Phase 3
Completed N=206
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
Source: ClinicalTrials.gov NCT02319005 ↗
Enrolled (actual)
206
Serious AEs
56.8%
Results posted
Jul 2018
Primary outcomePrimary: 6 Minute Walk Distance (6-MWD)
◆ Published Evidence
Highly cited
108citations · ~18 / year
Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR).
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.
Linked Publications
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Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR).
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 |
— | — |
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
Data sourced from ClinicalTrials.gov (NCT02319005) and the linked publication. 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. Informational only — not medical advice.