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OpenTrials
Completed

NCT Number: NCT06393465

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

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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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Pfizer

New York, 10001, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Tafamidis

Drug

61 milligrams under real world conditions

Other names: Vyndamax

Primary outcomes

  1. Rate of neurologic disease progression

    Time frame: 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 outcomes

  1. Change from Baseline in modified Body Mass Index (BMI)

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 1, 2024
Registry last updated
Apr 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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