Ignite Creation Date:
2025-12-25 @ 12:40 AM
Ignite Modification Date:
2025-12-27 @ 9:09 AM
Study NCT ID:
NCT04965467
Status:
WITHDRAWN
Last Update Posted:
2021-09-30
First Post:
2021-07-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Fabry Aim Children Early (ACE) Project
Sponsor:
Children's Hospital of Fudan University
Organization:
Study Overview
Official Title:
Fabry Aim Children Early (ACE) Project-Screening for Fabry Disease in a Pediatric Population at Risk
Status:
WITHDRAWN
Status Verified Date:
2021-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Withdrawn due to financial reasons
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to assess the frequency of Fabry disease in children with early symptoms.
Detailed Description:
Fabry disease is a complex, multisystemic and clinically heterogeneous disease that commonly presents in childhood and is caused by deficient activity of the lysosomal enzyme alpha-galactosidaseA (α-gal A). Symptoms of Fabry disease in the pediatric population are well described. Symptoms can occur in early childhood, before age 5 years. Incidence estimations of Fabry disease vary widely. The true incidence is likely to be higher than originally thought, owing to the existence of milder variants of the disease. The purpose of this study is to assess the frequency of Fabry disease in children with early symptoms. Patients would benefit from early diagnosis, appropriate treatment, follow-up and surveillance. Early detection of Fabry patients would also benefit affected relatives, many of whom do not have a clear diagnosis of their clinical condition.
Study Oversight
Has Oversight DMC:
None
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?: