Fabry Aim Children Early (ACE) Project

Status Withdrawn

Clinical Trial Summary

The purpose of this study is to assess the frequency of Fabry disease in children with early symptoms.

Clinical Trial 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 Design

Related Conditions & MeSH terms

Fabry Disease

Clinical Trial Details

NCT number	NCT04965467
Study type	Observational
Source	Children's Hospital of Fudan University
Contact
Status	Withdrawn
Phase
Start date	July 27, 2021
Completion date	February 28, 2022

View Details

See also
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