| Not Yet Recruiting | Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting NCT07109375 | Chiesi Italia | — |
| Not Yet Recruiting | Care Pathway for Patients With Fabry's Disease (Fabry-PATH) NCT07485660 | University Hospital, Toulouse | — |
| Recruiting | A Study of Patients With Fabry Disease (US Specific) NCT06906367 | Amicus Therapeutics | — |
| Recruiting | A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants NCT06904261 | Amicus Therapeutics | Phase 3 |
| Not Yet Recruiting | Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease NCT07351136 | Núcleo de Apoio à Investigação Clínica - FMUP | — |
| Recruiting | Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study NCT07235709 | Yonsei University | — |
| Recruiting | A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease NCT07187440 | Takeda | — |
| Enrolling By Invitation | Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT NCT07495410 | General University Hospital, Prague | N/A |
| Recruiting | A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry NCT06328608 | Chiesi Farmaceutici S.p.A. | Phase 2 / Phase 3 |
| Recruiting | Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® NCT06941025 | Chiesi Farmaceutici S.p.A. | — |
| Not Yet Recruiting | Aortic Dimensions in Patients With Fabry Disease NCT06956573 | University Hospital, Caen | — |
| Recruiting | the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease NCT06776419 | Caroline Michaela Kistorp | — |
| Enrolling By Invitation | Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease NCT06880250 | NPO Petrovax | — |
| Recruiting | A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Br NCT04252066 | Amicus Therapeutics | — |
| Not Yet Recruiting | The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease NCT06819514 | Guangzhou Jiayin Biotech Ltd | Phase 1 / Phase 2 |
| Recruiting | A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients NCT06663358 | Chiesi Farmaceutici S.p.A. | — |
| Completed | A Real-world Wearables Study in Fabry Disease. NCT06484478 | Royal Free Hospital NHS Foundation Trust | — |
| Recruiting | Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease NCT06539624 | The Children's Hospital of Zhejiang University School of Medicine | N/A |
| Recruiting | Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatme NCT07277361 | Wladimir MAUHIN, Dr | — |
| Recruiting | Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease NCT06270316 | UniQure Biopharma B.V. | Phase 1 / Phase 2 |
| Recruiting | Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease NCT06325488 | Caroline Michaela Kistorp | — |
| Completed | A Co-designed Physical Activity Intervention in Fabry Disease NCT06257901 | Brunel University | — |
| Recruiting | Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabr NCT06207552 | Children's Hospital of Fudan University | EARLY_Phase 1 |
| Completed | Early Detection of Fabry Disease NCT05106764 | Takeda | — |
| Recruiting | Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease NCT06114329 | AceLink Therapeutics, Inc. | Phase 2 |
| Unknown | Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertr NCT06169358 | Qilu Hospital of Shandong University | — |
| Recruiting | German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pe NCT06095713 | Universität Münster | — |
| Recruiting | Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease NCT05698901 | Mackay Memorial Hospital | — |
| Unknown | Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease NCT06065605 | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | — |
| Active Not Recruiting | Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan NCT06052800 | Sanofi | — |
| Recruiting | T1 Mapping in Fabry Disease NCT05923788 | Hospices Civils de Lyon | N/A |
| Recruiting | Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease NCT05710692 | Chiesi Farmaceutici S.p.A. | Phase 2 / Phase 3 |
| Unknown | Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients NCT05710367 | Albina Nowak, MD | Phase 2 |
| Unknown | Real World Evidence Study of Danish Fabry Patients NCT06303466 | Caroline Michaela Kistorp | — |
| Completed | Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry) NCT05671770 | Sanofi | — |
| Recruiting | RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascu NCT06935578 | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | — |
| Recruiting | Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease NCT06081062 | ISU Abxis Co., Ltd. | Phase 3 |
| Completed | Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients NCT04804566 | Amicus Therapeutics | — |
| Recruiting | Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transpla NCT06813443 | IRCCS Azienda Ospedaliero-Universitaria di Bologna | — |
| Terminated | Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing NCT05699265 | Children's Hospital Medical Center, Cincinnati | — |
| Unknown | FASHION Fabry Disease Hypertrophic Cardiomyopathy and Infammation NCT05761834 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Unknown | A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease NCT05679076 | Yonsei University | — |
