Fabry Disease Clinical Trial
Official title:
Lipidomics for Identification of New Biomarkers for Fabry Disease
Compare levels of lipids between well characterised enzymatically-genetically-phenotypically patients with Fabry disease and healthy controls (with no Fabry disease). Correlate levels of lipids in patients with Fabry disease to clinical outcomes/manifestations of the disease.
The hypothesis is that Sphingosine-1 Phosphate (S1P) or any other related sphingoid bases and/or other lipid class could be a marker of the severity of cardiovascular remodelling in Fabry disease. The overall approach is, by minimising possible pre-analytical and analytical biases, to study by lipidomics in well characterised enzymatically, genetically and phenotypically patients with Fabry disease, if S1P or any other lipid (including other glycosphingolipids) is shown to be a biomarker for the diagnosis, monitoring of disease activity and prognosis (including cardiovascular outcomes). ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
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N/A | |
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Phase 1/Phase 2 | |
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Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
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Phase 3 | |
| Completed |
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A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
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Phase 2 | |
| Withdrawn |
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Complement Activation in the Lysosomal Storage Disorders
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||
| Completed |
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Phase 3 | |
| Withdrawn |
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Phase 4 | |
| Recruiting |
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Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
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N/A | |
| Completed |
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N/A | |
| Recruiting |
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Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
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N/A | |
| Completed |
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Phase 1 | |
| Completed |
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A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
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Phase 3 | |
| Completed |
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Dosing Study of Replagal in Patients With Fabry Disease
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Phase 2 | |
| Completed |
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Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.
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Phase 4 | |
| Recruiting |
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Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
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||
| Recruiting |
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| Active, not recruiting |
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N/A | |
| Terminated |
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Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
|
Phase 2 | |
| Active, not recruiting |
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Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
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Phase 3 | |
| Recruiting |
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