Skip to main content
OpenTrials
Completed

NCT Number: NCT02319005

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) Alzheimer Disease Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Amyloidosis, Hereditary Amyloidosis, Hereditary, Transthyretin-Related Brain Diseases Cardiomyopathies Cardiovascular Diseases Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Familial Transthyretin Cardiac Amyloidosis Genetic Diseases, Inborn Heart Diseases Heart Failure Heredodegenerative Disorders, Nervous System Mental Disorders Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Peripheral Nervous System Diseases Proteostasis Deficiencies Tauopathies

Age range

18 year–90 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Anderlecht, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Revusiran (ALN-TTRSC)

Drug

Sterile Normal Saline (0.9% NaCl)

Drug

Primary outcomes

  1. 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

  2. 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

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. Cardiovascular (CV) Mortality

    Time frame: 18 months

    Number of cardiovascular-related deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group

  5. Cardiovascular (CV) Hospitalization

    Time frame: 18 months

    Number of cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group

  6. All-cause Mortality

    Time frame: 18 months

    Total number of deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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)

Important dates

Study start
2014
Primary completion
2017
Study completion
2017
First posted
Dec 18, 2014
Registry last updated
Jul 18, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.