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

July 7, 2020 updated by: Alnylam Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

25

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada
        • Clinical Trial Site
  • United Kingdom
      • London, United Kingdom, UK NW3 2PF
        • Clinical Trial Site
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02155
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10034
        • Clinical Trial Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 86 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Revusiran (ALN-TTRSC)
Drug: Revusiran (ALN-TTRSC)

What is the study measuring?

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.
Time Frame: 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.
Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Serum TTR Levels
Time Frame: 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
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
Time Frame: 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.
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
Time Frame: 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
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
Time Frame: Baseline, Month 6, and Month 12
Distance in meters walked in 6 minutes
Baseline, Month 6, and Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2014

Primary Completion (Actual)

February 22, 2017

Study Completion (Actual)

February 22, 2017

Study Registration Dates

First Submitted

November 10, 2014

First Submitted That Met QC Criteria

November 13, 2014

First Posted (Estimate)

November 17, 2014

Study Record Updates

Last Update Posted (Actual)

July 9, 2020

Last Update Submitted That Met QC Criteria

July 7, 2020

Last Verified

July 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTRSC-003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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