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 33 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
lyso-gb3
alpha-galactosidase
  • 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
agalsidase beta
agalsidase alfa
ventricular hypertrophy
  • 1 views
  • 18 Oct, 2022
  • 12 locations
A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months (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

neuropathic pain
abdominal pain
  • 0 views
  • 24 Oct, 2022
  • 8 locations
A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability 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

angiokeratoma
cornea verticillata
alpha-galactosidase
gene therapy
acroparesthesia
  • 114 views
  • 11 Oct, 2022
  • 15 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
agalsidase beta
agalsidase alfa
ventricular hypertrophy
  • 0 views
  • 04 Nov, 2022
  • 15 locations
A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months (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

neuropathic pain
abdominal pain
  • 69 views
  • 11 Nov, 2022
  • 8 locations
A Study of Replagal in Treatment-naïve Adults 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

deficiency
deficit
alpha-galactosidase
gla gene
ventricular hypertrophy
  • 0 views
  • 04 Oct, 2022
  • 2 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

gene therapy
deficiency
deficit
glycosphingolipids
  • 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

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