NCT04281537已完成不适用
A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase Beta
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
- 入组人数
- 82
- 试验地点
- 9
- 主要终点
- Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease
研究概览
简要总结
This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study comprised a prospective time and motion evaluation and a cross-sectional evaluation of patient and caregiver-reported outcomes. The study evaluated the time associated with the preparation and administration of a single dose of enzyme replacement therapy (ERT) in patients by health care providers as well as the impact on Fabry patients and caregivers time and costs associated with an ERT treatment. The study also evaluated the patients' quality of life wellbeing, fatigue and work productivity.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
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
- •Patient Inclusion Criteria:
- •Patients with a documented diagnosis of Fabry Disease (FD)
- •Patients who have received ≥4 doses of ERT (with agalsidase alfa or agalsidase beta) for the treatment of FD.
- •Patients who present to the participating hospital(s) or treatment centre(s) for administration of a dose of ERT (as part of their routine treatment) during the data collection period.
- •Caregiver Inclusion Criteria:
- •Self-identifies as a caregiver of a patient with FD for whom written informed consent has been obtained for inclusion in the study.
排除标准
- •Patients who are unable or unwilling to give consent for study participation.
- •Patients whose ERT preparation and administration takes place exclusively in the home setting with no healthcare professional (HCP) involvement in preparation of the infusion.
- •For the time and motion evaluation: Patients whose ERT is administered by a HCP who does not consent to be observed.
- •Caregiver Exclusion Criteria:
- •- Caregiver (and/or the patient with FD whom they support or care for) is unable or unwilling to give consent for study participation.
研究组 & 干预措施
Patient with Fabry Disease on Enzyme Replacement Therapy (ERT)
Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta)
干预措施: Enzyme Replacement Therapy (ERT) (Drug)
Caregiver
Caregiver of patient with Fabry Disease on Enzyme Replacement Therapy (ERT)
结局指标
主要结局
Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease
时间窗: up to 7 weeks
Total time (minutes) spent by HCPs on all activities related to administration of a single dose of ERT in patients with Fabry Disease including pre-infusion (pre-administration patient consultation; infusion and premedication preparation), ERT (and premedication) administration, and post-administration assessment and documentation. Results are shown for all patients with Fabry Disease on ERT in study
次要结局
- Total Time Spent by HCPs on Each Separate Task Associated With the Preparation and Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).(up to 7 weeks)
- Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).(up to 7 weeks)
- Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil.(up to 7 weeks)
- Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan.(up to 7 weeks)
- Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan.(up to 7 weeks)
- Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey(up to 7 weeks)
- Percentage of Patients With Work Absence Due to Attendance for This ERT Episode.(up to 7 weeks)
- Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.(up to 7 weeks)
- Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT(up to 7 weeks)
- Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT(up to 7 weeks)
- Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration.(up to 7 weeks)
- Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.(up to 7 weeks)
- Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)(HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic)
- Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit(WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic)
- Patients Level of Fatigue Measured by Fatigue Likert Scale(Questionnaire completed during ERT infusion (baseline), the same evening as date of ERT infusion, and once during the period of 1 to 7 days post-infusion)
- Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion(WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion)
- Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion(WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion))
- Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.(WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion)
- Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.(WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion))
- Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses).(up to 7 weeks)
研究者
研究点 (9)
Loading locations...
相似试验
已完成
不适用
Study to Describe the Real-world Treatment Patterns and Associated Outcomes in Patients With HER2-positive Unresectable or Metastatic Breast CancerMetastatic Breast CancerNCT04857619AstraZeneca763
已完成
不适用
A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As ChildrenQuality of LifeCardiac EventRenal InsufficiencyNCT04002531Baylor Research Institute5
招募中
不适用
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingFabry DiseaseNCT04252066Amicus Therapeutics20
招募中
不适用
Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular HypertrophyLeft Ventricular HypertrophyHypertrophic CardiomyopathyFabry DiseaseNCT06169358Qilu Hospital of Shandong University627
已完成
不适用
Registry of Fabry Disease - A Multicenter Observational StudyFabry DiseaseNCT00055016National Institute of Neurological Disorders and Stroke (NINDS)100