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

• Status: Completed
• Trial end date: 2017-02-06
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Diagnosis of FAP (familial amyloidotic polyneuropathy) with documented TTR mutation

• Received an orthotopic liver transplant ≥12 months before the date of informed consent

• An increase in polyneuropathy disability (PND) score post-transplant

• Polyneuropathy Disability score of ≤3b

Exclusion Criteria:

• New York Heart Association (NYHA) classification of >2

• Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)