Open-Label Extension Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
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|ClinicalTrials.gov Identifier: NCT02175004|
Recruitment Status : Active, not recruiting
First Posted : June 26, 2014
Last Update Posted : August 26, 2019
|Condition or disease||Intervention/treatment||Phase|
|FAP Familial Amyloid Polyneuropathy TTR Transthyretin Amyloidosis||Drug: IONIS-TTR Rx||Phase 3|
Expanded Access : An investigational treatment associated with this study has been approved for sale to the public. More info ...
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.
This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||135 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)|
|Actual Study Start Date :||June 2014|
|Estimated Primary Completion Date :||June 2022|
|Estimated Study Completion Date :||September 2022|
|Experimental: IONIS-TTR Rx||
Drug: IONIS-TTR Rx
300 mg IONIS-TTR Rx administered once weekly
- Types of adverse events that occur during treatment [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in blood pressure (systolic and diastolic), heart rate, and body weight [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in results of routine laboratory test panel (routine serum chemistry, hematology, and urinalysis) [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in QTcF determined from electrocardiogram measurements [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in number of concomitant medications used [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in visual acuity measured during ophthalmic exam [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in light detection ability measured by electroretinography [ Time Frame: Week 52 of Year 5 ]
- Change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: 78 and 156 Weeks ]
- Change from baseline in the Neuropathy Impairment Score [ Time Frame: Week 52 of Years 4 and 5 ]
- Change from baseline in the Norfolk Quality of Life Diabetic Neuropathy Questionnaire [ Time Frame: Week 78, 156 and Week 52 of Years 4 and 5 ]
- Change from baseline in the Modified body mass index (mBMI) and body mass index (BMI) [ Time Frame: 78 and 156 Weeks ]
- Change from baseline in the Polyneuropathy disability score (PND) [ Time Frame: Week 78, 156 and Week 52 of Years 4 and 5 ]
- Change in baseline in Transthyretin (TTR) and Retinol Binding Protein 4 (RBP4) [ Time Frame: Week 78, 156 and Week 52 of Years 4 and 5 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02175004