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Found 5 clinical trials
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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

gla gene
alpha-galactosidase
agalsidase beta
deficiency
fabrazyme
  • 31 views
  • 07 Nov, 2020
  • 1 location
None
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 …

acroparesthesia
alpha-galactosidase
cornea verticillata
angiokeratoma
anhidrosis
  • 108 views
  • 29 Apr, 2021
  • 9 locations
None
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

fabrazyme
agalsidase beta
  • 0 views
  • 05 Nov, 2021
  • 2 locations
None
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

alpha-galactosidase
deficit
deficiency
genetic disorder
neuropathic pain
  • 149 views
  • 24 Jan, 2021
  • 5 locations
None
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 …

renal function
replacement therapy
  • 43 views
  • 16 Sep, 2021
  • 2 locations