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

Recruiting studies: Gaucher Disease

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 · NCT07223944World 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 · NCT03721068A Long-term Follow-up Study of Gaucher Disease
Phase N/A · 1 location(s) · Duke University · NCT03190837Registry 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 · NCT03240653FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Phase N/A · 1 location(s) · xCures · NCT06539169International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Phase N/A · 318 location(s) · Genzyme, a Sanofi Company · NCT00358943A 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. · NCT06818838Prodromal Parkinsonian Features in GBA1 Mutation Carriers
Phase N/A · 1 location(s) · Shaare Zedek Medical Center · NCT05253560The GBA Multimodal Study in Parkinson's Disease
Phase N/A · 3 location(s) · Pacific Parkinson's Research Centre · NCT04101968PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Phase 1 · 1 location(s) · University of California, San Francisco · NCT04532047Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Phase N/A · 1 location(s) · Paul Szabolcs · NCT04528355Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00029965Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
Phase N/A · 1 location(s) · University of Minnesota · NCT02437396A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Phase 1/Phase 2 · 9 location(s) · Prevail Therapeutics · NCT05487599A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Dise
NA · 1 location(s) · He Huang · NCT06162338Drug Discovery for Parkinson's With Mutations in the GBA Gene
Phase N/A · 1 location(s) · New York Stem Cell Foundation Research Institute · NCT05536388MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
Phase N/A · 1 location(s) · University of Minnesota · NCT05586243PD Frontline (Part of RAPSODI GD) Remote Assessment of People With Parkinson's
Phase N/A · 1 location(s) · University College London Hospitals · NCT061510024SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
Phase 1/Phase 2 · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT04637503Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
Phase N/A · 1 location(s) · University of Minnesota · NCT05843552Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal G
Phase N/A · 23 location(s) · Fundación Española de Hematología y Hemoterapía · NCT05992532An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
EARLY_Phase 1 · 1 location(s) · Xinhua Hospital, Shanghai Jiao Tong University School of Med · NCT06272149Gaucher Disease Outcome Survey (GOS)
Phase N/A · 1 location(s) · Shire · NCT03291223Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Phase 2 · 1 location(s) · Paul Szabolcs · NCT01962415Institutional Registry of Rare Diseases
Phase N/A · 1 location(s) · Hospital Italiano de Buenos Aires · NCT06573723
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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.