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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

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease NCT06272149EARLY Phase 11 site📍 Shanghai Xinhua Hospital, Shanghai Jiao Tong University School of Me… · Tests: VGN-R08b A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease NCT06328608Phase 2, Phase 312 sites📍 Phoenix📍 Atlanta📍 Iowa City Chiesi Farmaceutici S.p.A. · Tests: PRX-102 1 mg/kg every two weeks Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford NCT01793168n/a1 site📍 Sioux Falls Sanford Health A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD) NCT06192576n/a6 sites📍 Chicago📍 Cincinnati📍 Columbus Sanofi · Tests: Olipudase alfa Taiwan Associated Genetic and Nongenetic Small Vessel Disease NCT05473637n/a1 site📍 Taipei National Taiwan University Hospital · Tests: MRI Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease NCT02437396n/a1 site📍 Minneapolis University of Minnesota 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, GM1 Gangliosidosis or GM2 Gangliosidosis NCT07054515Phase 339 sites📍 Oakland📍 Minneapolis📍 Rochester Azafaros B.V. · Tests: AZ-3102, Placebo Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT NCT01962415Phase 21 site📍 Pittsburgh Paul Szabolcs · Tests: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I NCT06162338NA1 site📍 Hanzhou He Huang · Tests: LY-M001 Injection 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 A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease NCT07187440n/a18 sites📍 Hefei📍 Beijing📍 Beijing Takeda · Tests: No intervention Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS) NCT06669949n/a1 site📍 San Francisco University of California, San Francisco · Tests: no intervention A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients NCT06663358n/a10 sites📍 Birmingham📍 Atlanta📍 Chicago Chiesi Farmaceutici S.p.A. · Tests: Pegunigalsidase-alfa Canadian Fabry Disease Initiative (CFDI) National Registry NCT00455104n/a5 sites📍 Calgary📍 Vancouver📍 Halifax Canadian Fabry Research Consortium · Tests: No intervention Krabbe Disease Global Patient Registry NCT02993796n/a1 site📍 Buffalo State University of New York at Buffalo 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 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 Gaucherite - A Study to Stratify Gaucher Disease NCT03240653n/a8 sites📍 Birmingham📍 Birmingham📍 Cambridge Cambridge University Hospitals NHS Foundation Trust · Tests: Stratified response to Enzyme Therapy 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 Long-term Follow-up Study of Gaucher Disease NCT03190837n/a1 site📍 Durham Duke University Cardiovascular Multimodality Imaging Study NCT06610019n/a1 site📍 The Bronx Montefiore Medical Center Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease NCT06207552EARLY Phase 11 site📍 Shanghai Children's Hospital of Fudan University · Tests: BBM-F101 injection 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 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 NCT06941025n/a5 sites📍 Washington D.C.📍 Berlin📍 Rome Chiesi Farmaceutici S.p.A. · Tests: Not applicable- observational study Evaluation of HEArt invoLvement in Patients With FABRY Disease NCT03362164n/a1 site📍 Würzburg Wuerzburg University Hospital Fabry Disease Registry & Pregnancy Sub-registry NCT00196742n/a284 sites📍 Birmingham📍 Birmingham📍 Phoenix Genzyme, a Sanofi Company 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 Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease NCT06869499NA1 site📍 Paris Wladimir MAUHIN, Dr · Tests: Blood sampling for dosage 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 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
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