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Open-Label Extension Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02175004
Recruitment Status : Active, not recruiting
First Posted : June 26, 2014
Last Update Posted : August 26, 2019
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE June 12, 2014
First Posted Date  ICMJE June 26, 2014
Last Update Posted Date August 26, 2019
Actual Study Start Date  ICMJE June 2014
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 18, 2017)
  • 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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 24, 2014)
  • Types of adverse events that occur during treatment [ Time Frame: 78 Weeks ]
  • Change from baseline in blood pressure (systolic and diastolic), heart rate, and body weight [ Time Frame: 78 Weeks ]
  • Change from baseline in results of routine laboratory test panel (routine serum chemistry, hematology, and urinalysis) [ Time Frame: 78 Weeks ]
  • Change from baseline in number of concomitant medications used [ Time Frame: 78 Weeks ]
  • Change from baseline in QTcF determined from electrocardiogram measurements [ Time Frame: 78 Weeks ]
  • Change from baseline in visual acuity measured during ophthalmic exam [ Time Frame: 78 Weeks ]
  • Change from baseline in light detection ability measured by electroretinography [ Time Frame: 78 Weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 18, 2017)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 24, 2014)
  • Change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: Baseline, Week 78 ]
  • Change from baseline in the Norfolk Quality of Life Diabetic Neuropathy Questionnaire [ Time Frame: Baseline, Week 78 ]
  • Change from baseline in the Polyneuropathy disability score (PND) [ Time Frame: Baseline, Week 78 ]
  • Change from baseline in the Modified body mass index (mBMI) and body mass index (BMI) [ Time Frame: Baseline, Week 78 ]
  • Change in baseline in Transthyretin (TTR) and Retinol Binding Protein 4 (RBP4) [ Time Frame: Baseline, Week 78 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-Label Extension Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
Official Title  ICMJE An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)
Brief Summary This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
Detailed Description

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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • FAP
  • Familial Amyloid Polyneuropathy
  • TTR
  • Transthyretin
  • Amyloidosis
Intervention  ICMJE Drug: IONIS-TTR Rx
300 mg IONIS-TTR Rx administered once weekly
Study Arms  ICMJE Experimental: IONIS-TTR Rx
Intervention: Drug: IONIS-TTR Rx
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: October 18, 2017)
135
Original Estimated Enrollment  ICMJE
 (submitted: June 24, 2014)
195
Estimated Study Completion Date  ICMJE September 2022
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2

Exclusion Criteria:

  • Any new condition or worsening of existing condition that could make the patient unsuitable for participation, or interfere with the patient participating in and/or completing the study
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Brazil,   France,   Germany,   Italy,   Portugal,   Spain,   United Kingdom,   United States
Removed Location Countries New Zealand
 
Administrative Information
NCT Number  ICMJE NCT02175004
Other Study ID Numbers  ICMJE ISIS 420915-CS3
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ionis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Ionis Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Ionis Pharmaceuticals, Inc.
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP