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Condition: Sphingolipidoses Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Sphingolipidoses

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeedin
Phase N/A · 1 location(s) · Amicus Therapeutics · NCT04252066Krabbe Disease Global Patient Registry
Phase N/A · 1 location(s) · State University of New York at Buffalo · NCT02993796Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI
NA · 1 location(s) · University Health Network, Toronto · NCT04856059World Data on Ambroxol for Patients With GD and GBA Related PD
Phase N/A · 3 location(s) · Shaare Zedek Medical Center · NCT04388969Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory
Phase 1 · 2 location(s) · UNC Lineberger Comprehensive Cancer Center · NCT03721068Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
Phase N/A · 7 location(s) · Chiesi Italia · NCT07109375A Long-term Follow-up Study of Gaucher Disease
Phase N/A · 1 location(s) · Duke University · NCT03190837Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT00344331Registry of Patients Diagnosed With Lysosomal Storage Diseases
Phase N/A · 1 location(s) · University of California, San Francisco · NCT05619900Gaucherite - A Study to Stratify Gaucher Disease
Phase N/A · 8 location(s) · Cambridge University Hospitals NHS Foundation Trust · NCT03240653UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Phase N/A · 1 location(s) · University of California, San Francisco · NCT06669949FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Phase N/A · 1 location(s) · xCures · NCT06539169Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Phase 2/Phase 3 · 10 location(s) · Chiesi Farmaceutici S.p.A. · NCT05710692Evaluation of HEArt invoLvement in Patients With FABRY Disease
Phase N/A · 1 location(s) · Wuerzburg University Hospital · NCT03362164Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal
Phase N/A · 2 location(s) · University of Minnesota · NCT01581424Direct Lentiviral Injection Gene Therapy for MLD
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT03725670Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes
Phase N/A · 1 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT07382128International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Phase N/A · 318 location(s) · Genzyme, a Sanofi Company · NCT00358943A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Typ
Phase 1/Phase 2 · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT03952637Fabry Disease Registry & Pregnancy Sub-registry
Phase N/A · 284 location(s) · Genzyme, a Sanofi Company · NCT00196742Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study
Phase N/A · 1 location(s) · Yonsei University · NCT07235709the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease
Phase N/A · 1 location(s) · Caroline Michaela Kistorp · NCT06776419Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
Phase N/A · 3 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT06813443Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
NA · 2 location(s) · The Children's Hospital of Zhejiang University School of Med · NCT06539624A Natural History Study of the Gangliosidoses
Phase N/A · 1 location(s) · University of Minnesota · NCT00668187A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Phase 1/Phase 2 · 3 location(s) · Lingyi Biotech Co., Ltd. · NCT06818838MPS (RaDiCo Cohort) (RaDiCo-MPS)
Phase N/A · 23 location(s) · Institut National de la Santé Et de la Recherche Médicale, F · NCT06036693Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT07046338
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.