Vutrisiran for Transthyretin Amyloidosis with Cardiomyopathy

Recruiting in Palo Alto (17 mi)

+100 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Alnylam Pharmaceuticals

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

Research Team

Medical Director

Principal Investigator

Alnylam Pharmaceuticals

Eligibility Criteria

This trial is for patients with a confirmed diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, which includes inherited or wild-type forms. Participants should have a history of heart failure, evidenced by hospitalization or clinical signs. Those with primary or leptomeningeal amyloidosis, severe polyneuropathy, advanced heart failure, very low kidney function, prior TTR treatments, or other types of cardiomyopathies cannot join.

Inclusion Criteria

I have been hospitalized for heart failure before or show signs of it.

I have been diagnosed with a type of heart disease caused by transthyretin amyloidosis.

Exclusion Criteria

I have been diagnosed with primary or leptomeningeal amyloidosis.

I have a heart condition not caused by TTR amyloidosis.

I have severe nerve damage affecting my daily activities.

Treatment Details

Interventions

• Vutrisiran (RNAi Therapeutics)

Trial Overview The study tests the effectiveness and safety of Vutrisiran given as an injection under the skin every three months compared to a placebo (saline solution). The goal is to see if Vutrisiran can help patients with ATTR amyloidosis affecting the heart.

Participant Groups

Group I: Vutrisiran 25 mg Experimental Treatment 1 Intervention

Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind (DB) period. After the DB period, participants enter the open-label randomized treatment extension (RTE) period to receive vutrisiran 25 mg SC q3M or vutrisiran 50 mg SC every 6 months (q6M). After implementation of Amendment 4, participants from the DB period will enter the open-label treatment extension (OLE) period to receive vutrisiran 25 mg SC q3M and participants who were previously receiving vutrisiran 50 mg q6M in the RTE period, will be transitioned to vutrisiran 25 mg q3M in the OLE period.

Group II: Placebo Placebo Group 2 Interventions

Participants will receive placebo during the double-blind period. After the DB period, participants enter the RTE period to receive vutrisiran 25 mg SC q3M or vutrisiran 50 mg SC q6M. After implementation of Amendment 4, participants from the DB period will enter the OLE period to receive vutrisiran 25 mg SC q3M and participants who were previously receiving vutrisiran 50 mg q6M in the RTE period, will be transitioned to vutrisiran 25 mg q3M in the OLE period.

Who Is Running the Clinical Trial?

Alnylam Pharmaceuticals

Lead Sponsor

Trials

81

Recruited

16,100+

Dr. Yvonne Greenstreet

Alnylam Pharmaceuticals

Chief Executive Officer since 2021

MD from the University of Leeds, MBA from INSEAD

Dr. Pushkal Garg

Alnylam Pharmaceuticals

Chief Medical Officer since 2016

MD from Columbia University