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 32 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)

  • 592 views
  • 23 Nov, 2020
  • 1 location
A Fabry Disease Gene Therapy Study (MARVEL1)

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.

gene therapy
alpha-galactosidase
lyso-gb3
  • 151 views
  • 14 Feb, 2022
  • 3 locations
A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy (CARAT)

alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.

migalastat
hypertrophy
ventricular hypertrophy
agalsidase alfa
agalsidase beta
  • 1 views
  • 22 Sep, 2022
  • 8 locations
A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy (CARAT)

alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.

hypertrophy
ventricular hypertrophy
agalsidase beta
agalsidase alfa
migalastat
  • 0 views
  • 23 Sep, 2022
  • 12 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

deficit
deficiency
glycosphingolipids
gene therapy
  • 18 views
  • 25 Jan, 2021
Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD)

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

replacement therapy
cardiomyopathy
genetic disorder
blackouts
  • 7 views
  • 16 Jun, 2022
  • 3 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.

gene therapy
hypertrophy
cornea verticillata
acroparesthesia
ventricular hypertrophy
  • 18 views
  • 24 Apr, 2022
  • 5 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 α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should

gene therapy
anhidrosis
cornea verticillata
acroparesthesia
lyso-gb3
  • 114 views
  • 29 Apr, 2022
  • 12 locations
A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects With Fabry Disease

In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney

deficit
deficiency
replacement therapy
alpha-galactosidase
replagal
  • 0 views
  • 19 Aug, 2022
  • 2 locations
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease (PERIDOT)

disease in adult participants with Fabry disease who are treatment-naïve or untreated for at least 6 months. Study visits will take place approximately every 3 months. The double

abdominal pain
neuropathic pain
  • 69 views
  • 17 Jun, 2022
  • 3 locations