Fabry Disease Clinical Trials

246 clinical trials for Fabry Disease across international registries, including 51 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study — RECRUITING
• A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease — RECRUITING · PHASE2
• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program — RECRUITING
• A Study of Patients With Fabry Disease (US Specific) — RECRUITING
• Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting — RECRUITING
• Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE) — RECRUITING
• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants — RECRUITING · PHASE3
• Fabry Disease Registry & Pregnancy Sub-registry — RECRUITING
• Validation of novel ECG Parameters to detect Fabry disease and other cardiomyopathies — RECRUITING
• Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI — RECRUITING · NA
• Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT — RECRUITING · NA
• Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding — RECRUITING
• Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease — RECRUITING · PHASE2
• Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease — RECRUITING · NA
• RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascular Diseases (ALIGNED) — RECRUITING
• Long Duration Holter ECG in Fabry Disease — RECRUITING · NA
• Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques — RECRUITING
• Taiwan Associated Genetic and Nongenetic Small Vessel Disease — RECRUITING
• A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease — RECRUITING
• Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease — RECRUITING
• Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment — RECRUITING
• Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study — RECRUITING
• A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients — RECRUITING
• Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease — RECRUITING · PHASE1
• A Study of Replagal in Children and Adults With Fabry Disease in India — RECRUITING · PHASE4
• A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding — RECRUITING
• Long-Term Follow-up of Subjects Who Were Treated With ST-920 — RECRUITING
• the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease — RECRUITING
• Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease — RECRUITING
• 4D-310 in Adults With Fabry Disease and Cardiac Involvement — RECRUITING · PHASE1
• Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation — RECRUITING
• Italian Anderson Fabry Disease Cardiovascular Registry — RECRUITING
• Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study — RECRUITING
• Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease — RECRUITING · PHASE1
• Canadian Fabry Disease Initiative (CFDI) National Registry — RECRUITING
• Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease — RECRUITING
• National Registry of Rare Kidney Diseases — RECRUITING
• Pilot-study: "serum neurofilament light chains as a marker for neurological manifestation in fabry disease" — RECRUITING
• Deep sensory-vascular profiling of Fabry patients — RECRUITING
• Evaluation of HEArt invoLvement in Patients With FABRY Disease — RECRUITING
• A RANDOMIZED, OPEN-LABEL, PARALLEL-GROUP, 18-MONTH PHASE 3 STUDY TO EVALUATE THE EFFECT OF VENGLUSTAT COMPARED WITH USUAL STANDARD OF CARE ON LEFT VENTRICULAR MASS INDEX IN PARTICIPANTS WITH FABRY DISEASE AND LEFT VENTRICULAR HYPERTROPHY — RECRUITING · PHASE3
• Investigation of complement activation and autoantibody formation in lysosomal storage diseases: Fabry disease and Gaucher's disease — RECRUITING
• A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, 12-MONTH PHASE 3 STUDY TO EVALUATE THE EFFECT OF VENGLUSTAT ON NEUROPATHIC AND ABDOMINAL PAIN IN MALE AND FEMALE PARTICIPANTS ¿16 YEARS OF AGE WITH FABRY DISEASE WHO ARE TREATMENT-NAÏVE OR UNTREATED FOR AT LEAST 6 MONTHS — RECRUITING · PHASE3
• A PHASE I/II, MULTICENTER, OPEN-LABEL, SINGLE-DOSE, DOSE-RANGING STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF ST-920, A AAV2/6 HUMAN ALPHA GALACTOSIDASE A GENE THERAPY IN SUBJECTS WITH FABRY DISEASE — RECRUITING · PHASE1
• A MULTI-CENTER, OPEN-LABEL, UNCONTROLLED, SINGLE-ARM, EXTENSION STUDY TO DETERMINE THE LONG-TERM SAFETY AND TOLERABILITY OF ORAL LUCERASTAT IN ADULT SUBJECTS WITH FABRY DISEASE — RECRUITING
• OPEN LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF PEGUNIGALSIDASE ALFA (PRX-102) IN PATIENTS WITH FABRY DISEASE — RECRUITING
• A MULTI-CENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO CONTROLLED, PARALLEL-GROUP STUDY TO DETERMINE THE EFFICACY AND SAFETY OF LUCERASTAT ORAL MONOTHERAPY IN ADULT SUBJECTS WITH FABRY DISEASE. — RECRUITING
• A PHASE 3, OPEN LABEL, SWITCH OVER STUDY TO ASSESS THE SAFETY, EFFICACY AND PHARMACOKINETICS OF PEGUNIGALSIDASE ALFA (PRX-102) 2MG/KG ADMINISTERED BY INTRAVENOUS INFUSION EVERY 4 WEEKS FOR 52 WEEKS IN PATIENTS WITH FABRY DISEASE CURRENTLY TREATED WITH ENZYME REPLACEMENT THERAPY: FABRAZYME (AGALSIDASE BETA) OR REPLAGAL (AGALSIDASE ALFA) — RECRUITING · PHASE3
• AN OPEN LABEL STUDY OF THE SAFETY AND EFFICACY OF PRX-102 IN PATIENTS WITH FABRY DISEASE CURRENTLY TREATED WITH REPLAGAL (AGALSIDASE ALFA) — RECRUITING
• A RANDOMIZED, DOUBLE BLIND, ACTIVE CONTROL STUDY OF THE SAFETY AND EFFICACY OF PRX-102 COMPARED TO AGALSIDASE BETA ON RENAL FUNCTION IN PATIENTS WITH FABRY DISEASE PREVIOUSLY TREATED WITH AGALSIDASE BETA — RECRUITING

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