALN-TTR02-007
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 |
|
More information
Publications
Publications are currently not available