A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis Who Have Previously Received ALN-TTRSC
Overview
- Phase
- Phase 2
- Intervention
- Revusiran (ALN-TTRSC)
- Conditions
- TTR-mediated Amyloidosis
- Sponsor
- Alnylam Pharmaceuticals
- Enrollment
- 25
- Locations
- 1
- Primary Endpoint
- Serum TTR Levels
- Status
- Completed
- Last Updated
- 5 years ago
Overview
Brief Summary
The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subjects who previously received and tolerated revusiran (ALN-TTRSC) in the ALN-TTRSC-002 study
- •Adequate liver function
- •Not Pregnant or nursing
Exclusion Criteria
- •Inadequate renal function
- •Uncontrolled hypertension, ischemic heart disease or clinically significant cardiac arrhythmia
- •Untreated hypo- or hyperthyroidism
- •Prior major organ transplant
Arms & Interventions
Revusiran (ALN-TTRSC)
Intervention: Revusiran (ALN-TTRSC)
Outcomes
Primary Outcomes
Serum TTR Levels
Time Frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR
Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.
Time Frame: 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 Outcomes
- Mortality(Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months)
- Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization(Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months)
- 6-minute Walk Test Performance(Baseline, Month 6, and Month 12)