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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.

RESULTS

Found (37) clinical trials

The Rare Disease Registry Program (including but not limited to, Gaucher, Fabry, MPS I, and Pompe diseases) is a multi-center, international, longitudinal, observational program that tracks the natural history and outcomes of patients with these rare diseases.

Phase N/A

This is an open-label study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects 12 to <18 years of age with Fabry disease and amenable GLA mutations.

Phase

Canadian Fabry Disease Initiative (CFDI) National Registry

CFDI NATIONAL REGISTRY: Canada-Wide Patient Recruitment There are over 600 people in Canada known to have Fabry Disease. For more details about Fabry Disease, please refer to the "Brief Summary." The goals of this nation-wide study are as follows: To maintain an established national registry which will collect information related ...

Phase N/A

Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy

Objectives - The purpose of this study is to evaluate the impact of ERT with Agalsidase Alfa on LV diastolic function and flow in patients with Fabry's cardiomyopathy using LV 2D strain, diastolic stress echocardiography, LV vortex flow and CMR. Primary / Secondary Endpoint 1) Primary endpoint: Change from baseline ...

Phase N/A

Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)

Objectives -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. Primary / Secondary Endpoint 1) Primary endpoint Changes of peak exercise E/E' by diastolic stress echocardiography (RECAP-F ...

Phase N/A

Kidney Information Network for Disease Research and Education

KINDRED is a prospective observational cohort study that will enroll up to 10,100 dialysis patients throughout the United States with and without known diagnosis of Fabry disease but at risk for Fabry disease (e.g., unknown cause of renal disease, young male, initiating dialysis without a kidney biopsy, etc).This tissue repository ...

Phase N/A

Open-Label Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease

The duration of each subject's participation in this study will be approximately 58 weeks (or 1 year, 6 weeks), comprised of a five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up). During the Screening Period (approximately 4 weeks), written informed consent (and assent, if applicable) will be obtained and ...

Phase

Screening of Fabry Disease in Patients With Left Ventricular Hypertrophy Detected in Echocardiography

The purpose of this study is to determine the prevalence in Belgium of Fabry disease in patients with unexplained hypertrophic cardiomyopathy measured by echocardiography and to determine in Fabry patients which was the most frequently initial symptom. Actually the early diagnosis is important because a treatment exists that can prevent ...

Phase N/A

Lipidomics and Functional Analyses of Platelets in Fabry Disease

Fabry disease (FD) is a severe X-linked inborn error of the lysosomal glycosphingolipid metabolism. FD patients have significantly increased risks for cardiac and cerebrovascular events, which can also occur early and in absence of the typical FD symptoms. However, the pathophysiological mechanisms leading to vascular occlusion and ischemia in FD ...

Phase N/A