An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

N
Noel Dasgupta

Primary Investigator

Enrolling By Invitation
18 years or above
All
Phase N/A
7 participants needed
1 Location

Brief description of study

What is the purpose of this study?

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

THIS STUDY IS ENROLLING BY INVITATION ONLY - This study is only enrolling individuals who have been participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003

Detailed description of study

What will happen during the study?

Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
  • Age: 18 years or above
  • Gender: All

Inclusion Criteria:

  • Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003

Exclusion Criteria:

  • Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
  • Future or current participation in another investigational device or drug study, scheduled to occur during this study
  • Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation

Updated on 22 Apr 2025. Study ID: PATH-ALNYLAM-VUTRISIRAN, 24994
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting referrals by invitation only