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

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 3

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

Treatments: Drug: Revusiran (ALN-TTRSC); Drug: Sterile Normal Saline (0.9% NaCl)

Study type: Interventional

Funder types: Industry

Identifiers

NCT02319005

ALN-TTRSC-004

Details and patient eligibility

About

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.

Enrollment: 206 patients

Sex: All

Ages: 18 to 90 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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206 participants in 2 patient groups, including a placebo group

Revusiran (ALN-TTRSC)

Active Comparator group

Description:

administered by subcutaneous (SC) injection

Treatment:

Drug: Revusiran (ALN-TTRSC)

Sterile Normal Saline (0.9% NaCl)

Placebo Comparator group

Description:

administered by subcutaneous (SC) injection

Treatment:

Drug: Sterile Normal Saline (0.9% NaCl)