This trial is active, not recruiting.

Conditions transthyretin (ttr) mediated familial amyloidotic cardiomyopathy (fac), amyloidosis, hereditary, amyloid neuropathies, familial, amyloid neuropathies, amyloidosis, hereditary, transthyretin-related, familial transthyretin cardiac amyloidosis
Treatments revusiran (aln-ttrsc), sterile normal saline (0.9% nacl)
Phase phase 3
Sponsor Alnylam Pharmaceuticals
Start date December 2014
End date October 2016
Trial size 206 participants
Trial identifier NCT02319005, ALN-TTRSC-004


Dosing discontinued; Follow-up of patients for safety

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
(Active Comparator)
administered by subcutaneous (SC) injection
revusiran (aln-ttrsc)
(Placebo Comparator)
administered by subcutaneous (SC) injection
sterile normal saline (0.9% nacl)

Primary Outcomes

6 Minute Walk Distance (6-MWD)
time frame: 18 months
Serum TTR levels
time frame: 18 months

Secondary Outcomes

Composite cardiovascular (CV) mortality and cardiovascular (CV) hospitalization
time frame: 18 months
New York Heart Association (NYHA) Class
time frame: 18 months
Kansas City Cardiomyopathy Questionnaire (KCCQ)
time frame: 18 months
Cardiovascular (CV) Mortality
time frame: 18 months
Cardiovascular (CV) hospitalization
time frame: 18 months
All-cause mortality
time frame: 18 months

Eligibility Criteria

Male or female participants from 18 years up to 90 years old.

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

Additional Information

Official title 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)
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Alnylam Pharmaceuticals.