| Completed | Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease NCT05843916 | Bio Sidus SA | Phase 3 |
| Withdrawn | Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease NCT03228940 | Resverlogix Corp | Phase 1 / Phase 2 |
| Completed | Anderson Fabry Disease - the Power of Information. NCT07136662 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | N/A |
| Recruiting | A Study of Replagal in Children and Adults With Fabry Disease in India NCT05067868 | Shire | Phase 4 |
| Active Not Recruiting | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease NCT04020055 | Amicus Therapeutics | Phase 3 |
| Active Not Recruiting | Natural History in Fabry Disease With IVS4+919G>A Mutations NCT07506083 | Chinese University of Hong Kong | — |
| Recruiting | 4D-310 in Adults With Fabry Disease and Cardiac Involvement NCT05629559 | 4D Molecular Therapeutics | Phase 1 / Phase 2 |
| Completed | Autoimmune and Inflammatory Response Biomarkers in Fabry Disease NCT06007768 | Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal | — |
| Active Not Recruiting | A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult P NCT05280548 | Sanofi | Phase 3 |
| Completed | A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease NCT04974749 | Takeda | Phase 3 |
| Completed | Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies NCT05409846 | Universidade do Porto | — |
| Active Not Recruiting | A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Part NCT05206773 | Sanofi | Phase 3 |
| Completed | Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion NCT05186324 | Chiesi Farmaceutici S.p.A. | — |
| Recruiting | Italian Anderson Fabry Disease Cardiovascular Registry NCT06806813 | IRCCS Azienda Ospedaliero-Universitaria di Bologna | — |
| Terminated | A Study of Replagal in Treatment-naïve Adults With Fabry Disease NCT04840667 | Shire | Phase 3 |
| Completed | Diagnostic Role of Renal Biopsy in Patients With Fabry Disease NCT06801288 | IRCCS Azienda Ospedaliero-Universitaria di Bologna | — |
| Completed | PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV In NCT05343715 | Bio Sidus SA | Phase 1 |
| Completed | Studying Lipids as Potential Biomarkers in Patients With Fabry Disease NCT05046379 | Vastra Gotaland Region | — |
| Completed | China Post-marketing Surveillance (PMS) Study of Fabrazyme® NCT05054387 | Genzyme, a Sanofi Company | Phase 4 |
| Unknown | Fabry Exercise Intolerance Study NCT05413876 | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | — |
| Enrolling By Invitation | Long-Term Follow-up of Subjects Who Were Treated With ST-920 NCT05039866 | Sangamo Therapeutics | — |
| Withdrawn | Fabry Aim Children Early (ACE) Project NCT04965467 | Children's Hospital of Fudan University | — |
| Active Not Recruiting | Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clin NCT04943991 | Wuerzburg University Hospital | N/A |
| Completed | Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease NCT04916977 | Chiesi Italia | — |
| Recruiting | Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparame NCT04856059 | University Health Network, Toronto | N/A |
| Enrolling By Invitation | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program NCT05368038 | Albert Einstein College of Medicine | — |
| Recruiting | Long Duration Holter ECG in Fabry Disease NCT04440254 | Institut National de la Santé Et de la Recherche Médicale, France | N/A |
| Completed | Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients NCT04577170 | National and Kapodistrian University of Athens | — |
| Unknown | Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial) NCT04639999 | Yonsei University | — |
| Completed | French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat NCT04602364 | Amicus Therapeutics France SAS | — |
| Withdrawn | Complement Activation in the Lysosomal Storage Disorders NCT04189601 | Melbourne Health | — |
| Unknown | Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dim NCT04893889 | University of Sao Paulo General Hospital | N/A |
| Completed | A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 NCT04455230 | Spur Therapeutics | Phase 1 / Phase 2 |
| Active Not Recruiting | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease NCT04519749 | 4D Molecular Therapeutics | Phase 1 / Phase 2 |
| Withdrawn | To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Ver NCT04143958 | Sanofi | Phase 4 |
| Unknown | Getting Global Rare Disease Insights Through Technology Study NCT04758130 | M.A.G.I.C. Clinic LTD | N/A |
| Withdrawn | Evaluation of Phenotypic Variability in Fabry Disease NCT03145779 | Boston Children's Hospital | — |
| Recruiting | Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study NCT06758648 | Technical University of Munich | — |
| Completed | A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease Wit NCT04281537 | Amicus Therapeutics | — |
| Completed | Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease NCT03678324 | University of Utah | N/A |
| Completed | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease NCT04049760 | Amicus Therapeutics | Phase 3 |
