Trial · NCT04281537
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
NCT04281537, Completed, Amicus Therapeutics; results first posted 2024-11-04
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. Trials, record NCT04281537. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04281537
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
- NCT number
- NCT04281537
- Status
- Completed
- Enrollment
- 82 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Fabry Disease
- Interventions
- Enzyme Replacement Therapy (ERT)
- First posted
- 2020-02-24
- Last updated
- 2026-02-05
- Results first posted
- 2024-11-04
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease | 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 |
| 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 | 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). |
| 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 | 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. |
| Secondary | 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 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. |
| Secondary | 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 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. |
| Secondary | 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 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. |
| Secondary | 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 | 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. |
| Secondary | Percentage of Patients With Work Absence Due to Attendance for This ERT Episode. | up to 7 weeks | The percentage of patients known to have taken time off from work during their ERT episode |
| Secondary | Number of Hours of Patient Work Absence Due to Attendance for ERT Episode. | up to 7 weeks | The number of paid and unpaid hours that patients were absent from work during their ERT episode |
| Secondary | 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 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). |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease | up to 7 weeks | 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. | minutes | MEAN |
| 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 | 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. | minutes | MEAN |
| 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 | 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. | minutes | MEAN |
| SECONDARY | 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 | 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. | R$ | MEAN |
| SECONDARY | 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 | 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. | NT$ | MEAN |
| SECONDARY | 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 | 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. | ¥ | MEAN |
| SECONDARY | 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 | 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. | ₺ | MEAN |
| SECONDARY | Percentage of Patients With Work Absence Due to Attendance for This ERT Episode. | up to 7 weeks | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Hours of Patient Work Absence Due to Attendance for ERT Episode. | up to 7 weeks | 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. | hours | MEAN |
| SECONDARY | Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT | up to 7 weeks | minutes | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group51
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease | Patients With Fabry Disease on ERT | 151.9 | Standard Deviation 62.5 | minutes | |
| 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). | Patients With Fabry Disease on ERT | Time Spent by HCP on Pre-infusion activities | 20.9 | Standard Deviation 13.4 | minutes |
| 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). | Patients With Fabry Disease on ERT | Time spent by HCP on ERT administration | 118.2 | Standard Deviation 56.3 | minutes |
| 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). | Patients With Fabry Disease on ERT | Time spent by HCP on post-infusion activities | 12.8 | Standard Deviation 9.6 | minutes |
| Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | Patients With Fabry Disease on ERT | 364.1 | Standard Deviation 193.9 | minutes | |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil. | Patients With Fabry Disease on ERT | 166.5 | Standard Deviation 214.8 | R$ | |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan. | Patients With Fabry Disease on ERT | 709.5 | Standard Deviation 855.9 | NT$ | |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan. | Patients With Fabry Disease on ERT | 8781.0 | Standard Deviation 15956.6 | ¥ | |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey | Patients With Fabry Disease on ERT | 470.8 | Standard Deviation 1005.5 | ₺ | |
| Percentage of Patients With Work Absence Due to Attendance for This ERT Episode. | Patients With Fabry Disease on ERT | 16 | Participants |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Patients With Fabry Disease on ERT | 76 | |
| Overall Study | STARTED | Caregiver | 6 | |
| Overall Study | COMPLETED | Patients With Fabry Disease on ERT | 76 | |
| Overall Study | COMPLETED | Caregiver | 6 | |
| Overall Study | NOT COMPLETED | Patients With Fabry Disease on ERT | 0 | |
| Overall Study | NOT COMPLETED | Caregiver | 0 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patient With Fabry Disease on ERT | 0 | 0 |
| Total, all-cause mortality | Caregiver | 0 | 0 |
| Total, other adverse events | Patient With Fabry Disease on ERT | 0 | 0 |
| Total, other adverse events | Caregiver | 0 | 0 |
| Total, serious adverse events | Patient With Fabry Disease on ERT | 0 | 0 |
| Total, serious adverse events | Caregiver | 0 | 0 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40790487 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04281537
GET https://conductscience.org/api/trials/v1/NCT04281537/registered?page=2