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

Launched by ALNYLAM PHARMACEUTICALS · Nov 6, 2024

Keywords

ClinConnect Summary

This is an open‑label extension study testing vutrisiran in adults with transthyretin amyloidosis with cardiomyopathy (ATTR‑CM). The trial aims to learn more about long‑term safety and possible benefits of the drug after participants have already completed related studies. About 700 people worldwide are expected to enroll. Eligibility includes adults 18 and older who are currently in the parent studies ALN‑TTR02‑011 or ALN‑TTR02‑014, or who completed the 24‑month extension in ALN‑TTRSC02‑003. People who have permanently stopped the drug in those studies, are enrolled in another investigational study, or have other medical conditions that could interfere with participation are not eligible.

If you join, you’ll receive vutrisiran 25 mg by under‑the‑skin injection every 3 months. The study will monitor safety by recording any adverse events for up to three years. It will also assess how you feel and function, using a heart‑related quality of life questionnaire (KCCQ‑OS), changes in heart failure symptoms (NYHA class), and blood tests that measure heart stress (NT‑proBNP) and heart muscle injury (Troponin I), plus changes in transthyretin levels. The trial will track overall death and cardiovascular events, with a focus on up to 36 months of follow‑up. It’s run across many international sites, and results aren’t available yet. The sponsor is Alnylam, and data from eligible participants may be shared in anonymized form after study completion.

Gender

ALL

Eligibility criteria

• 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