Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02319005 | ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | PHASE3 | COMPLETED | 206 | — | — | Dec 1, 2014 | Mar 30, 2017 | Jul 18, 2018 | 61 | United States, Belgium +7 |
The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
| Arm | Type | Description |
|---|---|---|
| Revusiran (ALN-TTRSC) | ACTIVE_COMPARATOR | administered by subcutaneous (SC) injection |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | administered by subcutaneous (SC) injection |
| Name | Type | Description |
|---|---|---|
| Revusiran (ALN-TTRSC) | DRUG | - |
| Sterile Normal Saline (0.9% NaCl) | DRUG | - |
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...