Ignite Creation Date:
2025-12-24 @ 4:03 PM
Ignite Modification Date:
2026-01-02 @ 1:54 PM
Study NCT ID:
NCT04804566
Status:
COMPLETED
Last Update Posted:
2024-04-03
First Post:
2021-03-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
Sponsor:
Amicus Therapeutics
Organization:
Study Overview
Official Title:
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
Status:
COMPLETED
Status Verified Date:
2024-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: