Overview

This trial is active, not recruiting.

Conditions transthyretin (ttr)-mediated amyloidosis, familial amyloidotic polyneuropathy (fap), attr amyloidosis, familial amyloid neuropathies
Treatment revusiran (aln-ttrsc)
Phase phase 2
Sponsor Alnylam Pharmaceuticals
Start date October 2015
End date October 2016
Trial size 12 participants
Trial identifier NCT02595983, ALN-TTRSC-005

Summary

Dosing discontinued; Follow-up of patients for safety

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
revusiran (aln-ttrsc)
500mg Revusiran by subcutaneous (sc) injection

Primary Outcomes

Measure
Percent reduction in serum TTR level compared to baseline
time frame: 18 months

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants at least 18 years old.

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)

Additional Information

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 Transplant
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Alnylam Pharmaceuticals.