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

Active, not recruiting

• Conditions: Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
• Interventions: Drug: Tafamidis

• Registration Number: NCT06393465
• Lead Sponsor: Pfizer

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-04-26
• Study First Submitted QC: 2024-04-26
• Study First Posted: 2024-05-01
• Study Start: 2024-06-15
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-05
• Last Update Posted: 2025-05-08
• Primary Completion: 2025-06-30
• Study Completion: 2025-06-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 50

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with mixed phenotype ATTR-CM	Tafamidis	ATTR-CM patients presenting with mixed phenotype

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Pfizer; 🇺🇸 New York, New York, United States