Fabry Disease Clinical Trial
Official title:
An Observational Study of Enzyme Replacement Therapy-Naïve and Agalsidase Beta-Treated Fabry Disease Patients With GLA IVS4 919 G>A Mutation in Taiwan
This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natural history on male and female Fabry disease adult patients who carry the GLA IVS4. This study aims to retrospectively and prospectively investigate the disease natural history, clinical manifestations, and the treatment outcomes upon agalsidase beta in Fabry disease (FD) patients carrying the GLA IVS4 mutation from medical records, physician assessments, and patient-reported outcomes.
Study Design Time Perspective: Retrospective and Prospective ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT04893889 -
Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dimensional Shear Wave Elastography) in Patients With Amyloidosis and Fabry Disease.
|
N/A | |
| Completed |
NCT04455230 -
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
|
Phase 1/Phase 2 | |
| Completed |
NCT01218659 -
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
|
Phase 3 | |
| Completed |
NCT00304512 -
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
|
Phase 2 | |
| Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
|
||
| Completed |
NCT03500094 -
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
|
Phase 3 | |
| Withdrawn |
NCT04143958 -
To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
|
Phase 4 | |
| Recruiting |
NCT02994303 -
Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
|
N/A | |
| Completed |
NCT01947634 -
Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study.
|
N/A | |
| Recruiting |
NCT01695161 -
Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
|
N/A | |
| Completed |
NCT01853852 -
A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
|
Phase 1 | |
| Completed |
NCT00701415 -
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
|
Phase 3 | |
| Completed |
NCT00068107 -
Dosing Study of Replagal in Patients With Fabry Disease
|
Phase 2 | |
| Completed |
NCT01997489 -
Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.
|
Phase 4 | |
| Recruiting |
NCT06007768 -
Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
|
||
| Recruiting |
NCT05698901 -
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
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||
| Active, not recruiting |
NCT03305250 -
Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease
|
N/A | |
| Terminated |
NCT00526071 -
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 |
NCT03566017 -
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 |
NCT06065605 -
Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease
|