Alnylam Pharmaceuticals

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

Official Title: 
Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
Brief Summary: 
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
Detailed Description: 

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.

Approved for marketing
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related

Drug: patisiran (ALN-TTR02)
patisiran (ALN-TTR02) administered by intravenous (IV) infusion

Eligibility Criteria: 

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

Eligibility Gender: 
All
Eligibility Age: 
Minimum: 18 Years
Countries: 
United States
Locations: 

Kaiser Permanente
Oakland, California,

Stanford University
Stanford, California,

MedStar Washington Hospital
Washington, District of Columbia,

Mayo Clinic - Jacksonville
Jacksonville, Florida,

Northwestern University
Evanston, Illinois,

University of Iowa
Iowa City, Iowa,

University of Kansas Medical Center
Kansas City, Kansas,

Ochsner Medical Center
New Orleans, Louisiana,

Johns Hopkins Medical Center
Baltimore, Maryland,

Boston University Medical Center
Boston, Massachusetts,

Barbara Ann Karmanos Cancer Institute
Detroit, Michigan,

Mayo Clinic - Rochester
Rochester, Minnesota,

Washington University School of Medicine
Saint Louis, Missouri,

Columbia University Medical Center
New York, New York,

Ohio State University
Columbus, Ohio,

Oregon Health & Science University
Portland, Oregon,

Penn Presbyterian Medical Center
Philadelphia, Pennsylvania,

University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania,

Vanderbilt University Medical Center
Nashville, Tennessee,

Seton Family of Hospitals
Austin, Texas,

Baylor
Dallas, Texas,

Jared Gollob, MD
Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
EA Number: 
Keywords: 
RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis
MeSH Terms: 
Polyneuropathies, Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, Familial, Amyloidosis
Disclaimer:
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.