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

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ClinicalTrials.gov Identifier: NCT02319005
Recruitment Status : Completed
First Posted : December 18, 2014
Results First Posted : July 18, 2018
Last Update Posted : July 18, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) Amyloidosis, Hereditary Amyloid Neuropathies, Familial Amyloid Neuropathies Amyloidosis, Hereditary, Transthyretin-Related Familial Transthyretin Cardiac Amyloidosis Drug: Revusiran (ALN-TTRSC) Drug: Sterile Normal Saline (0.9% NaCl) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 206 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official 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)
Study Start Date : December 2014
Actual Primary Completion Date : March 30, 2017
Actual Study Completion Date : March 30, 2017


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)



Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02319005


  Show 61 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alnylam Pharmaceuticals:
Study Protocol  [PDF] February 19, 2016
Statistical Analysis Plan  [PDF] October 21, 2015


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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02319005     History of Changes
Other Study ID Numbers: ALN-TTRSC-004
First Posted: December 18, 2014    Key Record Dates
Results First Posted: July 18, 2018
Last Update Posted: July 18, 2018
Last Verified: June 2018

Keywords provided by Alnylam Pharmaceuticals:
Cardiomyopathy
Heart Failure
FAC
Amyloid
Transthyretin
TTR
RNAi therapeutic

Additional relevant MeSH terms:
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Cardiomyopathies
Amyloidosis
Amyloid Neuropathies
Amyloidosis, Familial
Amyloid Neuropathies, Familial
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases