Fabry Disease Clinical Trial
Official title:
Fabry Disease Registry Protocol
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Registry are: - To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; - To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; - To characterize and describe the Fabry population as a whole; - To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.
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Status | Clinical Trial | Phase | |
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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.
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N/A | |
Completed |
NCT04455230 -
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
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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
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Phase 3 | |
Completed |
NCT00304512 -
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
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Phase 2 | |
Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
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Completed |
NCT03500094 -
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
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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
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Phase 4 | |
Recruiting |
NCT02994303 -
Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
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N/A | |
Completed |
NCT01947634 -
Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study.
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N/A | |
Recruiting |
NCT01695161 -
Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
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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
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Phase 1 | |
Completed |
NCT00701415 -
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 |
NCT00068107 -
Dosing Study of Replagal in Patients With Fabry Disease
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Phase 2 | |
Completed |
NCT01997489 -
Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.
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Phase 4 | |
Recruiting |
NCT06007768 -
Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
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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
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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
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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
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