A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Alnylam Pharmaceuticals

Status and phase: Invitation-only; Phase 3

Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Treatments: Drug: Vutrisiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT06679946

ALN-TTRSC02-007

2024-516568-27 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 800 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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800 participants in 1 patient group

Vutrisiran 25 mg

Experimental group

Description:

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

Treatment:

Drug: Vutrisiran