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

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: ATTR Amyloidosis; Transthyretin (TTR)-Mediated Amyloidosis; Familial Amyloidotic Polyneuropathy (FAP); Familial Amyloid Neuropathies

Treatments: Drug: Revusiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT02595983

ALN-TTRSC-005

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

All Patients

Experimental group

Description:

All patients who received at least 1 dose of revusiran (ALN-TTRSC)

Treatment:

Drug: Revusiran