119 completed, 37 recruiting, 37 unknown, 16 terminated. 34 Phase 2, 30 Phase 3, 24 Not applicable, 23 Phase 1, 13 Phase 4.
Follow Fabry Disease: new trials by email Literature Review of Fabry Disease trials Research Report on Fabry Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Therapeutic Implications of CMR Low Native T1 in Non-treated Non-hypertrophic Females With Fabry Disease (FD) Decision tree for initiating treatment - Fabry Disease | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 60 | 2026-11-01 | 2026-10-14 |
| A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease Lucerastat - Fabry Disease | Phase 3 | Not yet recruiting | Idorsia Pharmaceuticals Ltd. | 16 | 2026-09-01 | 2026-08-21 |
| Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF) Qualitative Interview - Fabry Disease | Enrolling by invitation | Vastra Gotaland Region | 35 | 2026-08-21 | 2026-08-27 | |
| Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting pegunigalsidase alfa - Fabry Disease | Recruiting | Chiesi Italia | 75 | 2026-06-15 | 2026-07-08 | |
| Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE) Observational assessment - Periodontal Disease, Periodontitis | Recruiting | Stefan Lujinschi | 100 | 2026-05-04 | 2026-06-26 | |
| Care Pathway for Patients With Fabry's Disease (Fabry-PATH) Self-questionnaire - Fabry Disease | Not yet recruiting | University Hospital, Toulouse | 200 | 2026-04-01 | 2026-03-20 | |
| A Study of Patients With Fabry Disease (US Specific) migalastat HCl, ERT - Fabry Disease | Recruiting | Amicus Therapeutics | 450 | 2026-02-13 | 2026-10-28 | |
| A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants Migalastat HCl 20 mg - Fabry Disease | Phase 3 | Recruiting | Amicus Therapeutics | 8 | 2026-01-08 | 2026-10-22 |
| Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients Low-FODMAP Diet - Fabry Disease | Not applicable | Active not recruiting | Acibadem University | 30 | 2026-01-02 | 2026-10-26 |
| Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease PET scan of 68Ga-FAPI - Fabry Disease | Not yet recruiting | Núcleo de Apoio à Investigação Clínica - FMUP | 20 | 2026-01-01 | 2026-01-20 | |
| Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study Fabry Disease | Recruiting | Yonsei University | 25 | 2025-11-12 | 2025-12-01 | |
| A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease No intervention - Fabry Disease | Recruiting | Takeda | 200 | 2025-11-01 | 2026-01-02 | |
| Anderson-Fabry Disease Fitness Improvement Training Physiotherapy exercise programe - Fabry Disease | Not applicable | Recruiting | General University Hospital, Prague | 50 | 2025-10-06 | 2026-09-21 |
| A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease PRX-102 1 mg/kg every two weeks - Fabry Disease | Phase 2 / Phase 3 | Recruiting | Chiesi Farmaceutici S.p.A. | 22 | 2025-07-29 | 2026-08-10 |
| Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes HCM - Hypertrophic Cardiomyopathy, Anderson Fabry Disease | Recruiting | IRCCS Azienda Ospedaliero-Universitaria di Bologna | 250 | 2025-06-01 | 2026-02-02 | |
| 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 Not applicable- observational study - Fabry Disease, Pregnancy | Recruiting | Chiesi Farmaceutici S.p.A. | 10 | 2025-05-23 | 2026-03-19 | |
| Aortic Dimensions in Patients With Fabry Disease aorta imaging - Fabry Disease | Not yet recruiting | University Hospital, Caen | 200 | 2025-05-02 | 2025-05-04 | |
