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Condition: Gaucher Disease Type I Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Gaucher Disease Type I

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 · NCT04528355Oxidative 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 · NCT055862434SCAR-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.