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



Status:Approved for marketing
Conditions:Neurology, Hematology
Therapuetic Areas:Hematology, Neurology
Healthy:No
Age Range:18 - Any
Updated:8/16/2018

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Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy

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

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.

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
We found this trial at
21
sites
101 Jessup Hall
Iowa City, Iowa 52242
(319) 335-3500
University of Iowa With just over 30,000 students, the University of Iowa is one of...
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Baltimore, Maryland 21231
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Baltimore, MD
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4100 John R
Detroit, Michigan 48201
800-527-6266
Barbara Ann Karmanos Cancer Institute Karmanos is based in southeast Michigan, in midtown Detroit, and...
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Detroit, MI
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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Nashville, TN
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Portland, OR
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Austin, Texas 78701
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Austin, TX
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1 Boston Medical Center Place
Boston, Massachusetts 02118
617.638.8000
Boston University Medical Center Boston Medical Center is an extraordinary community of health care providers...
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Boston, MA
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281 W. Lane Ave
Columbus, Ohio 43210
(614) 292-6446
Ohio State University The Ohio State University’s main Columbus campus is one of America’s largest...
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Columbus, OH
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Dallas, Texas 75246
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Dallas, TX
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Evanston, Illinois 60208
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Evanston, IL
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Jacksonville, Florida 32216
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Jacksonville, FL
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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Kansas City, KS
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1514 Jefferson Hwy.
New Orleans, Louisiana 70121
504-842-3000
Ochsner Medical Center Ochsner Medical Center is located near uptown New Orleans and includes acute...
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New Orleans, LA
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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New York, NY
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Oakland, California 94612
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Oakland, CA
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Philadelphia, Pennsylvania
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Philadelphia, PA
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200 Lothrop St
Pittsburgh, Pennsylvania 15213
University of Pittsburgh Medical Center UPMC is one of the leading nonprofit health systems in...
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Pittsburgh, PA
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200 First Street SW
Rochester, Minnesota 55905
507-284-2511
Mayo Clinic Rochester Mayo Clinic is a nonprofit worldwide leader in medical care, research and...
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Rochester, MN
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660 S Euclid Ave
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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Saint Louis, MO
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450 Serra Mall
Stanford, California 94305
(650) 723-2300
Stanford University Stanford University, located between San Francisco and San Jose in the heart of...
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Stanford, CA
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Washington, District of Columbia 20010
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Washington,
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