Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

May 16, 2024 updated by: Alnylam Pharmaceuticals

Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Study Overview

Status

Approved for marketing

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. For general information, see the links provided in More Information.

Study Type

Expanded Access

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 and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  • Male or female greater than or equal to 18 years of age
  • Have a diagnosis of hATTR
  • Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
  • Have adequate complete blood counts, liver function tests and coagulation tests

Exclusion Criteria:

  • Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
  • Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
  • Have inadequate cardiac function
  • Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
  • Have known serious comorbidities or considered unfit for the program by the investigator
  • Prior or planned liver or heart transplantation

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

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

October 18, 2016

First Submitted That Met QC Criteria

October 18, 2016

First Posted (Estimated)

October 20, 2016

Study Record Updates

Last Update Posted (Actual)

May 20, 2024

Last Update Submitted That Met QC Criteria

May 16, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTR02-007

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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