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 39 clinical trials
Featured trial
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
None
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

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

alpha-galactosidase
gene therapy
lyso-gb3
  • 138 views
  • 15 Feb, 2021
  • 4 locations
None
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

deficit
deficiency
gene therapy
glycosphingolipids
  • 17 views
  • 25 Jan, 2021
None
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

diabetes
functional mri
  • 0 views
  • 10 Mar, 2021
  • 1 location
None
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

cardiomyopathy
genetic disorder
replacement therapy
blackouts
  • 7 views
  • 24 Jan, 2021
  • 5 locations
None
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.

cornea verticillata
acroparesthesia
gene therapy
angiokeratoma
ventricular hypertrophy
  • 17 views
  • 12 Sep, 2021
  • 6 locations
None
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

cornea verticillata
acroparesthesia
alpha-galactosidase
gene therapy
lyso-gb3
  • 104 views
  • 29 Apr, 2021
  • 9 locations
None
Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

Patients will undergo a SmartPill test to gain additional understanding of Fabry disease manifestation via motility abnormalities in order to improve symptom targeted therapy. An additional

replacement therapy
enzyme testing
  • 14 views
  • 07 Mar, 2021
  • 1 location