Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
NCT ID: NCT02175004
Last Updated: 2023-11-18
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
135 participants
INTERVENTIONAL
2014-06-26
2021-01-07
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Previous Placebo-Inotersen 300 mg
Participants received subcutaneous (SC) doses of 300 milligrams (mg) inotersen once weekly for up to 260 weeks. Participants who received inotersen-matching placebo in the previous study- ISIS 420915-CS2 (NCT01737398) were included in this group.
Inotersen
Inotersen SC
Previous Inotersen-Inotersen 300 mg
Participants received SC doses of 300 mg inotersen once weekly for up to 260 weeks. Participants who received inotersen in the previous study- ISIS 420915-CS2 were included in this group.
Inotersen
Inotersen SC
Interventions
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Inotersen
Inotersen SC
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
18 Years
ALL
No
Sponsors
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Ionis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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University of California, Irvine
Orange, California, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Johns Hopkins University Bayview Medical Center
Baltimore, Maryland, United States
Boston University School of Medicine - Amyloid Treatment & Research Program
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Mount Sinai Medical Center
New York, New York, United States
Columbia University Medical Center - The Neurological Institute
New York, New York, United States
Oregon Health & Science University
Portland, Oregon, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States
FLENI
Buenos Aires, , Argentina
Federal University of Rio de Janeiro - University Hospital
Rio de Janeiro, , Brazil
AACD
São Paulo, , Brazil
CHU Henri Mondor - Department of Neurology
Créteil, , France
CHU Bicetre Aphp French Referral Center for FAP/Cornamyl Network
Le Kremlin-Bicêtre, , France
UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin
Münster, , Germany
Universita Degli Studi Di Messina - Azienda Ospedaliera Universitaria Policlinico "Gaetano Martino"
Messina, Sicily, Italy
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
CHLN - Hospital de Santa Maria
Lisbon, , Portugal
CHP-HGSA, Unidade Clinica de Paramiloidose
Porto, , Portugal
Hospital Universitari Vall D' Hebron
Barcelona, , Spain
Hospital Clinic
Barcelona, , Spain
University College London - National Amyloidosis Centre
London, , United Kingdom
Countries
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References
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Brannagan TH, Coelho T, Wang AK, Polydefkis MJ, Dyck PJ, Berk JL, Drachman B, Gorevic P, Whelan C, Conceicao I, Plante-Bordeneuve V, Merlini G, Obici L, Plana JMC, Gamez J, Kristen AV, Mazzeo A, Gentile L, Narayana A, Olugemo K, Aquino P, Benson MD, Gertz M; NEURO-T. T. R. Open-Label Extension Investigators. Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update. J Neurol. 2022 Dec;269(12):6416-6427. doi: 10.1007/s00415-022-11276-8. Epub 2022 Jul 31.
Karam C, Brown D, Yang M, Done N, Zhu JJ, Greatsinger A, Bozas A, Vera-Llonch M, Signorovitch J. Long-term treatment effects of inotersen on health-related quality of life in patients with hATTR amyloidosis with polyneuropathy: Analysis of the open-label extension of the NEURO-TTR trial. Muscle Nerve. 2022 Oct;66(4):438-446. doi: 10.1002/mus.27675. Epub 2022 Aug 4.
Yarlas A, Lovley A, McCausland K, Brown D, Vera-Llonch M, Conceicao I, Karam C, Khella S, Obici L, Waddington-Cruz M. Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR. Neurol Ther. 2021 Dec;10(2):865-886. doi: 10.1007/s40120-021-00268-x. Epub 2021 Aug 5.
Brannagan TH, Wang AK, Coelho T, Waddington Cruz M, Polydefkis MJ, Dyck PJ, Plante-Bordeneuve V, Berk JL, Barroso F, Merlini G, Conceicao I, Hughes SG, Kwoh J, Jung SW, Guthrie S, Pollock M, Benson MD, Gertz M; NEURO-TTR open-label extension investigators. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020 Aug;27(8):1374-1381. doi: 10.1111/ene.14285. Epub 2020 May 29.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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2013-004561-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ISIS 420915-CS3
Identifier Type: -
Identifier Source: org_study_id
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