Status:
ACTIVE_NOT_RECRUITING
Early Detection of Neuropathy in ATTRv
Lead Sponsor:
University of Pennsylvania
Collaborating Sponsors:
Ionis Pharmaceuticals, Inc.
AstraZeneca
Conditions:
Hereditary Amyloidosis, Transthyretin-Related
Eligibility:
All Genders
30-90 years
Brief Summary
The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.
Detailed Description
Early detection of peripheral neuropathy in patients with TTRv is important to support initiation of therapy that alters the course of the disease. Current tools used to detect peripheral neuropathy m...
Eligibility Criteria
Inclusion
- Patients with known TTR mutations and neuropathy
- Patients with TTR mutation and no symptoms within less than 10 years of typical onset of disease
- Age criteria must meet the following:
- Non V122I mutations, Age 40 or older.
- V122 I mutations, 55 or older.
- Healthy persons without neuropathy
- 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
- 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
- Patients with neuropathy other than TTR amyloid
- Subjects with risk factors for neuropathy (diabetes, history of neuropathy in the family, neurotoxic drugs) or with neurological disorder associated with elevated NFL
Key Trial Info
Start Date :
February 14 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 14 2029
Estimated Enrollment :
47 Patients enrolled
Trial Details
Trial ID
NCT05311488
Start Date
February 14 2022
End Date
February 14 2029
Last Update
September 18 2025
Active Locations (1)
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1
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104