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Active clinical trials for "Fabry Disease"

Results 51-60 of 204

Evaluation of HEArt invoLvement in Patients With FABRY Disease

Rare DiseasesFabry Disease3 more

This study evaluates predictors for the incidence of arrhythmias and sudden cardiac death as well as terminal heart failure in patients with Fabry disease.

Recruiting5 enrollment criteria

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

Fabry Disease

In Portugal, the prevalence of Fabry disease is largely unknown as recently has been stressed by the Portuguese hypertrophic cardiomyopathy registry investigators. On the other hand, few data on Fabry screening protocols in patients with compromised ejection fraction including burned-out hypertrophic cardiomyopathy series have been published. This project intends to perform screening of Fabry disease in patients with distinct cardiomyopathy phenotypes of unknown or dubious etiology and explore the less knew impact of the disease in other cardiac phenotypes.

Recruiting8 enrollment criteria

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

Fabry Disease

This is a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 8 to 12 male subjects 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of Screening.

Terminated17 enrollment criteria

French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat...

Fabry Disease

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.

Active7 enrollment criteria

A Fabry Disease Gene Therapy Study

Fabry DiseaseLysosomal Storage Diseases

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.

Terminated28 enrollment criteria

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry...

Fabry Disease

Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease

Terminated9 enrollment criteria

A Study of Migalastat in Fabry Disease

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 patients and it is the leading cause of death. Despite this, heart and blood vessel (cardiovascular system) manifestations of Fabry disease remain poorly characterised, and it remains unclear which patients benefit from therapy, or when therapy should be initiated. Migalastat is increasingly used to treat fabry disease however the impact of Migalastat on the cardiovascular system is poorly understood. Detailed assessment of the impact of Migalastat on heart and blood vessel structure and function is urgently needed. This observational study will use state of the art, non-invasive investigations to provide greater understanding of the cardiovascular manifestations of Fabry disease and the effects of Migalastat. It will provide insight into which patients respond more effectively to Migalastat, which in turn will facilitate personalisation of therapy, optimisation of the timing of therapy initiation and more cost-effective care.

Active2 enrollment criteria

A Study of Replagal in Treatment-naïve Adults With Fabry Disease

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 function and heart structure of participants with Fabry Disease. Participants will receive one Replagal infusion every other week for up to 104 weeks. They will visit the clinic every 12 to 14 weeks during treatment with a follow-up visit 2 weeks after treatment.

Terminated36 enrollment criteria

Open-Label Phase 3 Long-Term Safety Study of Migalastat

Fabry Disease

This was a long-term, open-label study of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) in participants with Fabry disease who completed treatment in a previous monotherapy trial with migalastat.

Terminated16 enrollment criteria

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Fabry DiseaseChronic Kidney Disease1 more

This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.

Terminated12 enrollment criteria
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