| Active Not Recruiting | Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease NCT03305250 | University Hospital Birmingham NHS Foundation Trust | N/A |
| Unknown | Screening of Fabry Disease in Patients With GI Symptoms NCT04184986 | General University Hospital, Prague | — |
| Unknown | Heidelberg In Vivo Confocal Microscopy to Evaluate the Ocular Surface Disorders of Healthy and Diseased Indivi NCT04025801 | National Taiwan University Hospital | — |
| Completed | Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Diseas NCT04046224 | Sangamo Therapeutics | Phase 1 / Phase 2 |
| Terminated | A Fabry Disease Gene Therapy Study NCT04040049 | Spur Therapeutics | Phase 1 / Phase 2 |
| Unknown | A Study of Migalastat in Fabry Disease NCT03949920 | Manchester University NHS Foundation Trust | — |
| Terminated | Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD NCT04999059 | AVROBIO | — |
| Completed | Therapeutic Exercise to Treat Neuropathic Pain NCT04050137 | Universidad de Zaragoza | N/A |
| Completed | Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences NCT04043273 | Amicus Therapeutics France SAS | — |
| Terminated | Screening for Fabry Disease in Renal Transplantation NCT03886714 | University Hospital, Montpellier | — |
| Recruiting | Taiwan Associated Genetic and Nongenetic Small Vessel Disease NCT05473637 | National Taiwan University Hospital | — |
| Active Not Recruiting | A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease NCT03737214 | Idorsia Pharmaceuticals Ltd. | Phase 3 |
| Active Not Recruiting | Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients NCT03614234 | Chiesi Farmaceutici S.p.A. | Phase 3 |
| Completed | Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Y NCT03500094 | Amicus Therapeutics | Phase 3 |
| Completed | Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease NCT03566017 | Chiesi Farmaceutici S.p.A. | Phase 3 |
| Completed | Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease NCT03425539 | Idorsia Pharmaceuticals Ltd. | Phase 3 |
| Unknown | Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients NCT05056636 | Chang Gung Memorial Hospital | — |
| Completed | Prevalence and Characteristics of Fabry Disease (FD) in Patients With Stroke or Small Fiber Neuropathy NCT03230149 | Assistance Publique - Hôpitaux de Paris | — |
| Terminated | Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease NCT03454893 | AVROBIO | Phase 1 / Phase 2 |
| Completed | Effect of Migalastat on Cardiac Involvement in Fabry Disease NCT03838237 | Ospedale San Donato | — |
| Terminated | Kidney Information Network for Disease Research and Education NCT03321604 | Massachusetts General Hospital | — |
| Unknown | MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study NCT03683966 | Wuerzburg University Hospital | — |
| Unknown | Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy NCT03230591 | Yonsei University | — |
| Completed | Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks NCT03180840 | Protalix | Phase 3 |
| Withdrawn | Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease NCT02921620 | Protalix | Phase 3 |
| Completed | German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat- NCT03135197 | University Hospital Muenster | — |
| Completed | Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alf NCT03018730 | Protalix | Phase 3 |
| Completed | Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease NCT02995993 | Greenovation Biotech GmbH | Phase 1 |
| Unknown | Study of the Relation Between Lipid Myocardial Overload Evaluated by Cardiac Magnetic Resonance Imaging (MRI), NCT03123523 | University Hospital, Bordeaux | — |
| Completed | Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan NCT02985710 | Massachusetts General Hospital | N/A |
| Completed | Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fab NCT02800070 | University Health Network, Toronto | Phase 1 |
| Completed | Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function NCT02795676 | Protalix | Phase 3 |
| Unknown | Study of the Prevalence of Fabry Disease in French Dialysis Patients NCT02843334 | Hospices Civils de Lyon | — |
| Unknown | Fabry Disease in Cerebrovascular Disease NCT02859363 | Chang Gung Memorial Hospital | — |
| Unknown | Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients NCT03596398 | Chiayi Christian Hospital | — |
| Unknown | Lipidomics and Functional Analyses of Platelets in Fabry Disease NCT02649660 | Spital Linth | — |
| Completed | Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Na NCT02489344 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance NCT02969200 | Ulla Feldt-Rasmussen | — |
| Completed | Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease NCT02194985 | Amicus Therapeutics | Phase 3 |
| Unknown | Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases NCT03199001 | University College, London | — |
| Completed | A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease NCT02930655 | Idorsia Pharmaceuticals Ltd. | Phase 1 |
| Completed | Cardiac Involvement in Adult Patients With Fabry Disease; Relation to Enzyme Replacement Therapy NCT02908724 | Ulla Feldt-Rasmussen | — |
