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 17 clinical trials
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
ventricular hypertrophy
hypertrophy
agalsidase beta
agalsidase alfa
  • 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

abdominal pain
neuropathic 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

cornea verticillata
gene therapy
anhidrosis
acroparesthesia
alpha-galactosidase
  • 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
ventricular hypertrophy
hypertrophy
agalsidase beta
agalsidase alfa
  • 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

abdominal pain
neuropathic 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

replacement therapy
deficiency
hypertrophy
alpha-galactosidase
deficit
  • 0 views
  • 04 Oct, 2022
  • 2 locations
Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI (FIESTA-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
  • 04 Oct, 2022
  • 1 location
Diagnostic Accuracy of Electrochemical Skin Conductance Measurement in Paediatric Small Fibre Neuropathy Following Neurotoxic Chemotherapy (SUDOPED)

The purpose of this study is to determine the diagnostic accuracy of a newly developed tool (Electrochemical Skin Conductances (ESC) measurement) easy-to-perform, non-invasive, highly reproductible and not requiring specific training, to identify pediatric chemotherapy-induced-peripheral-neuropathies (CIPN). CIPN are a frequent (20 to 75% depending on the drug), early and potentially severe …

neuralgia
carboplatin
alkaloids
taxane
bortezomib
  • 0 views
  • 19 Oct, 2022
  • 1 location
Fabry Disease Registry & Pregnancy Sub-registry

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment

replacement therapy
deficiency
alpha-galactosidase
agalsidase beta
fabrazyme
  • 460 views
  • 07 Oct, 2022
  • 249 locations
Fabry Disease Registry & Pregnancy Sub-registry

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment

replacement therapy
deficiency
alpha-galactosidase
agalsidase beta
fabrazyme
  • 2 views
  • 07 Oct, 2022
  • 245 locations