A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04281537
• Other study ID #: AT-NIS-0001
• Status: Completed
• Start date: March 1, 2020
• Est. completion date: May 18, 2022

Study information

• Verified date: April 2023
• Source: Amicus Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study will comprise a prospective time and motion evaluation and a cross-sectional evaluation of patient and caregiver-reported outcomes. The study will evaluate the time associated with the preparation and administration of a single dose of 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 will also evaluate the patients' quality of life wellbeing, fatigue and work productivity.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 76
• Est. completion date: May 18, 2022
• Est. primary completion date: May 18, 2022
• Gender: All
• Age group: 18 Years and older

Eligibility

Patient Inclusion Criteria:

• Patients with a documented diagnosis of 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 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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• Agalsidase Beta
Enzyme Replacement Therapy - Infusion every other week
• Agalsidase Alpha
Enzyme Replacement Therapy - Infusion every other week

Locations

Country	Name	City	State
Brazil	Instituto de Genética e Erros Inatos do Metabolismo (IGEIM)	São Paulo
Japan	Keio University Hospital	Tokyo
Japan	Yokohama Municipal Citizen's Hospital	Yokohama
Taiwan	National Taiwan University	Taipei
Taiwan	Taipei Veterans General Hospital	Taipei City
Turkey	Gazi University Hospital	Ankara
Turkey	Dokuz Eylul University Medical Faculty	Izmir
Turkey	Ege University Hospital	Izmir
United States	Amicus Therapeutics, Inc.	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Brazil,  Japan,  Taiwan,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total time spent by HCPs in the preparation and administration of a single dose of ERT in patients with FD; stratified by country and by ERT product (agalsidase alfa or agalsidase beta).		up to 7 weeks
Secondary	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
Secondary	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
Secondary	Total costs associated with attendance for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).		up to 7 weeks
Secondary	Proportion of patients with work absence due to attendance for this ERT episode.		up to 7 weeks
Secondary	Number of hours of patient work absence due to attendance for this ERT episode.		up to 7 weeks
Secondary	Total caregiver time associated with accompanying a patient with FD for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).		up to 7 weeks
Secondary	Total costs associated with accompanying a patient with FD for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).		up to 7 weeks
Secondary	Proportion of caregivers with work absence due to accompanying the patient for this ERT episode.		up to 7 weeks
Secondary	Number of hours of caregivers work absence due to accompanying the patient for this ERT episode.		up to 7 weeks
Secondary	HRQoL (SF-12 scores / responses)	Health Related Quality of Life measured by the 12 Item Short Form Survey [SF-12]). SF-12v2 component summary measure and health domain scale is described in terms of scale or item composition, number of score levels, lowest and highest possible T scores for the standard and acute forms, and the health states associated with the lowest and highest observable scores. These descriptions are based on the general content of the scales and measures and the pattern of responses necessary to achieve these extreme scores. This information can be used to summarize what each component summary measure and health domain scale measures and can serve as a basis for broad-level interpretation of SF-12v2 results.; These 8 domains form 2 subscales: physical component summary and mental component summary. The low scores indicate limitations in the domain measured. The high scores indicate less limitations in the domain measured.	up to 7 weeks
Secondary	Patients General wellbeing measured by WHO-5 scores / responses)	World Health Organization-5 Wellbeing Index [WHO-5] consists of 5 items and the timeframe for responses is based on the previous 2 weeks. The 5 items relate to feeling cheerful, calm, active, rested and being interested in life. The raw score is calculated by totaling the figures of the five answers. The raw score ranges from 0 to 25, 0 representing worst possible and 25 representing best possible quality of life.	up to 7 weeks
Secondary	Patients Level of fatigue measured by Fatigue Likert scale)	Questionnaire measuring Fatigue. The range is from 1-5. 1 not at all tired, 5 extremely tired	up to 7 weeks
Secondary	Patients Levels of work impairment (WPAI scores / responses)	Work Productivity and Activity Index. The WPAI consists of 6 items and ranges from 0-100%. WPAI outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity, i.e., worse outcomes.	up to 7 weeks
Secondary	Caregiver's Levels of work impairment (WPAI-CG scores / responses).	Work Productivity and Activity Index. The WPAI consists of 6 items and ranges from 0-100%. WPAI outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity, i.e., worse outcomes.	up to 7 weeks
Secondary	Level of strain in providing care for a patient with FD (CSI scores / responses).	Caregiver Strain index. The CSI consists of 13 items and measures the strain of care provision in five domains (financial, physical, psychological, social and personal). The range is from 0-13 and any positive answer may indicate a need for intervention in that area. A score of 7 or higher indicates a high level of stress.	up to 7 weeks