| Completed | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment NCT02228460 | Genzyme, a Sanofi Company | Phase 2 |
| Unknown | Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy NCT02994303 | University of Washington | — |
| Completed | Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients NCT02023086 | Université de Montréal | — |
| Completed | Screening for Fabry Disease in a Pediatric Population at Risk NCT02152189 | University Hospital Heidelberg | — |
| Completed | Characterisation of Heart Involvement in Fabry Disease With T1 Mapping NCT04708301 | Manchester University NHS Foundation Trust | — |
| Completed | A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers NCT02082327 | Amicus Therapeutics | Phase 1 |
| Unknown | Investigating Lysosomal Storage Diseases in Minority Groups NCT02120235 | O & O Alpan LLC | — |
| Completed | Extension Study of PRX-102 for up to 60 Months NCT01981720 | Protalix | Phase 1 / Phase 2 |
| Completed | Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. NCT01947634 | University of Zurich | N/A |
| Terminated | A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease NCT01839526 | Genzyme, a Sanofi Company | Phase 1 |
| Completed | Evaluate the Safety and Exploratory Efficacy of GC1119 NCT01653444 | Green Cross Corporation | Phase 1 |
| Completed | Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients NCT01678898 | Protalix | Phase 1 / Phase 2 |
| Unknown | Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. NCT01695161 | Johannes Gutenberg University Mainz | — |
| Completed | Pulmonary Involvement in Patients With Fabry Disease NCT01632111 | University of Zurich | — |
| Completed | Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta NCT01745185 | O & O Alpan LLC | — |
| Completed | A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsida NCT01650779 | Genzyme, a Sanofi Company | Phase 4 |
| Withdrawn | Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease NCT01588314 | University of Minnesota | Phase 2 |
| Completed | Paricalcitol in Fabry Disease NCT02090608 | Federico II University | N/A |
| Completed | This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. NCT01304277 | Shire | Phase 2 |
| Terminated | Open-Label Phase 3 Long-Term Safety Study of Migalastat NCT01458119 | Amicus Therapeutics | Phase 3 |
| Completed | Migalastat Food Effect Study NCT01489995 | Amicus Therapeutics | Phase 1 |
| Completed | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry NCT01218659 | Amicus Therapeutics | Phase 3 |
| Completed | A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Eval NCT01853852 | Amicus Therapeutics | Phase 1 |
| Completed | A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replace NCT01298141 | Shire | Phase 3 |
| Completed | A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) NCT01730482 | Amicus Therapeutics | Phase 1 |
| Completed | Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Fu NCT01730469 | Amicus Therapeutics | Phase 1 |
| Completed | Detection of Fabry Disease in Chronic Renal Failure Patients in Area Provence - Alpes - Côte d'Azur NCT01374997 | Centre Hospitalier Universitaire de Nice | N/A |
| Completed | Safety Study of Replagal® Therapy in Children With Fabry Disease NCT01363492 | Shire | Phase 2 |
| Completed | Home Therapy With Replagal in Fabry Disease NCT01355146 | Shire | — |
| Completed | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabr NCT01196871 | Amicus Therapeutics | Phase 2 |
| Completed | The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patient NCT01268241 | CENTOGENE GmbH Rostock | — |
| Completed | Androgenetic Alopecia in Fabry Disease NCT01295008 | Baylor Research Institute | — |
| Completed | Sophisticated Assessment of Disease Burden in Patients With Fabry Disease NCT01210196 | Shire | — |
| Recruiting | Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in F NCT01581424 | University of Minnesota | — |
| Completed | Establishment of Biomarkers for Fabry Disease NCT01165697 | Ohio State University | — |
| Completed | Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Di NCT01124643 | Shire | Phase 3 |
| Unknown | Pulmonary Disease and Exercise Tolerance in Boys With Fabry Disease NCT01304875 | Cedars-Sinai Medical Center | — |
| Recruiting | Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance NCT07336394 | Chinese Academy of Medical Sciences, Fuwai Hospital | — |
| Recruiting | National Registry of Rare Kidney Diseases NCT06065852 | UK Kidney Association | — |
| Completed | Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease NCT00925301 | Amicus Therapeutics | Phase 3 |
| Completed | Study of the Spermatic Characteristics of Patients With Fabry Disease NCT04073888 | University Hospital, Bordeaux | N/A |
| Completed | Fabry Screening Study NCT01019629 | Baylor Research Institute | — |
| Unknown | Viennese Prevalence Study of Anderson-Fabry Disease NCT00871611 | Medical University of Vienna | — |
| Completed | Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease NCT00864851 | Shire | Phase 3 |
| Completed | A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without NCT00701415 | Genzyme, a Sanofi Company | Phase 3 |
