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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 (15) 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

Biomarker for Patients With Fabry Disease

Fabry disease is a progressive, inherited, multisystemic lysosomal storage disease character-ized by specific neurological, cutaneous, renal, cardiovascular, cochleo-vestibular and cere-brovascular manifestations. Annual incidence is reported to be 1 in 80,000 live births but this figure may underestimate disease prevalence. When late-onset variants of the disease are considered, a prevalence of ...

Phase N/A

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with ...

Phase

Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL (Agalsidase Alfa)

This is an open label switch over study to assess the safety and efficacy of PRX-102. Patients treated with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months. Patients will be screened and evaluated over 3 months while continuing ...

Phase

Fabry Disease in Cerebrovascular Disease

This is a cross-sectional, population-based study to identify Fabry disease in patients with early cerebrovascular involvement. Eligible patients are age above 18 years old (<=55 years old) with early cerebrovascular involvement and have provided inform consent. Patients who have been diagnosed Fabry disease are not eligible. The present study will ...

Phase N/A

Pharmacokinetics Pharmacodynamics and Safety of Moss-aGalactosidase A in Patients With Fabry Disease

Six patients with Fabry disease will be recruited. Patients will receive a single dose of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion. Patients will be hospitalized during the infusion and for at least 24 hours after the end of the infusion. Treatment will be ...

Phase

Screening for Fabry Disease in a Pediatric Population at Risk

The purpose of this study is to assess the frequency of Fabry disease in children with chronic abdominal pain or chronic pain in the extremities

Phase N/A

Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation

In a group of young stroke patients with diagnosed Fabry disease the stroke rehabilitation will be investigated during different prophylactic therapeutic approaches. In this study the investigator will not be given any instructions on stroke and Fabry therapy. All patients with any etiology of stroke and a diagnosed Fabry disease ...

Phase N/A

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: 1. To maintain an established national registry which will collect information ...

Phase N/A