Vutrisiran for Amyloid Cardiomyopathy

Recruiting at 71 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Alnylam Pharmaceuticals

Must be taking: Vutrisiran, Patisiran

Disqualifiers: Other investigational studies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 36 months

800 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, it seems that participants are either continuing or switching from specific related treatments, so it's best to discuss your current medications with the study team.

What data supports the effectiveness of the drug Vutrisiran for amyloid cardiomyopathy?

Vutrisiran has shown effectiveness in reducing transthyretin (TTR) levels, which is important for treating amyloidosis, a condition related to amyloid cardiomyopathy. It has been approved for treating polyneuropathy in hereditary ATTR amyloidosis, indicating its potential in managing related conditions. ^{1 2 3 4 5}

How is the drug Vutrisiran unique for treating amyloid cardiomyopathy?

Vutrisiran is unique because it is a small interfering RNA (siRNA) drug that targets and reduces the production of transthyretin (TTR), a protein involved in amyloid cardiomyopathy. It is administered subcutaneously (under the skin) and has shown a potent and sustained reduction in TTR levels, which is different from other treatments that may not directly target TTR production. ^{1 2 5 6 7}

Research Team

Medical Director

Principal Investigator

Alnylam Pharmaceuticals

Eligibility Criteria

This trial is for patients with a heart condition called Transthyretin Amyloidosis with Cardiomyopathy. It's not specified, but typically participants should be adults who meet certain health standards and have the specific medical condition being studied.

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive vutrisiran 25 mg administered subcutaneously once every 3 months

36 months

Visits every 3 months for treatment administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue on extended use of vutrisiran or switch from patisiran

36 months

Treatment Details

Interventions

• Vutrisiran

Trial Overview The study is testing Vutrisiran, a medication aimed at treating Transthyretin Amyloidosis with Cardiomyopathy. The trial will assess its safety and effectiveness, including how it affects the body over an extended period or when switching from another drug, patisiran.

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).

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

References

1. United States pubmed.ncbi.nlm.nih.gov

Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects. [2022]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Vutrisiran: First Approval. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Clinical management of amyloid cardiomyopathy. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Traditional Heart Failure Therapies in Patients With Cardiac Amyloidosis and Heart Failure. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Reduction in CMR Derived Extracellular Volume With Patisiran Indicates Cardiac Amyloid Regression. [2021]

6. Japan pubmed.ncbi.nlm.nih.gov

Hemodynamic testing to guide vasodilator therapy in cardiac amyloidosis. [2023]

7. New Zealand pubmed.ncbi.nlm.nih.gov

Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran. [2022]