Trial Outcomes & Findings for A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. (NCT NCT04281537)
NCT ID: NCT04281537
Last Updated: 2026-02-05
Results Overview
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
COMPLETED
82 participants
up to 7 weeks
2026-02-05
Participant Flow
76 patients with Fabry Disease on enzyme replacement therapy (ERT) and 6 caregivers of patients with Fabry Disease on ERT were enrolled into the study per protocol for a total of 82 participants. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Participant milestones
| Measure |
Patients With Fabry Disease on ERT
Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week
|
Caregiver
Caregiver of patient with Fabry Disease on ERT
|
|---|---|---|
|
Overall Study
STARTED
|
76
|
6
|
|
Overall Study
COMPLETED
|
76
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Age of caregivers was not reported in same manner as for patients with Fabry Disease. Caregiver ages reported as counts within a range: age 31-40 (n=2), 41-50 (n=3), and 61-70 (n=1)
Baseline characteristics by cohort
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week
|
Caregiver
n=6 Participants
Caregiver of patient with Fabry Disease on ERT
|
Total
n=82 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
48.9 years
n=76 Participants • Age of caregivers was not reported in same manner as for patients with Fabry Disease. Caregiver ages reported as counts within a range: age 31-40 (n=2), 41-50 (n=3), and 61-70 (n=1)
|
—
|
48.9 years
n=76 Participants • Age of caregivers was not reported in same manner as for patients with Fabry Disease. Caregiver ages reported as counts within a range: age 31-40 (n=2), 41-50 (n=3), and 61-70 (n=1)
|
|
Age, Customized
Caregiver age range · 31 to 40 years
|
0 Participants
Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
2 Participants
n=6 Participants • Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
2 Participants
n=6 Participants • Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
|
Age, Customized
Caregiver age range · 41 to 50 years
|
0 Participants
Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
3 Participants
n=6 Participants • Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
3 Participants
n=6 Participants • Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
|
Age, Customized
Caregiver age range · 61 to 70 years
|
0 Participants
Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
1 Participants
n=6 Participants • Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
1 Participants
n=6 Participants • Caregiver ages were reported as a count within a range. Data for ages of patients with Fabry Disease on ERT are reported separately on a per patient basis
|
|
Sex: Female, Male
Female
|
31 Participants
n=76 Participants
|
5 Participants
n=6 Participants
|
36 Participants
n=82 Participants
|
|
Sex: Female, Male
Male
|
45 Participants
n=76 Participants
|
1 Participants
n=6 Participants
|
46 Participants
n=82 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
Turkey
|
19 participants
n=76 Participants
|
1 participants
n=6 Participants
|
20 participants
n=82 Participants
|
|
Region of Enrollment
Japan
|
4 participants
n=76 Participants
|
0 participants
n=6 Participants
|
4 participants
n=82 Participants
|
|
Region of Enrollment
Taiwan
|
30 participants
n=76 Participants
|
5 participants
n=6 Participants
|
35 participants
n=82 Participants
|
|
Region of Enrollment
Brazil
|
23 participants
n=76 Participants
|
0 participants
n=6 Participants
|
23 participants
n=82 Participants
|
|
ERT Administered to Patients
Agalsidase alfa/biosimilar
|
28 Participants
n=76 Participants • Type of ERT is not applicable to caregiver group
|
0 Participants
Type of ERT is not applicable to caregiver group
|
28 Participants
n=76 Participants • Type of ERT is not applicable to caregiver group
|
|
ERT Administered to Patients
Agalsidase beta/biosimilar
|
48 Participants
n=76 Participants • Type of ERT is not applicable to caregiver group
|
0 Participants
Type of ERT is not applicable to caregiver group
|
48 Participants
n=76 Participants • Type of ERT is not applicable to caregiver group
|
PRIMARY outcome
Timeframe: up to 7 weeksPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
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
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
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
Standard Deviation 62.5
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Total time spent (in minutes) by healthcare professionals (HCP) on tasks associated with preparation and administration of a single dose of ERT (with agalsidase alfa or agalsidase beta) in patients with Fabry Disease including pre-administration activities (patient consultation, infusion/pre-medication preparation activities), administration of ERT (and pre-medications), and post-infusion activities (monitoring of patient during and after administration, completion of clinical documentation, and any other ERT-administration related activities).
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
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).
Time Spent by HCP on Pre-infusion activities
|
20.9 minutes
Standard Deviation 13.4
|
|
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).
Time spent by HCP on ERT administration
|
118.2 minutes
Standard Deviation 56.3
|
|
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).
