Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
47 participants
OBSERVATIONAL
2022-02-14
2029-02-14
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_CONTROL
PROSPECTIVE
Study Groups
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Symptomatic TTRv
Patients with known TTR mutations and neuropathy
neurofilament light chain
Blood test
In-vivo Meissner Corpuscle imaging
Imaging
Nerve conduction study
Nerve conduction study
Asymptomatic TTRv
Patients with TTR mutation and no symptoms within less than 10 years of typical onset of disease
neurofilament light chain
Blood test
In-vivo Meissner Corpuscle imaging
Imaging
Nerve conduction study
Nerve conduction study
Healthy controls
Age and sex matched healthy controls without neuropathy or other neurological disorder.
neurofilament light chain
Blood test
In-vivo Meissner Corpuscle imaging
Imaging
Nerve conduction study
Nerve conduction study
Interventions
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neurofilament light chain
Blood test
In-vivo Meissner Corpuscle imaging
Imaging
Nerve conduction study
Nerve conduction study
Eligibility Criteria
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Inclusion Criteria
1. Patients with TTR mutation and no symptoms within less than 10 years of typical onset of disease
2. Age criteria must meet the following:
* Non V122I mutations, Age 40 or older.
* V122 I mutations, 55 or older.
Healthy persons without neuropathy
1. The following distribution of age ranges will be considered when enrolling healthy participants:
* 5 patients age 30-40
* 5 patients age 40-50
* 5 patients age 50-60
* 5 patients age 60-70
2. Healthy control subjects for this study are defined as subjects with no symptoms of neuropathy or risk factors for neuropathy such as family history of hereditary neuropathy, chemotherapy, diabetes, autoimmune disease, or vitamin deficiency. Their status will be verified by medical records review.
Exclusion Criteria
2. Subjects with risk factors for neuropathy (diabetes, history of neuropathy in the family, neurotoxic drugs) or with neurological disorder associated with elevated NFL
30 Years
90 Years
ALL
Yes
Sponsors
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Ionis Pharmaceuticals, Inc.
INDUSTRY
AstraZeneca
INDUSTRY
University of Pennsylvania
OTHER
Responsible Party
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Principal Investigators
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Chafic Karam, MD
Role: PRINCIPAL_INVESTIGATOR
University of Pennsylvania
Brian Drachman
Role: PRINCIPAL_INVESTIGATOR
University of Pennsylvania
Sami Khella, MD
Role: PRINCIPAL_INVESTIGATOR
University of Pennsylvania
Janice Pieretti, MD
Role: PRINCIPAL_INVESTIGATOR
University of Pennsylvania
Locations
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University of Pennsylvania
Philadelphia, Pennsylvania, United States
Countries
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Other Identifiers
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849579
Identifier Type: -
Identifier Source: org_study_id
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