Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
|ClinicalTrials.gov Identifier: NCT01737398|
Recruitment Status : Completed
First Posted : November 29, 2012
Last Update Posted : June 19, 2018
|Condition or disease||Intervention/treatment||Phase|
|FAP Familial Amyloid Polyneuropathy TTR Transthyretin Amyloidosis||Drug: Inotersen Drug: Placebo||Phase 2 Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
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 was 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 was to determine if inotersen could slow or stop the nerve damage caused by TTR deposits. This study enrolled late Stage 1 and early Stage 2 FAP participants. Participants received either inotersen or placebo for 65 weeks.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||172 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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)|
|Actual Study Start Date :||March 15, 2013|
|Actual Primary Completion Date :||March 3, 2017|
|Actual Study Completion Date :||November 7, 2017|
Active Comparator: Inotersen
300 mg inotersen administered subcutaneously (SC) 3 times on alternate days in the first week and then once-weekly for 64 weeks
Active Comparator: Placebo
Placebo administered SC 3 times on alternate days in the first week and then once-weekly for 64 weeks
- Efficacy of IONIS-TTR Rx as measured by change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: 65 weeks ]
- Efficacy of IONIS-TTR Rx as measured by change from baseline in the Norfolk Quality of Life Diabetic Neuropathy questionnaire [ Time Frame: 65 weeks ]
- Efficacy of IONIS-TTR Rx based on the change from baseline in the following measures: [ Time Frame: 65 weeks ]
- Modified Body Mass Index and Body Mass Index
- Individual components of the mNIS+7
- Pharmacodynamic effect of IONIS-TTR Rx based on the change from baseline in transthyretin and retinol binding protein 4 [ Time Frame: 65 weeks ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01737398
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