Found 5 clinical trials
Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for
- 31 views
- 07 Nov, 2020
- 1 location
Dose-Ranging Study of ST-920 an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease
This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of -Gal A at therapeutic levels …
- 108 views
- 29 Apr, 2021
- 9 locations
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study will last for up to 2 years (24 months). Full participation for both mother and infant is 24 months, full participation of mother and development of infant is 24 months, while full participation of mother and no infant participation is 6 months
- 0 views
- 05 Nov, 2021
- 2 locations
Canadian Fabry Disease Initiative (CFDI) National Registry
CFDI NATIONAL REGISTRY Fabry disease is a rare, inherited, genetic condition due to a deficiency of an enzyme called alpha-galactosidase A. This enzyme deficiency causes
- 149 views
- 24 Jan, 2021
- 5 locations
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients. This study component will detail kidney microscopic structural changes in Fabry patients before starting enzyme replacement therapy and will correlate these changes with kidney function, including glomerular filtration rate …
- 43 views
- 16 Sep, 2021
- 2 locations