Efficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy
Familial Amyloid Polyneuropathy
Drug: IONIS-TTR Rx
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|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|
- Efficacy of IONIS-TTR Rx as measured by change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: 65 weeks ] [ Designated as safety issue: No ]
- Efficacy of IONIS-TTR Rx as measured by change from baseline in the Norfolk Quality of Life Diabetic Neuropathy questionnaire [ Time Frame: 65 weeks ] [ Designated as safety issue: No ]
- Efficacy of IONIS-TTR Rx based on the change from baseline in the following measures: [ Time Frame: 65 weeks ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: No ]
|Study Start Date:||December 2012|
|Estimated Study Completion Date:||September 2017|
|Estimated Primary Completion Date:||March 2017 (Final data collection date for primary outcome measure)|
|Active Comparator: IONIS-TTR Rx||
Drug: IONIS-TTR Rx
300 mg IONIS-TTR Rx administered subcutaneously 3 times on alternate days in the first week and then once-weekly for 64 weeks.
|Active Comparator: Placebo||
Placebo administered subcutaneously 3 times on alternate days in the first week and then once-weekly for 64 weeks.
Familial Amyloid Polyneuropathy (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.
IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will 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 IONIS-TTR Rx can slow or stop the nerve damage caused by TTR deposits. This study will enroll late Stage 1 and early Stage 2 FAP patients. Patients will receive either IONIS-TTR Rx or placebo for 65 weeks.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01737398
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