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

Expanded access is currently available for this treatment.
Verified May 2017 by Alnylam Pharmaceuticals
Information provided by (Responsible Party):
Alnylam Pharmaceuticals Identifier:
First received: October 18, 2016
Last updated: May 4, 2017
Last verified: May 2017
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Condition Intervention
TTR-mediated Amyloidosis
Amyloidosis, Hereditary
Amyloid Neuropathies, Familial
Familial Amyloid Polyneuropathies
Amyloid Neuropathies
Amyloidosis, Hereditary, Transthyretin-Related
Drug: patisiran (ALN-TTR02)

Study Type: Expanded Access     What is Expanded Access?
Official Title: Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy

Resource links provided by NLM:

Further study details as provided by Alnylam Pharmaceuticals:

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

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02939820

Contact: Alnylam EAP Hotline (617) 715-0200

United States, Iowa
University of Iowa
Iowa City, Iowa, United States
United States, Louisiana
Ochsner Medical Center
New Orleans, Louisiana, United States
United States, Maryland
Johns Hopkins Medical Center
Baltimore, Maryland, United States
United States, Massachusetts
Boston University Medical Center
Boston, Massachusetts, United States
United States, Minnesota
Mayo Clinic - Rochester
Rochester, Minnesota, United States
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States
United States, New York
Columbia University Medical Center
New York, New York, United States
United States, Pennsylvania
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States
United States, Texas
Dallas, Texas, United States
Sponsors and Collaborators
Alnylam Pharmaceuticals
Study Director: Sunita Goyal, MD Alnylam Pharmaceuticals
  More Information

Additional Information:
Responsible Party: Alnylam Pharmaceuticals Identifier: NCT02939820     History of Changes
Other Study ID Numbers: ALN-TTR02-007
Study First Received: October 18, 2016
Last Updated: May 4, 2017

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
Familial Amyloid Polyneuropathies

Additional relevant MeSH terms:
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis, Familial
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors processed this record on May 23, 2017