ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
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)
1 other identifier
interventional
206
9 countries
61
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Dec 2014
61 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2014
CompletedFirst Submitted
Initial submission to the registry
December 12, 2014
CompletedFirst Posted
Study publicly available on registry
December 18, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 30, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
March 30, 2017
CompletedResults Posted
Study results publicly available
July 18, 2018
CompletedJuly 18, 2018
June 1, 2018
2.3 years
December 12, 2014
March 27, 2018
June 22, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
6 Minute Walk Distance (6-MWD)
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
The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
18 months
Secondary Outcomes (6)
Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization
18 months
New York Heart Association (NYHA) Class
18 months
Kansas City Cardiomyopathy Questionnaire (KCCQ)
18 months
Cardiovascular (CV) Mortality
18 months
Cardiovascular (CV) Hospitalization
18 months
- +1 more secondary outcomes
Study Arms (2)
Revusiran (ALN-TTRSC)
ACTIVE COMPARATORadministered by subcutaneous (SC) injection
Sterile Normal Saline (0.9% NaCl)
PLACEBO COMPARATORadministered by subcutaneous (SC) injection
Interventions
Eligibility Criteria
You may qualify if:
- Documented TTR mutation
- Amyloid deposits in cardiac or non-cardiac tissue
- Medical history of heart failure
- Evidence of cardiac involvement by echocardiogram
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (61)
Clinical Trial Site
Bakersfield, California, United States
Clinical Trial Site
Beverly Hills, California, United States
Clinical Trial Site
La Mesa, California, United States
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Los Angeles, California, United States
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San Francisco, California, United States
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Stanford, California, United States
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Torrance, California, United States
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Aurora, Colorado, United States
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Washington D.C., District of Columbia, 20037, United States
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Washington D.C., District of Columbia, 20422, United States
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Tampa, Florida, United States
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Atlanta, Georgia, United States
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Chicago, Illinois, 60611, United States
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Chicago, Illinois, 60612, United States
Clinical Trial Site
Chicago, Illinois, 60637, United States
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New Orleans, Louisiana, United States
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Baltimore, Maryland, 21201, United States
Clinical Trial Site
Baltimore, Maryland, 21287, United States
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Boston, Massachusetts, 02111, United States
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Boston, Massachusetts, 02118, United States
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Boston, Massachusetts, 02215, United States
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Detroit, Michigan, United States
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Rochester, Minnesota, United States
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St Louis, Missouri, United States
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Newark, New Jersey, United States
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New York, New York, United States
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Rosedale, New York, United States
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The Bronx, New York, United States
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Durham, North Carolina, United States
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Cleveland, Ohio, 44106, United States
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Cleveland, Ohio, 44195, United States
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Columbus, Ohio, United States
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Philadelphia, Pennsylvania, 19102, United States
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Philadelphia, Pennsylvania, 19104, United States
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Charleston, South Carolina, United States
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Nashville, Tennessee, United States
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Dallas, Texas, United States
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Fort Worth, Texas, United States
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Houston, Texas, United States
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Richmond, Virginia, United States
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Seattle, Washington, United States
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Anderlecht, Belgium
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Hasselt, Belgium
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Roeselare, Belgium
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Toronto, Canada
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Bordeaux, Aquitaine, France
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Créteil, France
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Marseille, France
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Paris, France
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Heidelberg, Germany
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Münster, Germany
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Bologna, Italy
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Messina, Italy
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Pavia, Italy
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Barcelona, 8003, Spain
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Barcelona, 8907, Spain
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Madrid, Spain
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Umeå, Sweden
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Croydon, England, United Kingdom
Clinical Trial Site
London, England, United Kingdom
Clinical Trial Site
Tooting, United Kingdom
Related Publications (1)
Judge DP, Kristen AV, Grogan M, Maurer MS, Falk RH, Hanna M, Gillmore J, Garg P, Vaishnaw AK, Harrop J, Powell C, Karsten V, Zhang X, Sweetser MT, Vest J, Hawkins PN. Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR). Cardiovasc Drugs Ther. 2020 Jun;34(3):357-370. doi: 10.1007/s10557-019-06919-4.
PMID: 32062791DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
While dosing of patients was discontinued early, a protocol amendment allowed additional safety follow-up visits. Per the new assessment schedule, this study did not meet the criteria of a Terminated study, as additional follow-up visits occurred.
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- Alnylam Pharmaceuticals Inc
Study Officials
- STUDY DIRECTOR
Jared Gollob, MD
Alnylam Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 12, 2014
First Posted
December 18, 2014
Study Start
December 1, 2014
Primary Completion
March 30, 2017
Study Completion
March 30, 2017
Last Updated
July 18, 2018
Results First Posted
July 18, 2018
Record last verified: 2018-06