Vutrisiran for Transthyretin Amyloidosis with Cardiomyopathy
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the drug Vutrisiran for treating transthyretin amyloidosis with cardiomyopathy?
Vutrisiran has shown to effectively reduce transthyretin levels in healthy subjects, which is promising for treating transthyretin amyloidosis. Additionally, similar drugs like patisiran have shown positive results in reducing heart-related symptoms in patients with transthyretin cardiac amyloidosis.12345
Is Vutrisiran safe for humans?
How is the drug Vutrisiran different from other treatments for transthyretin amyloidosis with cardiomyopathy?
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
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Double-blind Treatment
Participants receive vutrisiran 25 mg SC or placebo once every 3 months
Randomized Treatment Extension (RTE)
Participants receive vutrisiran 25 mg SC every 3 months or 50 mg SC every 6 months
Open-label Extension (OLE)
Participants receive vutrisiran 25 mg SC every 3 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Vutrisiran
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alnylam Pharmaceuticals
Lead Sponsor
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