Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
1 other identifier
expanded_access
N/A
1 country
20
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 11, 2022
CompletedFirst Posted
Study publicly available on registry
August 18, 2022
CompletedFebruary 16, 2024
February 1, 2024
August 11, 2022
February 15, 2024
Conditions
Keywords
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
Mayo Clinic Hospital
Phoenix, Arizona, 85054, United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
University of California San Diego
San Diego, California, 92093, United States
Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
University of Chicago
Chicago, Illinois, 60637, United States
NorthShore University
Evanston, Illinois, 60201, United States
Indiana University Health Hospital
Indianapolis, Indiana, 46202, United States
The University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
The Johns Hopkins University
Baltimore, Maryland, 21205, United States
Boston University
Boston, Massachusetts, 02118, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Washington University
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Columbia University Irving Medical Center
New York, New York, 10032, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
OhioHealth Research Institute
Columbus, Ohio, 43214, United States
Penn Presbyerian Medical Center
Philadelphia, Pennsylvania, 19104, United States
Vanderbilt University
Nashville, Tennessee, 37232, United States
Baylor Scott & White Research Institute
Dallas, Texas, 75204, United States
University of Utah
Salt Lake City, Utah, 84112, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Alnylam Pharmaceuticals
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 11, 2022
First Posted
August 18, 2022
Last Updated
February 16, 2024
Record last verified: 2024-02