Clinical trial NCT04281537: A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Fabry Disease
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2020-02-24
Enrollment: 82
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with FD who are receiving ERT and their caregivers will be identified and recruited from approximately 12 specialist centres in four countries (Taiwan, Turkey, Brazil and Japan).

Brief summary: 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.


Eligibility criteria: 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.

Patient Exclusion Criteria:

* 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.

Primary outcomes:
measure: Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease
timeFrame: up to 7 weeks
Secondary outcomes:
measure: 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).
timeFrame: up to 7 weeks
measure: Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).
timeFrame: up to 7 weeks
measure: Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil.
timeFrame: up to 7 weeks
measure: Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan.
timeFrame: up to 7 weeks
measure: Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan.
timeFrame: up to 7 weeks
measure: Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey
timeFrame: up to 7 weeks
measure: Percentage of Patients With Work Absence Due to Attendance for This ERT Episode.
timeFrame: up to 7 weeks
measure: Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.
timeFrame: up to 7 weeks
measure: Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
timeFrame: up to 7 weeks
measure: Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
timeFrame: up to 7 weeks
measure: Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration.
timeFrame: up to 7 weeks
measure: Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.
timeFrame: up to 7 weeks
measure: Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
timeFrame: HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic
measure: Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
timeFrame: WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic
measure: Patients Level of Fatigue Measured by Fatigue Likert Scale
timeFrame: 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
measure: Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
timeFrame: WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion
measure: Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
timeFrame: WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion)
measure: Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
timeFrame: WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion
measure: Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
timeFrame: WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion)
measure: Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses).
timeFrame: up to 7 weeks
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Amicus Therapeutics, Inc.
state: Pennsylvania
city: São Paulo
country: Brazil
facility: Instituto de Genética e Erros Inatos do Metabolismo (IGEIM)
city: Tokyo
country: Japan
facility: Keio University Hospital
city: Yokohama
country: Japan
facility: Yokohama Municipal Citizen's Hospital
city: Taipei City
country: Taiwan
facility: Taipei Veterans General Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University
city: Ankara
country: Turkey
facility: Gazi University Hospital
city: İzmir
country: Turkey
facility: Dokuz Eylul University Medical Faculty
city: İzmir
country: Turkey
facility: Ege University Hospital
Study officials:
References: