Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Last updated: November 17, 2023
Sponsor: Ionis Pharmaceuticals, Inc.
Overall Status: Completed

Phase

3

Condition

Amyloidosis

Treatment

Inotersen

Clinical Study ID

NCT02175004
ISIS 420915-CS3
2013-004561-13
  • Ages > 18
  • All Genders

Study Summary

This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2

Exclusion

Exclusion Criteria:

  • Any new condition or worsening of existing condition that could make the patientunsuitable for participation, or interfere with the patient participating in and/orcompleting the study

Study Design

Total Participants: 135
Treatment Group(s): 1
Primary Treatment: Inotersen
Phase: 3
Study Start date:
June 26, 2014
Estimated Completion Date:
January 07, 2021

Study Description

Familial Amyloid Polyneuropathy (FAP) is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP.

IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression.

This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Connect with a study center

  • FLENI

    Buenos Aires,
    Argentina

    Site Not Available

  • Federal University of Rio de Janeiro - University Hospital

    Rio de Janeiro, CEP 21941913
    Brazil

    Site Not Available

  • AACD

    Sao Paulo,
    Brazil

    Site Not Available

  • CHU Henri Mondor - Department of Neurology

    Creteil, 94000
    France

    Site Not Available

  • CHU Bicetre Aphp French Referral Center for FAP/Cornamyl Network

    Le Kremlin Bicetre, 94275
    France

    Site Not Available

  • UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin

    Munster, 48149
    Germany

    Site Not Available

  • Universita Degli Studi Di Messina - Azienda Ospedaliera Universitaria Policlinico "Gaetano Martino"

    Messina, Sicily 98124
    Italy

    Site Not Available

  • Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100
    Italy

    Site Not Available

  • Auckland City Hospital

    Auckland,
    New Zealand

    Site Not Available

  • CHLN - Hospital de Santa Maria

    Lisbon, 1649-035
    Portugal

    Site Not Available

  • CHP-HGSA, Unidade Clinica de Paramiloidose

    Porto, 4099-001
    Portugal

    Site Not Available

  • Hospital Clinic

    Barcelona, 08036
    Spain

    Site Not Available

  • Hospital Universitari Vall D' Hebron

    Barcelona, 08035
    Spain

    Site Not Available

  • University College London - National Amyloidosis Centre

    London, NW3 2PF
    United Kingdom

    Site Not Available

  • University of California, Irvine

    Orange, California 92868
    United States

    Site Not Available

  • Indiana University School of Medicine

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Johns Hopkins University Bayview Medical Center

    Baltimore, Maryland 21205
    United States

    Site Not Available

  • Boston University School of Medicine - Amyloid Treatment & Research Program

    Boston, Massachusetts 02118
    United States

    Site Not Available

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • Columbia University Medical Center - The Neurological Institute

    New York, New York 10032
    United States

    Site Not Available

  • Mount Sinai Medical Center

    New York, New York 10029
    United States

    Site Not Available

  • Oregon Health & Science University

    Portland, Oregon 97239
    United States

    Site Not Available

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

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