| Terminated | Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who NCT00526071 | Amicus Therapeutics | Phase 2 |
| Completed | Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation NCT00413595 | CENTOGENE GmbH Rostock | — |
| Completed | The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study NCT00446862 | University of Alabama at Birmingham | — |
| Recruiting | Canadian Fabry Disease Initiative (CFDI) National Registry NCT00455104 | Canadian Fabry Research Consortium | — |
| Completed | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fa NCT00304512 | Amicus Therapeutics | Phase 2 |
| Completed | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Dis NCT00283959 | Amicus Therapeutics | Phase 2 |
| Terminated | Study of the Effects of Fabrazyme Treatment on Lactation and Infants NCT00230607 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Dis NCT00283933 | Amicus Therapeutics | Phase 2 |
| Withdrawn | A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Rena NCT00312767 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease NCT00214500 | Amicus Therapeutics | Phase 2 |
| Completed | A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease NCT00140621 | Genzyme, a Sanofi Company | Phase 4 |
| Unknown | Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease NCT00487630 | Assistance Publique - Hôpitaux de Paris | Phase 4 |
| Completed | Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity NCT00106912 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Completed | Replagal Enzyme Replacement Therapy for Adults With Fabry Disease NCT00097890 | National Institute of Neurological Disorders and Stroke (NINDS) | Phase 4 |
| Completed | Replagal Enzyme Replacement Therapy for Children With Fabry Disease NCT00084084 | Shire | Phase 2 |
| Completed | A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease NCT00233870 | Genzyme, a Sanofi Company | — |
| Completed | Neuropathic Pain and Fabry Disease NCT00168974 | Danish Pain Research Center | — |
| Completed | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease NCT00081497 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | Alternative Dosing and Regimen of Replagal to Treat Fabry Disease NCT00075244 | National Institute of Neurological Disorders and Stroke (NINDS) | Phase 2 |
| Completed | An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease NCT00357786 | National Institute of Neurological Disorders and Stroke (NINDS) | Phase 1 |
| Completed | An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease NCT00071877 | National Institute of Neurological Disorders and Stroke (NINDS) | Phase 2 |
| Completed | Dosing Study of Replagal in Patients With Fabry Disease NCT00068107 | Baylor Research Institute | Phase 2 |
| Completed | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease NCT00196716 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Registry of Fabry Disease - A Multicenter Observational Study NCT00055016 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Terminated | Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme NCT00837824 | Genzyme, a Sanofi Company | Phase 2 |
| Terminated | Enzyme Replacement Therapy in Fabry Disease NCT00149318 | Mario Negri Institute for Pharmacological Research | — |
| Completed | Alpha-Galactosidase A Replacement Therapy for Fabry Disease NCT00048906 | National Institute of Neurological Disorders and Stroke (NINDS) | Phase 2 |
| Completed | A Study of Fabrazyme in Pediatric Patients With Fabry Disease NCT00074958 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Data Collection in Women With Fabry Disease NCT00030134 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Completed | Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. NCT01997489 | Rigshospitalet, Denmark | Phase 4 |
| Recruiting | Fabry Disease Registry & Pregnancy Sub-registry NCT00196742 | Genzyme, a Sanofi Company | — |
| Completed | Fabry Outcome Survey (FOS) NCT03289065 | Shire | — |
| Completed | A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Adva NCT00074984 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | Antiproteinuric Agents and Fabry Disease NCT00343577 | University of Alabama at Birmingham | — |
| Recruiting | Evaluation of HEArt invoLvement in Patients With FABRY Disease NCT03362164 | Wuerzburg University Hospital | — |
| Completed | PET Scans in Normal Volunteers and Patients With Fabry Disease NCT00005111 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Completed | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease NCT00074971 | Genzyme, a Sanofi Company | Phase 3 |
| Completed | Vasodilation in Patients With Fabry's Disease NCT00001774 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| No Longer Available | Treatment Protocol of Replagal for Patients With Fabry Disease NCT01031173 | Shire | — |
| Available | Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease NCT01476163 | Amicus Therapeutics | — |
| Approved For Marketing | Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients NCT04552691 | Chiesi Farmaceutici S.p.A. | — |