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)

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

Study Overview

• Status: Completed
• Conditions: Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
• Intervention / Treatment: Drug: Tafamidis

• Study Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10001
      • Pfizer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study will include patients with mixed-phenotype ATTR-CM who are treated in the Amyloidosis Program at MedStar in the U.S and three other study sites (Canada, Portugal and Italy).

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients with mixed phenotype ATTR-CM; ATTR-CM patients presenting with mixed phenotype	Drug: Tafamidis; 61 milligrams under real world conditions; Other Names: Vyndamax

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of neurologic disease progression	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.	Baseline through at least 12 months of treatment

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): June 15, 2024
• Primary Completion (Actual): April 7, 2026
• Study Completion (Actual): April 7, 2026

Study Registration Dates

• First Submitted: April 26, 2024
• First Submitted That Met QC Criteria: April 26, 2024
• First Posted (Actual): May 1, 2024

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 24, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Pathologic Processes; Heart Diseases; Disease Attributes; Metabolic Diseases; Proteostasis Deficiencies; Disease Progression; Cardiomyopathies; Nervous System Diseases; Amyloidosis; tafamidis
• Other Study ID Numbers: B3461121; NCT06393465 (Registry Identifier: ClinicalTrials.gov)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.