Fabry DiseaseNCT04281537
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
An observational study of Fabry Disease, run by Amicus Therapeutics at 9 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Enzyme Replacement Therapy (ERT)
- Type of treatment
- Injection or infusion
- How many people
- 82 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 1 Mar 2020to 18 May 2022
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
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).
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.
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
Turkey (Türkiye)3 sites
Gazi University Hospital
Ankara
Dokuz Eylul University Medical Faculty
Izmir
Ege University Hospital
Izmir
Japan2 sites
Keio University Hospital
Tokyo
Yokohama Municipal Citizen's Hospital
Yokohama
Taiwan2 sites
National Taiwan University
Taipei
Taipei Veterans General Hospital
Taipei
United States1 site
Amicus Therapeutics, Inc.
Philadelphia, Pennsylvania, 19104
Brazil1 site
Instituto de Genética e Erros Inatos do Metabolismo (IGEIM)
São Paulo
About this study
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
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.
- Conditions
- Fabry Disease
- Being tested
- Enzyme Replacement Therapy (ERT)
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Enzyme Replacement Therapy (ERT)Drug
Also called agalsidase alpha, agalsidase beta
ERT infusion every other week
| Study group | Role | What they receive |
|---|---|---|
| Patient with Fabry Disease on Enzyme Replacement Therapy (ERT) | Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) Receives: Drug: Enzyme Replacement Therapy (ERT) | |
| Caregiver | Caregiver of patient with Fabry Disease on Enzyme Replacement Therapy (ERT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease
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
Measured: up to 7 weeks
Secondary outcomes (20)
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).
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).
Measured: up to 7 weeks
Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).
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.
Measured: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil.
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.
Measured: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan.
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.
Measured: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan.
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.
Measured: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey
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.
Measured: up to 7 weeks
Percentage of Patients With Work Absence Due to Attendance for This ERT Episode.
The percentage of patients known to have taken time off from work during their ERT episode
Measured: up to 7 weeks
Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.
The number of paid and unpaid hours that patients were absent from work during their ERT episode
Measured: up to 7 weeks
Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
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).
Measured: up to 7 weeks
Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
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).
Measured: up to 7 weeks
Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration.
Percentage of caregivers with work absence due to accompanying the patient for this episode of ERT administration.
Measured: up to 7 weeks
Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.
Number of hours of caregivers work absence due to accompanying the patient for this ERT administration episode.
Measured: up to 7 weeks
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
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.
Measured: HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
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.
Measured: WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic
Patients Level of Fatigue Measured by Fatigue Likert Scale
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.
Measured: 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
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT 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). Data were collected 1 to 7 days after ERT infusion.
Measured: WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT 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). Data were collected on day of next ERT infusion.
Measured: WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion)
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT 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). Data were collected 1 to 7 days after ERT infusion.
Measured: WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT 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.
Measured: WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion)
Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses).
The 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.
Measured: up to 7 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2020
- Study start1 Mar 2020
- Primary completion18 May 2022
- Study completion18 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 82 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04281537
- Sponsor's protocol ID
- AT-NIS-0001
- Status last verified
- Jan 2026
- First submitted
- 7 Feb 2020
- Last updated
- 5 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Keyzor I, Martins AM, Ucar SK, Yamakawa H, Chien YH, Arslan N, Niu DM, Tumer L, Baldock L, Shohet S, Giuliano JD. A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta. Orphanet J Rare Dis. 2025 Aug 11;20(1):419. doi: 10.1186/s13023-025-03707-2.
PubMed 40790487
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04281537
- Acronym / sponsor ID
- AT-NIS-0001
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 82 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Mar 2020 (actual)
- Primary completion
- 18 May 2022 (actual)
- Study completion
- 18 May 2022 (actual)
- First posted
- 24 Feb 2020 (actual)
- Results
- Posted 4 Nov 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patient with Fabry Disease on Enzyme Replacement Therapy (ERT) | – | Drug: Enzyme Replacement Therapy (ERT)Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) |
| Caregiver | – | –Caregiver of patient with Fabry Disease on Enzyme Replacement Therapy (ERT) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Enzyme Replacement Therapy (ERT)Also: agalsidase alpha, agalsidase betaERT infusion every other week | Drug | Patient with Fabry Disease on Enzyme Replacement Therapy (ERT) |
Primary outcome
Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease
Time frame: 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
20 secondary outcomes
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 frame: up to 7 weeks
Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).
Time frame: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil.
Time frame: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan.
Time frame: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan.
Time frame: up to 7 weeks
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey
Time frame: up to 7 weeks
Percentage of Patients With Work Absence Due to Attendance for This ERT Episode.
Time frame: up to 7 weeks
Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.
Time frame: up to 7 weeks
Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
Time frame: up to 7 weeks
Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
Time frame: up to 7 weeks
Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration.
Time frame: up to 7 weeks
Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.
Time frame: up to 7 weeks
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Time frame: HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
Time frame: WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic
Patients Level of Fatigue Measured by Fatigue Likert Scale
Time frame: 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
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
Time frame: WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
Time frame: WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion)
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
Time frame: WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
Time frame: WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion)
Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses).
Time frame: up to 7 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40790487Keyzor I, Martins AM, Ucar SK, Yamakawa H, Chien YH, Arslan N, Niu DM, Tumer L, Baldock L, Shohet S, Giuliano JD. A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta. Orphanet J Rare Dis. 2025 Aug 11;20(1):419. doi: 10.1186/s13023-025-03707-2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Amicus Therapeutics at 9 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Turkey (Türkiye), Japan, Taiwan, United States, Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Nov 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease (time frame: up to 7 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04281537, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2026.