A Structured Survey Among Fabry Patients With a Focus on Adherence to Therapy, Quality of Life and Pain Control
试验速览
- 阶段
- 不适用
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Adherence to oral therapy with migalastat according to Medication Assessment Questionnaire
研究概览
简要总结
This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.
详细描述
Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Since 2016, chaperone therapy as a new therapeutic approach is available.
This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy.
All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Fabry disease (genetically confirmed)
- •Signed informed consent
- •18 years and older
排除标准
- •No informed consent
- •Withdrawal of informed consent
结局指标
主要结局
Adherence to oral therapy with migalastat according to Medication Assessment Questionnaire
时间窗: From date of inclusion up to104 weeks (2 years)
Pharmacological adherence according to adapted and translated 'Medication Assessment Questionnaire'.
次要结局
- Quality of Life according to SF-36 and Wuerzburg pain questionnaire(From date of inclusion up to104 weeks (2 years))
研究者
PD Dr. Peter Nordbeck
Head Consultant Cardiology and Chief of Electrophysiology
Wuerzburg University Hospital