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Trial record 89 of 368 for:    transthyretin

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
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief Summary:
This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

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 ...

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 135 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: IONIS-TTR Rx Drug: IONIS-TTR Rx
300 mg IONIS-TTR Rx administered once weekly




Primary Outcome Measures :
  1. Types of adverse events that occur during treatment [ Time Frame: Week 52 of Year 5 ]
  2. Change from baseline in blood pressure (systolic and diastolic), heart rate, and body weight [ Time Frame: Week 52 of Year 5 ]
  3. Change from baseline in results of routine laboratory test panel (routine serum chemistry, hematology, and urinalysis) [ Time Frame: Week 52 of Year 5 ]
  4. Change from baseline in QTcF determined from electrocardiogram measurements [ Time Frame: Week 52 of Year 5 ]
  5. Change from baseline in number of concomitant medications used [ Time Frame: Week 52 of Year 5 ]
  6. Change from baseline in visual acuity measured during ophthalmic exam [ Time Frame: Week 52 of Year 5 ]
  7. Change from baseline in light detection ability measured by electroretinography [ Time Frame: Week 52 of Year 5 ]

Secondary Outcome Measures :
  1. Change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: 78 and 156 Weeks ]
  2. Change from baseline in the Neuropathy Impairment Score [ Time Frame: Week 52 of Years 4 and 5 ]
  3. 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 ]
  4. Change from baseline in the Modified body mass index (mBMI) and body mass index (BMI) [ Time Frame: 78 and 156 Weeks ]
  5. Change from baseline in the Polyneuropathy disability score (PND) [ Time Frame: Week 78, 156 and Week 52 of Years 4 and 5 ]
  6. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

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


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Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.

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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02175004     History of Changes
Other Study ID Numbers: ISIS 420915-CS3
First Posted: June 26, 2014    Key Record Dates
Last Update Posted: August 26, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ionis Pharmaceuticals, Inc.:
FAP
Familial Amyloid Polyneuropathy
TTR
Transthyretin
Amyloidosis
Additional relevant MeSH terms:
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Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors