Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

NO_LONGER_AVAILABLE

Conditions studied: Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy

In brief

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).

Key facts

Study ID

NCT05505838

Run by

Alnylam Pharmaceuticals

Last updated by the study team

2024-02-16

Who can join

Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

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.
• Patients currently enrolled in, eligible for inclusion in, or who have dropped out of an ongoing interventional (therapeutic) clinical trial related to ATTR amyloidosis.

Where it is running

• 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

Full record on ClinicalTrials.gov

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