Transthyretin Amyloid Cardiomyopathy Clinical Trial
Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
The objective of the study is to assess the long-term safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy as assessed by a review of adverse events (AEs).
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Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hATTR amyloidosis with cardiomyopathy or wtATTR amyloidosis with cardiomyopathy; AND
Had an inadequate response to or could not tolerate standard of care, in the opinion of the investigator.
Is not eligible for on-label use of commercial patisiran in the opinion of the investigator.
New York Heart Association (NYHA) Class IV
NYHA Class III AND ATTR amyloidosis disease Stage 3 (defined as both N-terminal prohormone B-type natriuretic peptide (NT-proBNP) >3000 ng/L and estimated glomerular filtration rate [eGFR] <45 ml/min/1.73 m^2). [Gillmore 2018]
Current or future participation in another investigational device or drug study, scheduled to occur during this study, or has received an investigational agent or device within 30 days (or 5 half-lives of the investigational drug, whichever is longer) prior to dosing (Day 1). Patients who have previously participated in a gene therapy trial for hATTR amyloidosis.
Patients currently enrolled in, eligible for inclusion in, or who have dropped out of an ongoing interventional (therapeutic) clinical trial related to ATTR amyloidosis.
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There are 22 Locations for this study
Phoenix Arizona, 85054, United States
Los Angeles California, 90048, United States
San Diego California, 92093, United States
Jacksonville Florida, 32224, United States
Chicago Illinois, 60637, United States
Evanston Illinois, 60201, United States
Indianapolis Indiana, 46202, United States
Kansas City Kansas, 66160, United States
Baltimore Maryland, 21205, United States
Boston Massachusetts, 02118, United States
Rochester Minnesota, 55905, United States
Saint Louis Missouri, 63110, United States
Omaha Nebraska, 68198, United States
New York New York, 10032, United States
Cleveland Ohio, 44195, United States
Columbus Ohio, 43214, United States
Portland Oregon, 97239, United States
Philadelphia Pennsylvania, 19104, United States
Nashville Tennessee, 37232, United States
Austin Texas, 78759, United States
Dallas Texas, 75204, United States
Salt Lake City Utah, 84112, United States
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