Ignite Creation Date:
2025-12-24 @ 5:23 PM
Ignite Modification Date:
2025-12-24 @ 5:23 PM
Study NCT ID:
NCT06880250
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-03-17
First Post:
2025-03-11
Is Possible Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy and Safety of Enzyme Replacement Therapy in Patients with Fabry Disease
Sponsor:
NPO Petrovax
Organization:
Study Overview
Official Title:
Open-label Comparative Cohort Non-interventional Epidemiological Study of the Efficacy and Safety of Enzyme Replacement Therapy in Russian Patients with Fabry Disease
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:
FAVORIT
Brief Summary:
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is:
What medical problems do participants have?
Participants already on enzyme replacement therapy as part of their regular medical care for Fabry disease will be observed as usual. Their data will be collected by their doctor.
What medical problems do participants have?
Participants already on enzyme replacement therapy as part of their regular medical care for Fabry disease will be observed as usual. Their data will be collected by their doctor.
Detailed Description:
All patients on enzyme replacement therapy could be enrolled in the study. Three medicines are currently available in Russia (Fabagal®, Fabrazyme®, Replagal®) so the patients form three cohorts.
For all patients retrospective data are collected for 12 months prior enrollment.
All patients are observed prospectively for 6 months. At the point of 6 months patients taking Fabrazyme or Replagal at the moment finish the study.
Patients on Fabagal are observed for 12 more months (up to 18 months after enrollment).
For all patients retrospective data are collected for 12 months prior enrollment.
All patients are observed prospectively for 6 months. At the point of 6 months patients taking Fabrazyme or Replagal at the moment finish the study.
Patients on Fabagal are observed for 12 more months (up to 18 months after enrollment).
Study Oversight
Has Oversight DMC:
True
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?:
False
Is an FDA AA801 Violation?: