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
Diagnostic of Chemotherapy Induced Neuropathy in Children

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 …

vincristine
carboplatin
taxane
bortezomib
alkaloids
  • 0 views
  • 15 Dec, 2021
  • 1 location
A Study of Migalastat in Fabry Disease

Fabry disease is a rare metabolic condition characterised by the widespread deposition of sphingolipids in multiple organ systems. Cardiac involvement is common, it occurs in fifty percent of

migalastat
  • 0 views
  • 22 Jan, 2022
  • 1 location
French Prospective Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat

This is a cohort study to evaluate the effects of migalastat, on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease.

migalastat
  • 0 views
  • 25 Jan, 2021
  • 14 locations
Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease

outcomes and extension of clinical impairment in the Italian Fabry Disease patients' population

  • 0 views
  • 22 Dec, 2021
  • 8 locations
Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy

In Fabry disease (FD), -galactosidase A deficiency leads to the accumulation of globotriaosylceramide (Lyso-Gb3 and Gb3), triggering a pathologic cascade that causes progressive damage to

  • 0 views
  • 05 Feb, 2021
  • 1 location
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or

migalastat
  • 0 views
  • 13 Jun, 2021
  • 2 locations
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients. This study component will detail kidney microscopic structural

replacement therapy
renal function
  • 43 views
  • 16 Sep, 2021
  • 2 locations
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences

of Fabry patients has been built at the initiative of Amicus. In addition, this questionnaire also evaluates the benefit of treatment from the patient's perspective. Nothing is known until now on

migalastat
  • 1 views
  • 23 Jan, 2021
  • 16 locations
Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy

treatment of their cardiac condition. 25 patients with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D strain, diastolic stress

vortex
strain
agalsidase alfa
cardiomyopathy
  • 39 views
  • 05 Jul, 2021
  • 1 location
Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)

The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR.

vortex
replacement therapy
cardiomyopathy
hypertrophy
  • 36 views
  • 04 Jul, 2021
  • 1 location