Ignite Creation Date:
2025-12-25 @ 12:31 AM
Ignite Modification Date:
2025-12-25 @ 10:38 PM
Study NCT ID:
NCT06906367
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-08
First Post:
2025-03-26
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of Patients With Fabry Disease (US Specific)
Sponsor:
Amicus Therapeutics
Organization:
Study Overview
Official Title:
A Prospective, Observational Study of Patients With Fabry Disease (US Specific)
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-03
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:
This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.
Detailed Description:
This is a prospective, multicenter, observational, effectiveness, safety, and outcomes study enrolling at least 450 patients with Fabry disease globally (at least 250 patients in the migalastat-treated group, approximately 100 patients in the ERT-treated group, and approximately 100 patients in the untreated group \[patients who have never been on treatment for Fabry disease\]). Enrollment will continue for a period of 5 years and all patients will be followed for up to 5 years after their enrollment.
Disclaimer: This is a global study, the country level requirements may vary from site to site. The requirements noted in this posting are specific to the US.
Disclaimer: This is a global study, the country level requirements may vary from site to site. The requirements noted in this posting are specific to the US.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
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?: