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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02939820
Expanded Access Status : Approved for marketing
First Posted : October 20, 2016
Last Update Posted : October 19, 2020
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date October 18, 2016
First Posted Date October 20, 2016
Last Update Posted Date October 19, 2020
Descriptive Information
Brief Title Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
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.
Study Type Expanded Access
Intervention Drug: patisiran (ALN-TTR02)
patisiran (ALN-TTR02) administered by intravenous (IV) infusion
Publications * Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27. Review.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Approved for marketing
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Not Provided
Removed Location Countries United States
Administrative Information
NCT Number NCT02939820
Responsible Party Alnylam Pharmaceuticals
Study Sponsor Alnylam Pharmaceuticals
Collaborators Not Provided
Study Director: Medical Director Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date October 2020