N/A
N=82
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
Fabry Disease
Bottom Line
View on ClinicalTrials.gov: NCT04281537 ↗Enrolled (actual)
82
Serious AEs
—
Results posted
Nov 2024
Primary outcome: Primary: Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease — 151.9 minutes
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Enzyme Replacement Therapy (ERT) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amicus Therapeutics
- Primary completion
- May 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease |
151.9 | — |
| 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). |
20.9; 118.2; 12.8 | — |
| SECONDARY Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). |
364.1 | — |
| SECONDARY Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil. |
166.5 | — |
| SECONDARY Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan. |
709.5 | — |
| SECONDARY Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan. |
8781.0 | — |
| SECONDARY Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey |
470.8 | — |
| SECONDARY Percentage of Patients With Work Absence Due to Attendance for This ERT Episode. |
16 | — |
| SECONDARY Number of Hours of Patient Work Absence Due to Attendance for ERT Episode. |
6.6; 5.3 | — |
| SECONDARY Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT |
160 | — |
| SECONDARY Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT |
2085 | — |
| SECONDARY Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration. |
3 | — |
| SECONDARY Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration. |
6.0; 3.0 | — |
| SECONDARY Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) |
2.9; 2.2; 3.3; 3.6; 3.6; 3.5 | — |
| SECONDARY Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit |
3.1; 3.2; 2.8; 2.6; 3.4; 15.1 | — |
| SECONDARY Patients Level of Fatigue Measured by Fatigue Likert Scale |
2.4; -0.2; 0.1 | — |
| SECONDARY Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion |
5.0; 28.6; 28.9; 30.8 | — |
| SECONDARY Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion |
3.7; 22.6; 25.7; 32.3 | — |
| SECONDARY Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion. |
0; 17.5; 17.5; 18.3 | — |
| SECONDARY Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion. |
5.0; 17.5; 21.0; 16.7 | — |
| SECONDARY Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses). |
4.0 | — |
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.
Data sourced from ClinicalTrials.gov (NCT04281537). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.