ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

June 22, 2018 updated by: Alnylam Pharmaceuticals

A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

206

Phase

  • Phase 3

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

  • Belgium
      • Anderlecht, Belgium
        • Clinical Trial Site
      • Hasselt, Belgium
        • Clinical Trial Site
      • Roeselare, Belgium
        • Clinical Trial Site
  • Canada
      • Toronto, Canada
        • Clinical Trial Site
  • France
      • Creteil, France
        • Clinical Trial Site
      • Marseille, France
        • Clinical Trial Site
      • Paris, France
        • Clinical Trial Site
    • Aquitaine
      • Bordeaux, Aquitaine, France
        • Clinical Trial Site
  • Germany
      • Heidelberg, Germany
        • Clinical Trial Site
      • Muenster, Germany
        • Clinical Trial Site
  • Italy
      • Bologna, Italy
        • Clinical Trial Site
      • Messina, Italy
        • Clinical Trial Site
      • Pavia, Italy
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain, 8003
        • Clinical Trial Site
      • Barcelona, Spain, 8907
        • Clinical Trial Site
      • Madrid, Spain
        • Clinical Trial Site
  • Sweden
      • Umea, Sweden
        • Clinical Trial Site
  • United Kingdom
      • Tooting, United Kingdom
        • Clinical Trial Site
    • England
      • Croydon, England, United Kingdom
        • Clinical Trial Site
      • London, England, United Kingdom
        • Clinical Trial Site
  • United States
    • California
      • Bakersfield, California, United States
        • Clinical Trial Site
      • Beverly Hills, California, United States
        • Clinical Trial Site
      • La Mesa, California, United States
        • Clinical Trial Site
      • Los Angeles, California, United States
        • Clinical Trial Site
      • San Francisco, California, United States
        • Clinical Trial Site
      • Stanford, California, United States
        • Clinical Trial Site
      • Torrance, California, United States
        • Clinical Trial Site
    • Colorado
      • Aurora, Colorado, United States
        • Clinical Trial Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20037
        • Clinical Trial Site
      • Washington, District of Columbia, United States, 20422
        • Clinical Trial Site
    • Florida
      • Tampa, Florida, United States
        • Clinical Trial Site
    • Georgia
      • Atlanta, Georgia, United States
        • Clinical Trial Site
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Clinical Trial Site
      • Chicago, Illinois, United States, 60637
        • Clinical Trial Site
      • Chicago, Illinois, United States, 60612
        • Clinical Trial Site
    • Louisiana
      • New Orleans, Louisiana, United States
        • Clinical Trial Site
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • Clinical Trial Site
      • Baltimore, Maryland, United States, 21287
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Clinical Trial Site
      • Boston, Massachusetts, United States, 02111
        • Clinical Trial Site
      • Boston, Massachusetts, United States, 02215
        • Clinical Trial Site
    • Michigan
      • Detroit, Michigan, United States
        • Clinical Trial Site
    • Minnesota
      • Rochester, Minnesota, United States
        • Clinical Trial Site
    • Missouri
      • Saint Louis, Missouri, United States
        • Clinical Trial Site
    • New Jersey
      • Newark, New Jersey, United States
        • Clinical Trial Site
    • New York
      • Bronx, New York, United States
        • Clinical Trial Site
      • New York, New York, United States
        • Clinical Trial Site
      • Rosedale, New York, United States
        • Clinical Trial Site
    • North Carolina
      • Durham, North Carolina, United States
        • Clinical Trial Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Clinical Trial Site
      • Cleveland, Ohio, United States, 44106
        • Clinical Trial Site
      • Columbus, Ohio, United States
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Clinical Trial Site
      • Philadelphia, Pennsylvania, United States, 19102
        • Clinical Trial Site
    • South Carolina
      • Charleston, South Carolina, United States
        • Clinical Trial Site
    • Tennessee
      • Nashville, Tennessee, United States
        • Clinical Trial Site
    • Texas
      • Dallas, Texas, United States
        • Clinical Trial Site
      • Fort Worth, Texas, United States
        • Clinical Trial Site
      • Houston, Texas, United States
        • Clinical Trial Site
    • Virginia
      • Richmond, Virginia, United States
        • Clinical Trial Site
    • Washington
      • Seattle, Washington, United States
        • 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

18 years to 90 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Documented TTR mutation
  • Amyloid deposits in cardiac or non-cardiac tissue
  • Medical history of heart failure
  • Evidence of cardiac involvement by echocardiogram

Exclusion Criteria:

  • Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non-FAC hereditary cardiomyopathy, hypertensive cardiomyopathy, or cardiomyopathy due to valvular heart disease
  • Has known peripheral vascular disease affecting ambulation
  • Has a Polyneuropathy Disability score >2
  • Has a New York Heart Association (NYHA) classification of IV

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Revusiran (ALN-TTRSC)
administered by subcutaneous (SC) injection
Drug: Revusiran (ALN-TTRSC)
Placebo Comparator: Sterile Normal Saline (0.9% NaCl)
administered by subcutaneous (SC) injection
Drug: Sterile Normal Saline (0.9% NaCl)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
6 Minute Walk Distance (6-MWD)
Time Frame: 18 months
The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
18 months
Serum TTR Levels
Time Frame: 18 months
The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
18 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization
Time Frame: 18 months
Number of cardiovascular-related deaths and cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
18 months
New York Heart Association (NYHA) Class
Time Frame: 18 months
The difference between revusiran (ALN-TTRSC) and placebo group in the change from baseline to 18 months in the NYHA class
18 months
Kansas City Cardiomyopathy Questionnaire (KCCQ)
Time Frame: 18 months
The difference between revusiran (ALN-TTRSC) and placebo group in the change from Baseline to 18 months in the Kansas City Cardiomyopathy Questionnaire
18 months
Cardiovascular (CV) Mortality
Time Frame: 18 months
Number of cardiovascular-related deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
18 months
Cardiovascular (CV) Hospitalization
Time Frame: 18 months
Number of cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
18 months
All-cause Mortality
Time Frame: 18 months
Total number of deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
18 months

Collaborators and Investigators

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

Sponsor

Alnylam Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

December 1, 2014

Primary Completion (Actual)

March 30, 2017

Study Completion (Actual)

March 30, 2017

Study Registration Dates

First Submitted

December 12, 2014

First Submitted That Met QC Criteria

December 17, 2014

First Posted (Estimate)

December 18, 2014

Study Record Updates

Last Update Posted (Actual)

July 18, 2018

Last Update Submitted That Met QC Criteria

June 22, 2018

Last Verified

June 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTRSC-004

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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