Transthyretin Amyloid Cardiomyopathy Clinical Trial
This Study Will Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
Summary
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Hereditary Transthyretin Amyloidosis using data that already exist in patients' medical records.
Eligibility Criteria
Inclusion Criteria:
Age ≥18 years at diagnosis
Diagnosed with ATTRv-CM, mixed phenotype
Treated with tafamidis (VYNDAQEL 80 mg [four 20-mg tafamidis meglumine capsules] orally once daily or VYNDAMAX 61 mg [one 61-mg tafamidis capsule] orally once daily) for ≥6 months
Have had ≥1 pre- and ≥2 post-treatment neurologic assessments
Exclusion Criteria:
History of organ transplant
Wild-type TTR genotype
Individuals who are non-ambulatory
Prior treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally once daily or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily
Peripheral neuropathy attributed to causes other than ATTR amyloidosis (e.g., diabetes mellitus, B12 deficiency, HIV infection)
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There is 1 Location for this study
New York New York, 10017, United States
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