A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

10 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-03-08

Study Completion Date

2023-05-19

Brief Summary

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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.

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Detailed Description

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Conditions

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Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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Patients with mixed phenotype ATTRv-CM

Hereditary ATTR-CM patients presenting with mixed phenotype

tafamidis

Intervention Type DRUG

80 or 61 milligrams (mg)

Interventions

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tafamidis

80 or 61 milligrams (mg)

Intervention Type DRUG

Other Intervention Names

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Vyndaqel Vyndamax

Eligibility Criteria

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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)

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No