Phase 3 N=206 Randomized Double-blind Treatment

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

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

Bottom Line

View on ClinicalTrials.gov: NCT02319005 ↗

Enrolled (actual)

206

Serious AEs

56.8%

Results posted

Jul 2018

Primary outcome: Primary: 6 Minute Walk Distance (6-MWD)

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Revusiran (ALN-TTRSC) (Drug); Sterile Normal Saline (0.9% NaCl) (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Mar 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY 6 Minute Walk Distance (6-MWD)	—	—
PRIMARY Serum TTR Levels	—	—
SECONDARY Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization	—	—
SECONDARY New York Heart Association (NYHA) Class	—	—
SECONDARY Kansas City Cardiomyopathy Questionnaire (KCCQ)	—	—
SECONDARY Cardiovascular (CV) Mortality	—	—
SECONDARY Cardiovascular (CV) Hospitalization	—	—
SECONDARY All-cause Mortality	—	—

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.

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02319005). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.