NCT06393465已完成不适用
Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Rate of neurologic disease progression
研究概览
简要总结
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
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Patients with mixed phenotype ATTR-CM
ATTR-CM patients presenting with mixed phenotype
干预措施: Tafamidis (Drug)
结局指标
主要结局
Rate of neurologic disease progression
时间窗: 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.
次要结局
- Change from Baseline in modified Body Mass Index (BMI)(Baseline (BL) through at least 12 months of treatment)
研究者
研究点 (1)
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