Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy



Status:Completed
Conditions:Neurology, Hematology
Therapuetic Areas:Hematology, Neurology
Healthy:No
Age Range:18 - 82
Updated:1/25/2019
Start Date:March 15, 2013
End Date:November 7, 2017

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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)

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).

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.

Inclusion Criteria:

- Stage 1 and Stage 2 FAP participants with the following:

1. NIS score within protocol criteria

2. Documented transthyretin variant by genotyping

3. 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

Exclusion Criteria:

- 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
We found this trial at
10
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Rochester, Minnesota 55905
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1 Gustave L Levy Pl # 271
New York, New York 10029
 (212) 241-6500
Mount Sinai Med Ctr Founded in 1852, The Mount Sinai Hospital is a 1,171-bed, tertiary-care...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Baltimore, MD
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Buenos Aires,
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Buenos Aires,
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340 W 10th St #6200
Indianapolis, Indiana 46202
(317) 274-3772
Indiana University School of Medicine With more than 2,000 students in 2013, the Indiana University...
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Indianapolis, IN
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Orange, California 92868
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Orange, CA
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Philadelphia, Pennsylvania
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Philadelphia, PA
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