Fabry Disease Clinical Trials

A listing of Fabry Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 37 clinical trials
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Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)

Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)

  • 584 views
  • 23 Nov, 2020
  • 1 location
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

agalsidase alfa
renal function
alpha-galactosidase
fabrazyme
gla gene
  • 31 views
  • 07 Nov, 2020
  • 1 location
A Fabry Disease Gene Therapy Study

This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.

lyso-gb3
gene therapy
alpha-galactosidase
  • 138 views
  • 15 Feb, 2021
  • 4 locations
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the -galactosidase A (GLA) enzyme. The GLA enzyme is ubiquitously expressed

deficiency
glycosphingolipids
deficit
gene therapy
  • 17 views
  • 25 Jan, 2021
Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

The purpose of this research project is: to use an advanced quantitative MRI technique (FBFI) to detect and quantify brain lesion in patients with FD to use fMRI to identify altered brain function to use FBFI and fMRI together to map altered connectivity in response to brain lesions

functional mri
diabetes
  • 0 views
  • 10 Mar, 2021
  • 1 location
Arrhythmia Burden Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease

Fabry disease (FD) is a genetic disorder that leads to progressive accumulation of fat or 'sphingolipid' within the tissues, including the heart muscle and conductive tissue. Improvements in the

genetic disorder
cardiomyopathy
replacement therapy
blackouts
  • 7 views
  • 24 Jan, 2021
  • 5 locations
An Open-label Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Fabry Disease

is comprised of adult males with Fabry Disease.

acroparesthesia
angiokeratoma
gene therapy
hypertrophy
ventricular hypertrophy
  • 17 views
  • 15 May, 2021
  • 6 locations
Dose-Ranging Study of ST-920 an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

ascending doses of ST-920. ST-920 aims to provide stable, long-term production of -Gal A at therapeutic levels in subjects with Fabry disease. The constant production of -Gal A in humans should, importantly

alpha-galactosidase
acroparesthesia
angiokeratoma
lyso-gb3
gene therapy
  • 104 views
  • 29 Apr, 2021
  • 9 locations
Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI

This study will evaluate whether cardiac MRI T1 and T2 mapping improves our ability to detect early abnormalities in the heart in patients with Fabry disease and identify patients at increase

cardiomyopathy
  • 0 views
  • 24 Jul, 2021
  • 1 location