| A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (NCT05280548) | Sanofi | 2022-05-03 |
| A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease (NCT04974749) | Takeda | 2022-05-01 |
| Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies (NCT05409846) | Universidade do Porto | 2022-04-15 |
| A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease (NCT05206773) | Sanofi | 2022-03-11 |
| Italian Anderson Fabry Disease Cardiovascular Registry (NCT06806813) | IRCCS Azienda Ospedaliero-Universitaria di Bologna | 2022-01-26 |
| Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion (NCT05186324) | Chiesi Farmaceutici S.p.A. | 2022-01-26 |
| A Study of Replagal in Treatment-naïve Adults With Fabry Disease (NCT04840667) | Shire | 2021-12-28 |
| Diagnostic Role of Renal Biopsy in Patients With Fabry Disease (NCT06801288) | IRCCS Azienda Ospedaliero-Universitaria di Bologna | 2021-12-01 |
| PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion (NCT05343715) | Bio Sidus SA | 2021-10-23 |
| Studying Lipids as Potential Biomarkers in Patients With Fabry Disease (NCT05046379) | Vastra Gotaland Region | 2021-10-14 |
| China Post-marketing Surveillance (PMS) Study of Fabrazyme® (NCT05054387) | Genzyme, a Sanofi Company | 2021-10-13 |
| Fabry Exercise Intolerance Study (NCT05413876) | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | 2021-10-10 |
| Long-Term Follow-up of Subjects Who Were Treated With ST-920 (NCT05039866) | Sangamo Therapeutics | 2021-08-16 |
| Fabry Aim Children Early (ACE) Project (NCT04965467) | Children's Hospital of Fudan University | 2021-07-27 |
| Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score (NCT04943991) | Wuerzburg University Hospital | 2021-07-01 |
| Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease (NCT04916977) | Chiesi Italia | 2021-06-23 |
| Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI (NCT04856059) | University Health Network, Toronto | 2021-06-15 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (NCT05368038) | Albert Einstein College of Medicine | 2021-05-10 |
| Long Duration Holter ECG in Fabry Disease (NCT04440254) | Institut National de la Santé Et de la Recherche Médicale, France | 2021-05-05 |
| Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients (NCT04577170) | National and Kapodistrian University of Athens | 2020-11-20 |
| Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial) (NCT04639999) | Yonsei University | 2020-11-05 |
| French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat (NCT04602364) | Amicus Therapeutics France SAS | 2020-10-15 |
| Complement Activation in the Lysosomal Storage Disorders (NCT04189601) | Melbourne Health | 2020-09-30 |
| A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 (NCT04455230) | Spur Therapeutics | 2020-09-08 |
| An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (NCT04519749) | 4D Molecular Therapeutics | 2020-09-01 |
| 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 (NCT04143958) | Sanofi | 2020-09-01 |
| Getting Global Rare Disease Insights Through Technology Study (NCT04758130) | M.A.G.I.C. Clinic LTD | 2020-08-07 |
| Evaluation of Phenotypic Variability in Fabry Disease (NCT03145779) | Boston Children's Hospital | 2020-07-01 |
| Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study (NCT06758648) | Technical University of Munich | 2020-06-25 |
| A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. (NCT04281537) | Amicus Therapeutics | 2020-03-01 |
| Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease (NCT03678324) | University of Utah | 2020-01-27 |
| Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease (NCT04049760) | Amicus Therapeutics | 2019-10-11 |
| Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (NCT03305250) | University Hospital Birmingham NHS Foundation Trust | 2019-09-18 |
| Screening of Fabry Disease in Patients With GI Symptoms (NCT04184986) | General University Hospital, Prague | 2019-09-01 |
| Heidelberg In Vivo Confocal Microscopy to Evaluate the Ocular Surface Disorders of Healthy and Diseased Individuals (NCT04025801) | National Taiwan University Hospital | 2019-07-31 |
| A Fabry Disease Gene Therapy Study (NCT04040049) | Spur Therapeutics | 2019-07-08 |
| A Study of Migalastat in Fabry Disease (NCT03949920) | Manchester University NHS Foundation Trust | 2019-05-16 |
| Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201 (NCT04999059) | AVROBIO | 2019-05-08 |
| Therapeutic Exercise to Treat Neuropathic Pain (NCT04050137) | Universidad de Zaragoza | 2019-05-02 |
| Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (NCT04043273) | Amicus Therapeutics France SAS | 2019-04-04 |
| Screening for Fabry Disease in Renal Transplantation (NCT03886714) | University Hospital, Montpellier | 2019-03-25 |
| A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease (NCT03737214) | Idorsia Pharmaceuticals Ltd. | 2018-12-18 |
| 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. | 2018-11-13 |
| Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) (NCT03500094) | Amicus Therapeutics | 2018-09-27 |
| Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease (NCT03566017) | Chiesi Farmaceutici S.p.A. | 2018-09-16 |
| Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (NCT03425539) | Idorsia Pharmaceuticals Ltd. | 2018-06-21 |
| Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients (NCT05056636) | Chang Gung Memorial Hospital | 2018-06-01 |
| Prevalence and Characteristics of Fabry Disease (FD) in Patients With Stroke or Small Fiber Neuropathy (NCT03230149) | Assistance Publique - Hôpitaux de Paris | 2018-03-12 |
| Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease (NCT03454893) | AVROBIO | 2018-02-21 |
| Effect of Migalastat on Cardiac Involvement in Fabry Disease (NCT03838237) | Ospedale San Donato | 2018-01-10 |