| the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease Cardiac Magnetic Ressonance Imaging, 82Rubidium-positron emission tomography and computer-tomography - Fabry Disease, Cardiovascular Diseases | Recruiting | Caroline Michaela Kistorp | 54 | 2025-05-01 | 2025-04-08 | |
| Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease Fabry Disease | Enrolling by invitation | NPO Petrovax | 100 | 2025-03-24 | 2025-12-31 | |
| A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding migalastat - Fabry Disease | Recruiting | Amicus Therapeutics | 20 | 2025-03-18 | 2026-10-28 |
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Lysosomal Storage Diseases (18) · Stroke (16) · Cardiomyopathy, Hypertrophic (9) · Renal Insufficiency, Chronic (8) · Cardiomyopathies (7) · Gaucher Disease (7) · Proteinuria (7) · Hypertrophy, Left Ventricular (6) · Kidney Diseases (6) · Rare Diseases (6) · Amyloid Neuropathies, Familial (5) · Cardiovascular Diseases (5) · Fabry Disease, Cardiac Variant (5) · Kidney Failure, Chronic (5)
Brain Diseases (243) · Brain Diseases, Metabolic, Inborn (243) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (243) · Lipid Metabolism, Inborn Errors (243) · Lysosomal Storage Diseases, Nervous System (243) · Metabolism, Inborn Errors (243) · Nutritional and Metabolic Diseases (243) · Brain Diseases, Metabolic (242) · Genetic Diseases, X-Linked (242) · Central Nervous System Diseases (241) · Genetic Diseases, Inborn (241) · Lipid Metabolism Disorders (241) · Lipidoses (241) · Nervous System Diseases (241) · Sphingolipidoses (241)
agalsidase beta (28) · migalastat (28) · agalsidase alfa (24) · larazotide acetate (19) · Enzyme Replacement Therapy (5) · Blood Specimen Collection (4) · alpha-Galactosidase (4) · venglustat (4) · Mass Screening (3) · Echocardiography (2) · Exercise Test (2) · Genetic Testing (2) · Interviews as Topic (2) · Acetaminophen (1) · Biological Assay (1)
Amicus Therapeutics (24) · Genzyme, a Sanofi Company (17) · Shire (12) · National Institute of Neurological Disorders and Stroke (NINDS) (11) · Chiesi Farmaceutici S.p.A. (8) · CENTOGENE GmbH Rostock (6) · Protalix (6) · Sanofi (6) · Idorsia Pharmaceuticals Ltd. (5) · University Hospital, Bordeaux (5) · Yonsei University (5) · IRCCS Azienda Ospedaliero-Universitaria di Bologna (4) · Baylor Research Institute (3) · Caroline Michaela Kistorp (3) · Massachusetts General Hospital (3)
United States (93) · United Kingdom (51) · France (42) · Germany (31) · Italy (31) · Canada (29) · Australia (27) · Spain (21) · Poland (20) · Denmark (18) · Czechia (17) · Belgium (16) · Austria (15) · Brazil (15) · China (14)
Arndt Rolfs, MD (3) · Ozlem Goker-Alpan, MD (3) · Alan H Cohen, MD (2) · Antonio Pisani, MD (2) · Daniel Franzen, MD (2) · David G Warnock, MD (2) · Elena Biagini, MD, PhD (2) · Inderpal Panesar, MRPharmS (2)
PMID 27657681, PMID 39847314, PMID 23176611, PMID 25795794, PMID 27509102, PMID 31889231, PMID 32994552, PMID 36481125, PMID 10665494, PMID 15100373, PMID 17179052, PMID 17656478, PMID 18346516, PMID 19150871, PMID 19515805, PMID 20730727, PMID 21196729, PMID 22723456, PMID 22805550, PMID 23150649, PMID 23306324, PMID 23468548, PMID 23474038, PMID 23531228, PMID 23748977, PMID 24186912, PMID 24496231, PMID 24607068, PMID 25380809, PMID 25425121, PMID 25659436, PMID 25899239, PMID 25955246, PMID 26038521, PMID 26185417, PMID 26227782, PMID 26252393, PMID 26275228, PMID 26490103, PMID 26566914, PMID 26717401, PMID 26993266, PMID 27059467, PMID 27136905, PMID 27247331, PMID 27265676, PMID 27307708, PMID 27536442, PMID 27834756, PMID 27899752
2019 (3), U54NS065768 (3), 2009 (2), 2014 (2), 2018 (2), 2017 (1), 2023 (1), R01HD073292 (1)