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

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06393465

Enrollment

Registered

2024-05-01

Start date

2024-06-15

Completion date

2026-04-07

Last updated

2026-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Brief summary

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

Interventions

DRUGTafamidis

61 milligrams under real world conditions

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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.

Design outcomes

Primary

Measure	Time frame	Description
Rate of neurologic disease progression	Baseline through at least 12 months of treatment	Describe and compare the rate of neurologic disease progression before and after initiation of tafamidis in patients with mixed-phenotype ATTR-CM receiving tafamidis 61 mg daily in a real world setting.

Secondary

Measure	Time frame	Description
Change from Baseline in modified Body Mass Index (BMI)	Baseline (BL) through at least 12 months of treatment	Assess change from BL in mBMI in patients with mixed phenotype ATTR-CM 61 mg tafamidis

Countries

United States

Contacts

STUDY_DIRECTORPfizer CT.gov Call Center

Pfizer

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 30, 2026