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NCT04281537 · ClinicalTrials.gov registry record
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
A clinical trial, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- 82
- Enrollment target
NCT04281537: Completed study, sponsored by Amicus Therapeutics.
NCT04281537 is a clinical trial that has completed, run by Amicus Therapeutics. The registered enrollment target is 82 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04281537 has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- 82 participants
- Enrollment target
Study 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.
Primary Outcome
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
Interventions
- DRUG Enzyme Replacement Therapy (ERT)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 82 participants |
| Start Date | 2020-03-01 |
| Est. Completion | 2022-05-18 |
What the finished NCT04281537 record still lists
NCT04281537 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 82 participants, a relatively small participant target.
The record links to 0 conditions, and to 1 intervention - of which Enzyme Replacement Therapy (ERT) is the first listed.
NCT04281537 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04281537 about?
NCT04281537 is a clinical study titled "A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.". 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 wit...
What is the current status of trial NCT04281537?
This trial is currently completed. The enrollment target is 82 participants. The study started on 2020-03-01. Estimated completion is 2022-05-18.
What interventions are being tested in trial NCT04281537?
The interventions under investigation include: Enzyme Replacement Therapy (ERT) (DRUG).
Who is sponsoring clinical trial NCT04281537?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
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