Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Completed · Phase 2/Phase 3 · Has a placebo group

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

In brief

The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).

Key facts

Study ID

NCT01737398

Run by

Ionis Pharmaceuticals, Inc.

People needed

173

Starts

2013-03-15

Expected to finish

2017-11-07

Last updated by the study team

2019-07-17

Who can join

Age: 18 and older, up to 82. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Stage 1 and Stage 2 FAP participants with the following:
• NIS score within protocol criteria
• Documented transthyretin variant by genotyping
• Documented amyloid deposit by biopsy
• Females of child-bearing potential must use appropriate contraception and be non-pregnant and non-lactating. Males engaging in relations of child-bearing potential are to use appropriate contraception

You may not qualify if…

• Low Retinol level at screen
• Karnofsky performance status ≤50
• Poor Renal function
• Known type 1 or type 2 diabetes mellitus
• Other causes of sensorimotor or autonomic neuropathy (for example, autoimmune disease)
• If previously treated with Vyndaqel®, will need to have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, will need to have discontinued treatment for 3 days prior to Study Day 1
• Previous treatment with any oligonucleotide or siRNA within 12 months of screening
• Prior liver transplant or anticipated liver transplant within 1 year of screening
• New York Heart Association (NYHA) functional classification of ≥3
• Acute Coronary Syndrome or major surgery within 3 months of screening
• Known Primary or Leptomeningeal Amyloidosis
• Anticipated survival less than 2 years
• Any other conditions in the opinion of the investigator which interfere with the participant participating in 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
• UNIFESP — 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
• Auckland City Hospital — Auckland, New Zealand
• CHLN - Hospital de Santa Maria — Lisbon, Portugal
• CHP-HGSA, Unidade Clinica de Paramiloidose — Porto, Portugal
• Hospital Universitari Vall D' Hebron — Barcelona, Spain
• Hospital Clínic — Barcelona, Spain
• University College London - National Amyloidosis Centre — London, United Kingdom

Full record on ClinicalTrials.gov

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