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Revusiran

Phase 3

Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Small molecule | Rare Disease |Alnylam Pharmaceuticals, Inc.|Last Updated: Jul 9, 2020

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment206

FDA Designations

No designations recorded

Clinical trial landscape

Revusiran · 3 trials · 11 indications

Phase 3 1Phase 2 2
NCT02319005ENDEAVOUR: 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)
COMPLETED206 Analytics
PHASE3COMPLETED
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)Unlock trial analytics

Study Endpoints

Primary Endpoints

6 Minute Walk Distance (6-MWD)
18 months

The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes

Serum TTR Levels
18 months

The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months

Percentage Change From Baseline in Serum TTR at Month 6
Month 6

A negative percentage change from baseline at Month 6 indicates a reduction in serum TTR level.

Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.
Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months

The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study \[drug\] discontinuation.

Secondary Endpoints

Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization
18 months
New York Heart Association (NYHA) Class
18 months
Kansas City Cardiomyopathy Questionnaire (KCCQ)
18 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Revusiran (ALN-TTRSC)ACTIVE_COMPARATORadministered by subcutaneous (SC) injection
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATORadministered by subcutaneous (SC) injection
All PatientsEXPERIMENTALAll patients who received at least 1 dose of revusiran (ALN-TTRSC)

Interventions

NameTypeDescription
Revusiran (ALN-TTRSC)DRUG -
Sterile Normal Saline (0.9% NaCl)DRUG -
RevusiranDRUG500mg Revusiran by subcutaneous (sc) injection
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Eligibility Criteria

Age Range18 Years to 90 Years
SexALL
Healthy VolunteersNo
Study Sites61

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 cardiom...

Countries:United StatesBelgiumCanadaFranceGermanyItalySpainSwedenUnited KingdomPortugal
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Frequently asked questions about Revusiran

What is Revusiran used for?

Revusiran is an investigational drug being developed for transthyretin (TTR)-mediated amyloidosis, including TTR-mediated familial amyloidotic cardiomyopathy (FAC) and TTR-mediated amyloidosis in patients whose disease worsened after liver transplant. It is a small molecule being studied in clinical trials for these rare disease indications.

Who makes Revusiran?

Revusiran is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ALNY. Alnylam is conducting clinical trials to evaluate Revusiran for the treatment of transthyretin-mediated amyloidosis conditions.

What phase is Revusiran in?

Revusiran has been studied in Phase 2 and Phase 3 clinical trials. The Phase 3 ENDEAVOUR study evaluated Revusiran in patients with transthyretin-mediated familial amyloidotic cardiomyopathy, while Phase 2 trials assessed it in TTR-mediated amyloidosis. All trials listed are completed, and Revusiran remains investigational.

What clinical trials is Revusiran in?

Revusiran has been studied in three completed clinical trials: NCT02292186, a Phase 2 extension study in TTR cardiac amyloidosis; NCT02319005, the Phase 3 ENDEAVOUR study in TTR-mediated familial amyloidotic cardiomyopathy; and NCT02595983, a Phase 2 study in TTR-mediated amyloidosis patients whose disease worsened after liver transplant.

Is Revusiran the same as ALN-TTRSC?

Yes, Revusiran is also known as ALN-TTRSC. Clinical trial records refer to Revusiran as ALN-TTRSC, and the drug has been evaluated under this name in studies for transthyretin-mediated amyloidosis, including familial amyloidotic cardiomyopathy and amyloidosis after liver transplant.