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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02595983
Recruitment Status : Completed
First Posted : November 4, 2015
Last Update Posted : December 12, 2017
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Transthyretin (TTR)-Mediated Amyloidosis Familial Amyloidotic Polyneuropathy (FAP) ATTR Amyloidosis Familial Amyloid Neuropathies Drug: Revusiran (ALN-TTRSC) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : October 2015
Actual Primary Completion Date : February 6, 2017
Actual Study Completion Date : February 6, 2017

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Revusiran (ALN-TTRSC) Drug: Revusiran (ALN-TTRSC)
500mg Revusiran by subcutaneous (sc) injection

Primary Outcome Measures :
  1. Percent reduction in serum TTR level compared to baseline [ Time Frame: 18 months ]

Secondary Outcome Measures :
  1. Change from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score [ Time Frame: 18 months ]
  2. Changes from baseline in quality of life using the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire [ Time Frame: 18 months ]
  3. Change from baseline in polyneuropathy disability (PND) score [ Time Frame: 18 months ]
  4. 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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02595983

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Clinical Trial SIte
Paris, France
Clinical Trial SIte
Münster, Germany
Clinical Trial SIte
Porto, Portugal
Clinical Trial SIte
Majorca, Spain
Clinical Trial Site
Umea, Sweden
United Kingdom
Clinical Trial SIte
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
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Study Director: John Vest, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals Identifier: NCT02595983     History of Changes
Other Study ID Numbers: ALN-TTRSC-005
First Posted: November 4, 2015    Key Record Dates
Last Update Posted: December 12, 2017
Last Verified: December 2017

Keywords provided by Alnylam Pharmaceuticals:
Familial Amyloidotic Polyneuropathy
ATTR Amyloidosis
Orthotopic Liver Transplant
RNAi therapeutic

Additional relevant MeSH terms:
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Amyloid Neuropathies
Amyloid Neuropathies, Familial
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors