Patisiran for Cardiomyopathy

Recruiting at 63 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Alnylam Pharmaceuticals

Must be taking: Tafamidis

Must not be taking: TTR lowering treatment

Disqualifiers: Primary amyloidosis, Heart failure IV, Neuropathy, others

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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

360 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that participants can be on tafamidis if they have been taking it for at least 6 months and still show disease progression.

How does the drug Patisiran differ from other treatments for cardiomyopathy?

Patisiran is unique because it is a small interfering RNA (siRNA) therapy that specifically targets and reduces the production of transthyretin, a protein that can misfold and accumulate in the heart, leading to cardiomyopathy. This mechanism of action is different from other treatments like sacubitril/valsartan or valsartan, which primarily work by blocking certain receptors or enzymes to manage heart failure symptoms. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial is testing a medication called patisiran. It aims to help people with a heart condition caused by abnormal protein buildup. The medication works by lowering the levels of these harmful proteins in the body. Patisiran has been shown to significantly reduce symptoms and improve quality of life in patients.

Research Team

Medical Director

Principal Investigator

Alnylam Pharmaceuticals

Eligibility Criteria

This trial is for adults with a heart condition called ATTR amyloidosis with cardiomyopathy. They should have had heart failure symptoms or hospitalization, be stable without recent cardiovascular hospitalizations, able to walk a short distance in 6 minutes, and not severely affected by other diseases like severe lung disease or arthritis. People who've had certain organ transplants or infections like hepatitis B/C or HIV can't join.

Inclusion Criteria

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

I can walk more than 150 meters in 6 minutes.

Your blood test for NT-proBNP shows levels above 300 ng/L and below 8500 ng/L. If you have atrial fibrillation, your NT-proBNP levels should be above 600 ng/L and below 8500 ng/L.

Exclusion Criteria

My heart condition is serious and considered high risk.

I need a cane or wheelchair to walk due to nerve damage.

I have been diagnosed with primary or leptomeningeal amyloidosis.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of patisiran or placebo in the double-blind period

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants receive multiple doses of patisiran in the open-label extension period

Treatment Details

Interventions

• Patisiran
• Placebo

Trial Overview The study tests the effectiveness and safety of Patisiran compared to a placebo in treating ATTR amyloidosis affecting the heart. Participants will either receive Patisiran or an inactive substance without knowing which one they're getting to see if there's an improvement in their heart condition.

Participant Groups

Group I: Patisiran Experimental Treatment 1 Intervention

Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.

Group II: Placebo Placebo Group 2 Interventions

Participants will be administered multiple doses of placebo in the double-blind period. In the open-label extension period, participants will be administered multiple doses of patisiran.

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. England pubmed.ncbi.nlm.nih.gov

A putative placebo analysis of the effects of sacubitril/valsartan in heart failure across the full range of ejection fraction. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Sacubitril-Valsartan in a routine community population: attention to volume status critical to achieving target dose. [2021]

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

Angiotensin II type 1 receptor blocker, valsartan, prevented cardiac fibrosis in rat cardiomyopathy after autoimmune myocarditis. [2015]

4. Thailand pubmed.ncbi.nlm.nih.gov

Short-term evaluation of captopril in patients with chronic left sided valvular regurgitations. [2013]

5. England pubmed.ncbi.nlm.nih.gov

Metoprolol benefits in cardiomyopathy. [2019]