A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (NEURO-TTR Study)

CompletadoFase 3ClinicalTrials.gov

ID: NCT01737398Tipo: INTERVENTIONALInicio: 15 de mar de 2013Fin estimado: 7 de nov de 2017

Traducción no disponible, mostrando original

Resumen

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).

Descripción detallada

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. Inotersen (also known as ISIS 420915) is an antisense drug that was designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein would result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression. The purpose of this study is to determine if inotersen can slow or stop the nerve damage caused by TTR deposits. This study will enroll late Stage 1 and early Stage 2 FAP participants. Participants will receive either inotersen or placebo for 65 weeks.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 82 YearsSexo: ALL

Criterios de inclusión

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

Criterios de exclusión

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

Intervenciones

drug

Inotersen

drug

Placebo

Ubicaciones

FLENI

Buenos Aires, Argentina

Patrocinadores

PrincipalIonis Pharmaceuticals, Inc.

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