Trials / Completed
CompletedNCT04804566
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 44 (actual)
- Sponsor
- Amicus Therapeutics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.
Detailed description
This will be a cross-sectional study conducted in approximately 130 individuals (or representative parents/caregivers of patients) living with Fabry disease. All study participants will complete the RSVP followed by a structured interview conducted by trained interviewers. It is estimated that each respondent will need up to 60 minutes for the entire process; 10 minutes to complete the RSVP including uploading the proof of Fabry disease diagnosis or verifying membership with Fabry groups, including but not limited to: Fabry Support and Information Group, National Fabry Disease Foundation, MPS Society UK, Morbus Fabry Selbsthilfergruppe, Fabry International Network, or others, and approximately 50 minutes to complete the interview.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | This is a non-interventional study | This is a non-interventional study |
Timeline
- Start date
- 2023-03-01
- Primary completion
- 2023-11-01
- Completion
- 2023-11-01
- First posted
- 2021-03-18
- Last updated
- 2024-04-03
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT04804566. Inclusion in this directory is not an endorsement.