Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
- Conditions
- Transthyretin-mediated Amyloidosis With CardiomyopathyATTR Amyloidosis With Cardiomyopathy
- Registration Number
- NCT05505838
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
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).
- 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.
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
-
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.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (20)
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
Mayo Clinic Florida
🇺🇸Jacksonville, Florida, United States
The University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Mayo Clinic Hospital
🇺🇸Phoenix, Arizona, United States
University of California San Diego
🇺🇸San Diego, California, United States
University of Chicago
🇺🇸Chicago, Illinois, United States
NorthShore University
🇺🇸Evanston, Illinois, United States
Indiana University Health Hospital
🇺🇸Indianapolis, Indiana, United States
The Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Boston University
🇺🇸Boston, Massachusetts, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
OhioHealth Research Institute
🇺🇸Columbus, Ohio, United States
Vanderbilt University
🇺🇸Nashville, Tennessee, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Penn Presbyerian Medical Center
🇺🇸Philadelphia, Pennsylvania, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Baylor Scott & White Research Institute
🇺🇸Dallas, Texas, United States