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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

Know your stage or your test results (such as EGFR, HER2 or BRCA)? Match trials to your own profile - nothing you type is saved.

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30 recruiting trials

for Sphingolipidoses

Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI NCT04856059NA1 site📍 Toronto University Health Network, Toronto · Tests: Cardiac MRI, ECG/Holter and Blood Biomarkers Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study NCT06758648n/a1 site📍 München Technical University of Munich · Tests: Dynamic retinal vessel analysis (DVA), Biochemistry and immune phenotyping, Questionnaires (Patient reported outcomes),… Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease NCT06539624NA2 sites📍 Shanghai📍 Hangzhou The Children's Hospital of Zhejiang University School of Me… · Tests: EXG110 injection Parkinson's Disease in Type 1 Gaucher Disease NCT07886879n/a1 site📍 New York Icahn School of Medicine at Mount Sinai Retinal Hyperspectral Imaging in Neurodegenerative Diseases NCT07545473NA1 site📍 Melbourne Center for Eye Research Australia · Tests: Hyperspectral camera A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) NCT07082725Phase 334 sites📍 Oakland📍 Minneapolis📍 Rochester Azafaros B.V. · Tests: Nizubaglustat, Placebo Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC NCT04528355n/a1 site📍 Pittsburgh Paul Szabolcs · Tests: data collection Anderson-Fabry Disease Fitness Improvement Training NCT07495410NA1 site📍 Prague General University Hospital, Prague · Tests: Physiotherapy exercise programe Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease NCT05698901n/a1 site📍 Taipei Mackay Memorial Hospital · Tests: Agalsidase beta A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease NCT06818838Phase 1, Phase 23 sites📍 Guangzhou📍 Taiyuan📍 Tianjin Lingyi Biotech Co., Ltd. · Tests: LY-M001 A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis NCT03952637Phase 1, Phase 21 site📍 Bethesda National Human Genome Research Institute (NHGRI) · Tests: AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) NCT04532047Phase 11 site📍 San Francisco University of California, San Francisco · Tests: Aldurazyme (laronidase) Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques NCT07336394n/a1 site📍 Beijing Chinese Academy of Medical Sciences, Fuwai Hospital Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease NCT05843552n/a1 site📍 Minneapolis University of Minnesota · Tests: no intervention GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma NCT05992532n/a23 sites📍 Vitoria-Gasteiz📍 Almería📍 Palma de Mallorca Fundación Española de Hematología y Hemoterapía A Study of Patients With Fabry Disease (US Specific) NCT06906367n/a8 sites📍 Birmingham📍 Little Rock📍 Atlanta Amicus Therapeutics · Tests: migalastat HCl, ERT A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C NCT05163288Phase 314 sites📍 Rochester📍 Parkville📍 Prague IntraBio Inc · Tests: N-Acetyl-L-Leucine, Placebo National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children NCT06985212n/a1 site📍 Paris Wladimir MAUHIN, Dr Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma NCT03721068Phase 12 sites📍 Atlanta📍 Chapel Hill UNC Lineberger Comprehensive Cancer Center · Tests: iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation NCT06813443n/a3 sites📍 Bologna📍 Roma📍 Florence IRCCS Azienda Ospedaliero-Universitaria di Bologna Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia NCT04925349n/a2 sites📍 Le Kremlin-Bicêtre📍 Paris Assistance Publique - Hôpitaux de Paris · Tests: Blood sample collection A Natural History Study of the Gangliosidoses NCT00668187n/a1 site📍 Minneapolis University of Minnesota A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding NCT04252066n/a1 site📍 Philadelphia Amicus Therapeutics · Tests: migalastat A Long-term Follow-up Study of Gaucher Disease NCT03190837n/a1 site📍 Durham Duke University the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease NCT06776419n/a1 site📍 Copenhagen Caroline Michaela Kistorp · Tests: Cardiac Magnetic Ressonance Imaging, 82Rubidium-positron emission tomography and computer-tomography Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study NCT01581424n/a2 sites📍 Minneapolis📍 Minneapolis University of Minnesota 4D-310 in Adults With Fabry Disease and Cardiac Involvement NCT05629559Phase 1, Phase 24 sites📍 Melbourne📍 Perth📍 Westmead 4D Molecular Therapeutics · Tests: 4D-310 UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells NCT02254863Phase 11 site📍 Durham Joanne Kurtzberg, MD · Tests: DUOC-01 Gaucher Disease Outcome Survey (GOS) NCT03291223n/a1 site📍 Lexington Shire Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford NCT01793168n/a2 sites📍 Sioux Falls📍 Sydney Sanford Health
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