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

Phase 2

Completed

• Conditions: TTR-mediated Amyloidosis
• Interventions: Drug: Revusiran (ALN-TTRSC)

• Registration Number: NCT02292186
• Lead Sponsor: Alnylam Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-10
• Study First Submitted: 2014-11-10
• Study First Submitted QC: 2014-11-13
• Study First Posted: 2014-11-17
• Primary Completion: 2017-02-22
• Study Completion: 2017-02-22
• Results First Submitted: 2018-02-22
• Results First Submitted QC: 2018-05-16
• Results First Posted: 2018-06-15
• Status Verified: 2020-07
• Last Update Submitted: 2020-07-07
• Last Update Posted: 2020-07-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Revusiran (ALN-TTRSC)	Revusiran (ALN-TTRSC)	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Mortality	Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months	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.
Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization	Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months	Hospitalization events were adjudicated by an independent committee as cardiovascular (CV) or non-CV events
6-minute Walk Test Performance	Baseline, Month 6, and Month 12	Distance in meters walked in 6 minutes

Trial Locations

Locations (1)

Clinical Trial Site; 🇬🇧 London, United Kingdom