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wtATTR-CIAP Burden Study

Title Dissecting the neuropathic burden in wtATTR: its role in staging and burden of disease, and in differentiating from chronic idiopathic axonal polyneuropathy
Therapeutic Area Transthyretin Cardiac Amyloidosis
Principal Investigator Dr. Oscar Soto and Dr. Maria Bartkus
Min Age 18 Years
Gender Any
More Information N/A


This is an observational study to assess the the burden of neuropathic involvement in wild-type transthyretin amyloidosis

Study Details

Inclusion Criteria

1. At least 18 years of age

2. Ability to provide informed consent

3. Adult diagnosed with of ATTR by immunohistochemistry and TTR genetic testing without pathogenic variant. Tafamidis (+) and tafamidis (-) or Chronic idiopathic axonal polyneuropathy (CIAP)

Exclusion Criteria

1. Evidence of polyneuropahty of any cause or disorder commonly associated with polyneuropathy such as: diabetes mellitus, renal insufficiency, hypothyroidism, vitamin B12 deficiency, and paraproteinemia

Study Requirements

Day 1 Screening

• Review of your medical record

• Obtain informed consent

• Phone interview with Dr. Bartkus Day 1 Baseline assessments- in Neurology Dept. at TMC

• Questionnaire Administration • Nerve conduction studies

• Quantitative sensory testing • Heart rate deep breathing

• Quantitative Sweat Measurement System (Q-Sweat)

• Cutaneous and Mixed Nerve Silent Period (CSP, MSP) Week 52 (1-year) Follow-up

• Longitudinal assessment phone call (optional)