Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
NCT ID: NCT05505838
Last Updated: 2024-02-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NO_LONGER_AVAILABLE
EXPANDED_ACCESS
Brief Summary
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Detailed Description
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Conditions
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Interventions
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Patisiran
Patisiran administered by intravenous (IV) infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
18 Years
85 Years
ALL
No
Sponsors
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Alnylam Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Alnylam Pharmaceuticals
Locations
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Mayo Clinic Hospital
Phoenix, Arizona, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of California San Diego
San Diego, California, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of Chicago
Chicago, Illinois, United States
NorthShore University
Evanston, Illinois, United States
Indiana University Health Hospital
Indianapolis, Indiana, United States
The University of Kansas Medical Center
Kansas City, Kansas, United States
The Johns Hopkins University
Baltimore, Maryland, United States
Boston University
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University
St Louis, Missouri, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Columbia University Irving Medical Center
New York, New York, United States
Cleveland Clinic
Cleveland, Ohio, United States
OhioHealth Research Institute
Columbus, Ohio, United States
Penn Presbyerian Medical Center
Philadelphia, Pennsylvania, United States
Vanderbilt University
Nashville, Tennessee, United States
Baylor Scott & White Research Institute
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Countries
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Other Identifiers
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ALN-TTR02-014
Identifier Type: -
Identifier Source: org_study_id
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