A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Study Overview
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Brief Summary
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The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.

Official Title
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A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis Who Have Previously Received ALN-TTRSC

Conditions
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TTR-mediated Amyloidosis

Intervention / Treatment
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* Drug: Revusiran (ALN-TTRSC)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects who previously received and tolerated revusiran (ALN-TTRSC) in the ALN-TTRSC-002 study Adequate liver function Not Pregnant or nursing Exclusion Criteria: Inadequate renal function Uncontrolled hypertension, ischemic heart disease or clinically significant cardiac arrhythmia Untreated hypo- or hyperthyroidism Prior major organ transplant

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 90 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Revusiran (ALN-TTRSC)<br> | Drug: Revusiran (ALN-TTRSC)<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients. | The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study [drug] discontinuation. | Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months | 
| Serum TTR Levels | Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR | Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mortality | Total number of deaths in the study and total number of deaths adjudicated as being related to cardiovascular causes. Deaths were adjudicated by an independent adjudication committee as cardiovascular (CV) or non-CV events. | Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months | 
| Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization | Hospitalization events were adjudicated by an independent committee as cardiovascular (CV) or non-CV events | Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months | 
| 6-minute Walk Test Performance | Distance in meters walked in 6 minutes | Baseline, Month 6, and Month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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FAC, Cardiomyopathy, Heart Failure, Amyloid, Transthyretin, TTR, RNAi therapeutic