Purpose

The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

Condition

Eligibility

Eligible Ages
Between 18 Years and 85 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with cardiomyopathy - Medical history of heart failure with at least 1 prior hospitalization for heart failure, or current clinical evidence (signs and symptoms of heart failure) - Clinically stable with no cardiovascular related hospitalizations within 6 weeks of study start - Has never taken tafamidis before (tafamidis naïve) or currently on tafamidis for ≥6 months with evidence of disease progression while on tafamidis treatment - Able to complete ≥150 m on the 6-minute walk test - Screening N-terminal pro B-type natriuretic peptide (NT-proBNP), a blood marker of heart failure severity, >300 ng/L and <8500 ng/L; in participants with permanent or persistent atrial fibrillation, screening NT-proBNP> 600 ng/L and <8500 ng/L

Exclusion Criteria

  • Known primary amyloidosis (AL) or leptomeningeal amyloidosis. - Received prior TTR lowering treatment - New York Heart Association heart failure classification of III and at high risk - New York Heart Association heart failure classification of IV - Neuropathy requiring cane or stick to walk, or is wheelchair bound - Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2 - Abnormal liver function - Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection - Has non-amyloid disease that significantly affects ability to walk (e.g., severe chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular disease affecting ambulation) - Prior or planned heart, liver, or other organ transplant - Other cardiomyopathy not related to ATTR amyloidosis

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Patisiran
Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.
  • Drug: Patisiran
    Patisiran will be administered by intravenous (IV) infusion.
    Other names:
    • ALN-TTR02
    • patisiran-LNP
Placebo Comparator
Placebo
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.
  • Drug: Placebo
    Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously.
  • Drug: Patisiran
    Patisiran will be administered by intravenous (IV) infusion.
    Other names:
    • ALN-TTR02
    • patisiran-LNP

More Details

Status
Active, not recruiting
Sponsor
Alnylam Pharmaceuticals

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.