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

Pfizer

Status: Completed

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

Treatments: Drug: tafamidis

Study type: Observational

Funder types: Industry

Identifiers

NCT05139680

B3461099

Details and patient eligibility

About

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.

Enrollment: 10 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

10 participants in 1 patient group

Patients with mixed phenotype ATTRv-CM

Description:

Hereditary ATTR-CM patients presenting with mixed phenotype

Treatment:

Drug: tafamidis