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ClinicalTrials.gov public records Last synced Oct 10, 2026, 12:59 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 73–92 of 92 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Glucosylceramide Beta-Glucosidase Deficiency Disease Clear all

Conditions: Parkinson Disease, Gaucher Disease
Interventions: 15-0 H20; Drug
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years to 110 Years
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gaucher Disease
Interventions: Eliglustat tartrate; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 170 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 10
States / cities: San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more

Conditions: Neuroblastoma, Osteosarcoma
Interventions: iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine; Biological · Drug
Lead sponsor: UNC Lineberger Comprehensive Cancer Center; Other
Eligibility: 18 Months and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2044
U.S. locations: 2
States / cities: Atlanta, Georgia • Chapel Hill, North Carolina

Conditions: Gaucher Disease, Type 2
Interventions: LY3884961, Methylprednisolone, Sirolimus, Prednisone; Genetic · Drug
Lead sponsor: Prevail Therapeutics; Industry
Eligibility: 0 Months to 24 Months
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 5
States / cities: Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more

Conditions: Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Week to 110 Years
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 1995
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions: venglustat (GZ402671), imiglucerase; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 3
States / cities: New Haven, Connecticut • Dallas, Texas • Fairfax, Virginia

Conditions: Gaucher's Disease
Interventions: Eliglustat; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 18 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Gaucher Disease
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: Not listed
Enrollment: 1,257 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2028
U.S. locations: 1
States / cities: Lexington, Massachusetts

Conditions: Gaucher Disease
Interventions: AVR-RD-02; Biological
Lead sponsor: John Bernat; Other
Eligibility: 18 Years and older
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2037
U.S. locations: 1
States / cities: Iowa City, Iowa

Conditions: Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: Albert Einstein College of Medicine; Other
Eligibility: Up to 4 Weeks
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2029
U.S. locations: 9
States / cities: Brooklyn, New York • Manhasset, New York • New York, New York + 2 more

Conditions: Gaucher Disease
Interventions: Gaucher disease DNA mutation analysis; Other
Lead sponsor: NYU Langone Health; Other
Eligibility: 18 Years and older
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: New York, New York

Conditions: Gaucher Disease, Type 1
Interventions: FLT201; Genetic
Lead sponsor: Spur Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 2
States / cities: Los Angeles, California • Fairfax, Virginia

Conditions: Lysosomal Storage Diseases
Interventions: Enzyme assay and molecular sequencing; Diagnostic Test
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Gaucher Disease
Interventions: Taliglucerase alfa; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 2
States / cities: Decatur, Georgia • New York, New York

Conditions: Gaucher Disease
Interventions: Investigational procedure; Procedure
Lead sponsor: Sanofi; Industry
Eligibility: Not listed
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024
U.S. locations: 3
States / cities: Gainesville, Florida • New York, New York • Milwaukee, Wisconsin

Conditions: MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions: Palliative Care, Hematopoetic Stem Cell Transplantation; Other · Biological
Lead sponsor: University of Pittsburgh; Other
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2035
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Gaucher Disease, Type 1
Interventions: Eliglustat tartrate, Imiglucerase; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2015
U.S. locations: 17
States / cities: Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more

Conditions: Parkinson's Disease, Gaucher's Disease
Interventions: N-acetylcysteine; Drug
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years and older
Enrollment: 9 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2012
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Gaucher Disease, Type 3
Interventions: No intervention; Other
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years to 80 Years
Enrollment: 5 participants
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

Genetic Studies of Lysosomal Storage Disorders

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

Gaucher Disease Outcome Survey (GOS)

Long Term Follow-Up for Safety of AVR-RD-02

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Genzyme Osteopenia/Osteoporosis Study

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

Longitudinal Study of Neurodegenerative Disorders

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)