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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-06-15

Study Completion Date

2025-12-31

Brief Summary

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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

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Detailed Description

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Conditions

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Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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Patients with mixed phenotype ATTR-CM

ATTR-CM patients presenting with mixed phenotype

Tafamidis

Intervention Type DRUG

61 milligrams under real world conditions

Interventions

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Tafamidis

61 milligrams under real world conditions

Intervention Type DRUG

Other Intervention Names

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Vyndamax

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No