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

Launched by PFIZER · Apr 26, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness of a medication called Tafamidis in treating a heart condition known as Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), specifically in patients who have a mixed type of this disease. The research will use existing medical records to see how well Tafamidis slows down the progression of neurological symptoms in patients who have been taking the medication for at least 12 months.

To be eligible for this study, patients need to be at least 18 years old and must have been diagnosed with either the hereditary or non-hereditary forms of ATTR-CM. Additionally, they should have been treated with Tafamidis for a year and have had at least one neurological assessment before and after starting the treatment. It's important to note that patients who have had organ transplants, are unable to walk, or have received other specific treatments for their condition may not qualify. If you or a loved one meets these criteria and has been on Tafamidis, this study could provide valuable insights into the medication's effects on neurological health.

Gender

ALL

Eligibility criteria

• 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.