Clinical Trial: The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-label Study to Evaluate the Efficacy and Safety of Revusiran in Patients With Transthyretin-mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post-Orthotopic Liver Transpla
Brief Summary: The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis, whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.
Detailed Summary:
Sponsor: Alnylam Pharmaceuticals
Current Primary Outcome: Percent reduction in serum TTR level compared to baseline [ Time Frame: 18 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score [ Time Frame: 18 months ]
- Changes from baseline in quality of life using the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire [ Time Frame: 18 months ]
- Change from baseline in polyneuropathy disability (PND) score [ Time Frame: 18 months ]
- Evaluate safety and tolerability of revusiran (ALN-TTRSC) as measured by the number of treatment-related adverse events in patients with familial amyloidotic polyneuropathy (FAP) [ Time Frame: 18 months ]Number of treatment-related AEs and SAEs
Original Secondary Outcome: Same as current
Information By: Alnylam Pharmaceuticals
Dates:
Date Received: October 30, 2015
Date Started: October 2015
Date Completion:
Last Updated: May 23, 2017
Last Verified: May 2017
