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

NCT06393465 | Completed

• 2 other identifiers: B3461121NCT06393465
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

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

Conditions

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Change from Baseline in modified Body Mass Index (BMI)

  Baseline (BL) through at least 12 months of treatment

Study Arms (1)

Patients with mixed phenotype ATTR-CM

ATTR-CM patients presenting with mixed phenotype

Drug: Tafamidis

Interventions

Tafamidis DRUG

61 milligrams under real world conditions

Also known as: Vyndamax

Patients with mixed phenotype ATTR-CM

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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).

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Pfizer

New York, New York, 10001, United States

Location

Related Links

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

MeSH Terms

Interventions

tafamidis

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2024
• First Posted: May 1, 2024
• Study Start: June 15, 2024
• Primary Completion: April 7, 2026
• Study Completion: April 7, 2026
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)