Ignite Creation Date:
2025-12-24 @ 4:25 PM
Ignite Modification Date:
2026-01-24 @ 3:54 AM
Study NCT ID:
NCT03683966
Status:
UNKNOWN
Last Update Posted:
2022-07-11
First Post:
2018-09-03
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
Sponsor:
Wuerzburg University Hospital
Organization:
Study Overview
Official Title:
A Structured Survey Among Fabry Patients With a Focus on Adherence to Therapy, Quality of Life and Pain Control
Status:
UNKNOWN
Status Verified Date:
2022-07
Last Known Status:
RECRUITING
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:
MALTA-FABRY
Brief Summary:
This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.
Detailed Description:
Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Since 2016, chaperone therapy as a new therapeutic approach is available.
This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy.
All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided.
This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy.
All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided.
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: