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

February 15, 2024 updated by: Alnylam Pharmaceuticals

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis (ATTR 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).

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

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.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Alnylam EAP Hotline
  • Phone Number: (617) 715-0200
  • Email: eap@alnylam.com

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Mayo Clinic Hospital
    • California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center
      • San Diego, California, United States, 92093
        • University of California San Diego
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic Florida
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago
      • Evanston, Illinois, United States, 60201
        • NorthShore University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University Health Hospital
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • The University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • The Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Boston University
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University Of Nebraska Medical Center
    • New York
      • New York, New York, United States, 10032
        • Columbia University Irving Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
      • Columbus, Ohio, United States, 43214
        • OhioHealth Research Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Penn Presbyerian Medical Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University
    • Texas
      • Dallas, Texas, United States, 75204
        • Baylor Scott & White Research Institute
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • University of Utah

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 85 years (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

August 11, 2022

First Submitted That Met QC Criteria

August 17, 2022

First Posted (Actual)

August 18, 2022

Study Record Updates

Last Update Posted (Actual)

February 16, 2024

Last Update Submitted That Met QC Criteria

February 15, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTR02-014

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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