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

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06393465
• Other study ID #: B3461121
• Status: Not yet recruiting
• Start date: April 29, 2024
• Est. completion date: June 30, 2024

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 50
• Est. completion date: June 30, 2024
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.
• 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.

Related Conditions & MeSH terms

• Amyloidosis
• Cardiomyopathies
• Disease Progression
• Nervous System Diseases
• Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Intervention

Drug:
• Tafamidis
61 milligrams under real world conditions

Locations

Country	Name	City	State
United States	Pfizer	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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