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 Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-10-31

Study Completion Date

2017-02-06

Brief Summary

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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.

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Detailed Description

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Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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All Patients

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

Group Type EXPERIMENTAL

Revusiran

Intervention Type DRUG

500mg Revusiran by subcutaneous (sc) injection

Interventions

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Revusiran

500mg Revusiran by subcutaneous (sc) injection

Intervention Type DRUG

Other Intervention Names

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ALN-TTRSC

Eligibility Criteria

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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