Time spent by HCP on post-infusion activities
|
12.8 minutes
Standard Deviation 9.6
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. Data were not available for 1 patient.
Total patient time associated with attendance for administration of a single dose of ERT includes the total time from when the patient left home, to when they returned home and based on the reported time spent travelling, total waiting time, the total time spent during infusion episode (pre, during and post) and the other recorded activities.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=75 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).
|
364.1 minutes
Standard Deviation 193.9
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: Patients with Fabry Disease in Brazil. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. Data were not available for 2 patients.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Brazil that were directly related to attendance for the administration of a single dose of ERT.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=21 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil.
|
166.5 R$
Standard Deviation 214.8
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: Patients with Fabry Disease in Taiwan. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. Data were not available for 1 patient.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Taiwan that were directly related to attendance for the administration of a single dose of ERT.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=29 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan.
|
709.5 NT$
Standard Deviation 855.9
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: Patients with Fabry Disease in Japan. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Japan that were directly related to attendance for the administration of a single dose of ERT.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=4 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan.
|
8781.0 ¥
Standard Deviation 15956.6
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: Patients with Fabry Disease in Turkey. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. Data were not available for 1 patient.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Turkey that were directly related to attendance for the administration of a single dose of ERT.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=18 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey
|
470.8 ₺
Standard Deviation 1005.5
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. Note: data were missing for 54% \[41/76\] of patients.
The percentage of patients known to have taken time off from work during their ERT episode
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=35 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Percentage of Patients With Work Absence Due to Attendance for This ERT Episode.
|
16 Participants
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. Note: data were missing for 54% \[41/76\] of patients.
The number of paid and unpaid hours that patients were absent from work during their ERT episode
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=35 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.
Number of Paid Hours Absent from Work
|
6.6 hours
Standard Deviation 5.4
|
|
Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.
Number of Unpaid Hours Absent from Work
|
5.3 hours
Standard Deviation 5.4
|
SECONDARY outcome
Timeframe: up to 7 weeksTotal caregiver time (in minutes) associated with accompanying a patient with Fabry Disease for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=6 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
|
160 minutes
Standard Deviation 70.43
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: Data presented are from Taiwan in New Taiwan dollars (NT$). No data were reported for the 1 caregiver in Turkey for this outcome measure.
Total costs associated with accompanying a patient with Fabry Disease for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=5 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
|
2085 NT$
Standard Deviation 1728.6
|
SECONDARY outcome
Timeframe: up to 7 weeksPercentage of caregivers with work absence due to accompanying the patient for this episode of ERT administration.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=6 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration.
|
3 Participants
|
SECONDARY outcome
Timeframe: up to 7 weeksPopulation: Data are available from 3 caregivers in Taiwan.
Number of hours of caregivers work absence due to accompanying the patient for this ERT administration episode.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=3 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.
Number of Paid Hours Absent from Work
|
6.0 hours
Interval 6.0 to 6.0
|
|
Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.
Number of Unpaid Hours Absent from Work
|
3.0 hours
Interval 1.5 to 4.5
|
SECONDARY outcome
Timeframe: HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinicPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately. The HRQoL was completed only one time per patient.
Health Related Quality of Life (HRQoL) measured by the 12 Item Short Form Survey \[SF-12\]). In the SF-12, patients were asked to rate aspects of their health across 8 domains; all domains have a maximum value of 5 (range 0-5) except physical functioning, for which the maximum score is 3 (range 0-3). Higher scores indicate better HRQoL while low scores indicate worse HRQoL.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
General Health
|
2.9 score on a scale
Standard Deviation 1.1
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Physical Functioning
|
2.2 score on a scale
Standard Deviation 0.7
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Role Physical
|
3.3 score on a scale
Standard Deviation 1.1
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Role Emotional
|
3.6 score on a scale
Standard Deviation 1.1
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Bodily Pain
|
3.6 score on a scale
Standard Deviation 1.2
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Mental Health
|
3.5 score on a scale
Standard Deviation 0.9
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Vitality
|
3.1 score on a scale
Standard Deviation 1.1
|
|
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Social Function
|
3.6 score on a scale
Standard Deviation 1.2
|
SECONDARY outcome