| Kidney Information Network for Disease Research and Education (NCT03321604) | Massachusetts General Hospital | 2018-01-01 |
| MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study (NCT03683966) | Wuerzburg University Hospital | 2017-10-27 |
| Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy (NCT03230591) | Yonsei University | 2017-07-12 |
| Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks (NCT03180840) | Protalix | 2017-07-10 |
| Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease (NCT02921620) | Protalix | 2017-07-01 |
| German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. (NCT03135197) | University Hospital Muenster | 2017-06-08 |
| Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) (NCT03018730) | Protalix | 2017-05-17 |
| Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease (NCT02995993) | Greenovation Biotech GmbH | 2016-11-01 |
| Study of the Relation Between Lipid Myocardial Overload Evaluated by Cardiac Magnetic Resonance Imaging (MRI), Alteration of Longitudinal Myocardial Deformations by Echocardiography, and Clinical Achievements (Functional, Biological and Electrical) in Fabry Disease, and Its Outcomes. (NCT03123523) | University Hospital, Bordeaux | 2016-10-18 |
| Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan (NCT02985710) | Massachusetts General Hospital | 2016-08-01 |
| Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease (NCT02800070) | University Health Network, Toronto | 2016-07-01 |
| Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function (NCT02795676) | Protalix | 2016-06-01 |
| Study of the Prevalence of Fabry Disease in French Dialysis Patients (NCT02843334) | Hospices Civils de Lyon | 2016-05-01 |
| Fabry Disease in Cerebrovascular Disease (NCT02859363) | Chang Gung Memorial Hospital | 2016-03-01 |
| Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients (NCT03596398) | Chiayi Christian Hospital | 2016-01-01 |
| Lipidomics and Functional Analyses of Platelets in Fabry Disease (NCT02649660) | Spital Linth | 2015-10-01 |
| Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease (NCT02489344) | Genzyme, a Sanofi Company | 2015-07-07 |
| Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance (NCT02969200) | Ulla Feldt-Rasmussen | 2015-04-01 |
| Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease (NCT02194985) | Amicus Therapeutics | 2015-03-14 |
| Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases (NCT03199001) | University College, London | 2015-02-19 |
| A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease (NCT02930655) | Idorsia Pharmaceuticals Ltd. | 2015-02-01 |
| Cardiac Involvement in Adult Patients With Fabry Disease; Relation to Enzyme Replacement Therapy (NCT02908724) | Ulla Feldt-Rasmussen | 2014-11-01 |
| Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease (NCT02228460) | Genzyme, a Sanofi Company | 2014-11-01 |
| Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy (NCT02994303) | University of Washington | 2014-09-01 |
| Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients (NCT02023086) | Université de Montréal | 2014-08-01 |
| Screening for Fabry Disease in a Pediatric Population at Risk (NCT02152189) | University Hospital Heidelberg | 2014-05-01 |
| Characterisation of Heart Involvement in Fabry Disease With T1 Mapping (NCT04708301) | Manchester University NHS Foundation Trust | 2014-03-12 |
| A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers (NCT02082327) | Amicus Therapeutics | 2014-03-01 |
| Investigating Lysosomal Storage Diseases in Minority Groups (NCT02120235) | O & O Alpan LLC | 2014-02-01 |
| Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. (NCT01947634) | University of Zurich | 2013-09-01 |
| A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease (NCT01839526) | Genzyme, a Sanofi Company | 2013-05-01 |
| Evaluate the Safety and Exploratory Efficacy of GC1119 (NCT01653444) | Green Cross Corporation | 2012-11-01 |
| Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. (NCT01695161) | Johannes Gutenberg University Mainz | 2012-09-01 |
| Pulmonary Involvement in Patients With Fabry Disease (NCT01632111) | University of Zurich | 2012-07-01 |
| Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta (NCT01745185) | O & O Alpan LLC | 2012-06-01 |
| A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (NCT01650779) | Genzyme, a Sanofi Company | 2012-04-01 |
| Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease (NCT01588314) | University of Minnesota | 2012-04-01 |
| Paricalcitol in Fabry Disease (NCT02090608) | Federico II University | 2012-03-01 |
| This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. (NCT01304277) | Shire | 2011-11-17 |
| Open-Label Phase 3 Long-Term Safety Study of Migalastat (NCT01458119) | Amicus Therapeutics | 2011-10-14 |
| Migalastat Food Effect Study (NCT01489995) | Amicus Therapeutics | 2011-10-01 |
| Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease (NCT01218659) | Amicus Therapeutics | 2011-09-08 |
| 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 (NCT01853852) | Amicus Therapeutics | 2011-09-01 |
| A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease (NCT01298141) | Shire | 2011-08-10 |
| A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) (NCT01730482) | Amicus Therapeutics | 2011-08-01 |
| Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function (NCT01730469) | Amicus Therapeutics | 2011-08-01 |
| Detection of Fabry Disease in Chronic Renal Failure Patients in Area Provence - Alpes - Côte d'Azur (NCT01374997) | Centre Hospitalier Universitaire de Nice | 2011-06-01 |
| Safety Study of Replagal® Therapy in Children With Fabry Disease (NCT01363492) | Shire | 2011-05-12 |
| Home Therapy With Replagal in Fabry Disease (NCT01355146) | Shire | 2011-03-15 |
| Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease (NCT01196871) | Amicus Therapeutics | 2011-02-02 |