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

NCT ID: NCT02319005

Last Updated: 2018-07-18

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

206 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-12-31

Study Completion Date

2017-03-30

Brief Summary

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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.

Detailed Description

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Conditions

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Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) Amyloidosis, Hereditary Amyloid Neuropathies, Familial Amyloid Neuropathies Amyloidosis, Hereditary, Transthyretin-Related Familial Transthyretin Cardiac Amyloidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Revusiran (ALN-TTRSC)

administered by subcutaneous (SC) injection

Group Type ACTIVE_COMPARATOR

Revusiran (ALN-TTRSC)

Intervention Type DRUG

Sterile Normal Saline (0.9% NaCl)

administered by subcutaneous (SC) injection

Group Type PLACEBO_COMPARATOR

Sterile Normal Saline (0.9% NaCl)

Intervention Type DRUG

Interventions

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Revusiran (ALN-TTRSC)

Intervention Type DRUG

Sterile Normal Saline (0.9% NaCl)

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jared Gollob, MD

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

Bakersfield, California, United States

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Beverly Hills, California, United States

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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, United States

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Washington D.C., District of Columbia, United States

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Tampa, Florida, United States

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Chicago, Illinois, United States

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Chicago, Illinois, United States

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New Orleans, Louisiana, United States

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Baltimore, Maryland, United States

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Baltimore, Maryland, United States

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Boston, Massachusetts, United States

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Boston, Massachusetts, United States

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Boston, Massachusetts, 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, United States

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Cleveland, Ohio, United States

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Columbus, Ohio, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, 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, , Spain

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Barcelona, , Spain

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Madrid, , Spain

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Umeå, , Sweden

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Croydon, England, United Kingdom

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London, England, United Kingdom

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Tooting, , United Kingdom

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Countries

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United States Belgium Canada France Germany Italy Spain Sweden United Kingdom

References

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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.

Reference Type DERIVED
PMID: 32062791 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ALN-TTRSC-004

Identifier Type: -

Identifier Source: org_study_id

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