Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

Pfizer

Status: Active, not recruiting

Conditions: Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Treatments: Drug: Tafamidis

Study type: Observational

Funder types: Industry

Identifiers

NCT06393465

B3461121

Details and patient eligibility

About

This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records

Enrollment: 50 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥18 years at diagnosis.
• Diagnosed with ATTRv-CM or ATTRwt-CM, mixed phenotype.
• Treated with tafamidis, as VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily for ≥12 months or started on tafamidis 20 mg then switched to VYNDAMAX 61 mg for ≥12 months
• Have had ≥1 pre- and ≥1 post-treatment neurologic assessments.

Exclusion criteria

• History of any organ transplant.
• Individuals who are non-ambulatory.
• Prior or current treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as 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 (eg, diabetes mellitus, B12 deficiency, hypothyroidism, shingles,Lyme disease, HIV infection, secondary to injury, chronic kidney disease).
• Patient's data fails to pass data quality checks.

Trial design

50 participants in 1 patient group

Patients with mixed phenotype ATTR-CM

Description:

ATTR-CM patients presenting with mixed phenotype

Treatment:

Drug: Tafamidis