Timeframe: WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinicPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
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. For each question, patients rated the extent to which the statement applies between 0 (none of the time) and 5 (all of the time), with higher scores indicating better wellbeing. The WHO-5 total score is calculated by totaling the values of each of the five questions. The total scores range from 0 to 25, where 0 represents the worst possible quality of life and 25 represents the best possible quality of life.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
Item 1: felt cheerful and in good spirts
|
3.1 score on a scale
Standard Deviation 1.3
|
|
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
Item 2: felt calm and relaxed
|
3.2 score on a scale
Standard Deviation 1.3
|
|
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
Item 3: felt active and vigorous
|
2.8 score on a scale
Standard Deviation 1.4
|
|
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
Item 4: wake up feeling fresh and restful
|
2.6 score on a scale
Standard Deviation 1.5
|
|
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
Item 5: daily life filled with things that interest me
|
3.4 score on a scale
Standard Deviation 1.2
|
|
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
WHO-5 Total Score
|
15.1 score on a scale
Standard Deviation 5.6
|
SECONDARY outcome
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-infusionPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Questionnaire measuring Fatigue. Possible scores range from 1 (not at all tired) to 5 (extremely tired); higher scores therefore indicating worse fatigue. Negative value in change from during ERT infusion to evening of ERT infusion indicates more fatigue in the evening of infusion. Negative value in change from baseline (during ERT infusion) to 1-7 days after ERT infusion indicates more fatigue during the period occurring 1-7 days after ERT infusion.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Patients Level of Fatigue Measured by Fatigue Likert Scale
Level of fatigue during infusion
|
2.4 score on a scale
Standard Deviation 1.4
|
|
Patients Level of Fatigue Measured by Fatigue Likert Scale
Change in level of fatigue from during ERT infusion to evening of ERT infusion
|
-0.2 score on a scale
Standard Deviation 1.4
|
|
Patients Level of Fatigue Measured by Fatigue Likert Scale
Change in level of fatigue from during ERT infusion to 1-7 days after ERT infusion
|
0.1 score on a scale
Standard Deviation 1.3
|
SECONDARY outcome
Timeframe: WPAI completed once by patients during the period of 1 to 7 Days After ERT InfusionPopulation: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected 1 to 7 days after ERT infusion.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
% Activity Impairment Due to Health
|
30.8 Percent
Standard Deviation 29.3
|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
% Work Time Missed Due to Health (Absenteeism)
|
5.0 Percent
Standard Deviation 9.5
|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
% Impairment While Working Due to Health (Presenteeism)
|
28.6 Percent
Standard Deviation 29.5
|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
% Overall Work Impairment Due to Health
|
28.9 Percent
Standard Deviation 27.7
|
SECONDARY outcome
Timeframe: WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion)Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected on day of next ERT infusion.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=76 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
% Work Time Missed Due to Health (Absenteeism)
|
3.7 Percent
Standard Deviation 7.3
|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
% Impairment While Working Due to Health (Presenteeism)
|
22.6 Percent
Standard Deviation 24.9
|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
% Overall Work Impairment Due to Health
|
25.7 Percent
Standard Deviation 26.4
|
|
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
% Activity Impairment Due to Health
|
32.3 Percent
Standard Deviation 30.4
|
SECONDARY outcome
Timeframe: WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusionWork Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected 1 to 7 days after ERT infusion.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=6 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
% Work Time Missed Due to Health (Absenteeism)
|
0 Percent
Standard Deviation 0
|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
% Impairment While Working Due to Health (Presenteeism)
|
17.5 Percent
Standard Deviation 20.6
|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
% Overall Work Impairment Due to Health
|
17.5 Percent
Standard Deviation 20.6
|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
% Activity Impairment Due to Health
|
18.3 Percent
Standard Deviation 18.3
|
SECONDARY outcome
Timeframe: WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion)Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). WPAI was completed by caregiver on day of next ERT infusion.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=6 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
% Work Time Missed Due to Health (Absenteeism)
|
5.0 Percent
Standard Deviation 10.0
|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
% Impairment While Working Due to Health (Presenteeism)
|
17.5 Percent
Standard Deviation 20.6
|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
% Overall Work Impairment Due to Health
|
21.0 Percent
Standard Deviation 24.3
|
|
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
% Activity Impairment Due to Health
|
16.7 Percent
Standard Deviation 18.6
|
SECONDARY outcome
Timeframe: up to 7 weeksThe Caregiver Strain Index (CSI) consists of 12 questions with Yes/No answer and measures the strain of care provision in five domains (financial, physical, psychological, social and personal). Yes = 1 and No = 0. The range is from 0-12. Total scores are the sum of all responses. A score of 7 or higher indicates a high level of stress.
Outcome measures
| Measure |
Patients With Fabry Disease on ERT
n=6 Participants
Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week
|
|---|---|
|
Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses).
|
4.0 score on a scale
Standard Deviation 2.3
|
Adverse Events
Patient With Fabry Disease on ERT
Caregiver
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place