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

Completed · Phase 3 · Has a placebo group

Conditions studied: FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

In brief

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

Key facts

Study ID

NCT02175004

Run by

Ionis Pharmaceuticals, Inc.

People needed

135

Starts

2014-06-26

Expected to finish

2021-01-07

Last updated by the study team

2023-11-18

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

• 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

Full record on ClinicalTrials.gov

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