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Condition: Lysosomal Storage Diseases Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Lysosomal Storage Diseases

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 →

Cardiac Structure and Function in MPS
Phase N/A · 3 location(s) · Children's Hospital of Orange County · NCT07361536A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Phase 1/Phase 2 · 3 location(s) · JCR Pharmaceuticals Co., Ltd. · NCT06488924Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT06749366A 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 · NCT00344331Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Phase 2 · 28 location(s) · Shionogi · NCT07123155Registry 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 · NCT03240653ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Phase 1 · 2 location(s) · Immusoft of CA, Inc. · NCT05682144Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Phase N/A · 1 location(s) · Universitätsklinikum Hamburg-Eppendorf · NCT04613089UCB 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 · NCT06669949Lysosomal Acid Lipase (LAL) Deficiency Registry
Phase N/A · 104 location(s) · Alexion Pharmaceuticals, Inc. · NCT01633489Cognitive and Neurological Pathologies in Pompe Disease
Phase N/A · 1 location(s) · Duke University · NCT04639336FLOWER: 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 Study of Participants With Sanfilippo Syndrome Type IIIC
Phase N/A · 2 location(s) · Phoenix Nest · NCT05825131RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Phase N/A · 1 location(s) · Hospital Universitari Vall d'Hebron Research Institute · NCT06213402A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharido
Phase 1 · 1 location(s) · West China Hospital · NCT06519552Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal
Phase N/A · 2 location(s) · University of Minnesota · NCT01581424Pompe Disease Registry Protocol
Phase N/A · 272 location(s) · Genzyme, a Sanofi Company · NCT00231400Direct Lentiviral Injection Gene Therapy for MLD
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT03725670
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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.