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Showing 1–24 of 64 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Type 2 Gaucher Disease Clear all
Completed No phase listed Observational

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156
View directory record Official record
Conditions
Gaucher Disease
Interventions
Blood sample
Procedure
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
7
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2010 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 2 Interventional Results available

Therapy for Children With Advanced Stage Neuroblastoma

NCT01857934
View directory record Official record
Conditions
Neuroblastoma
Interventions
cyclophosphamide, topotecan, hu14.18K322A, peripheral blood stem cell harvest, surgical resection, cisplatin, etoposide, doxorubicin, vincristine, busulfan, melphalan, peripheral blood stem cell transplantation, natural killer cell infusion, radiation therapy, GM-CSF, G-CSF, mesna, levetiracetam, interleukin-2, Isotretinoin, CliniMACS
Drug · Biological · Procedure + 2 more
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 18 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2025
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Recruiting Phase 1 Interventional

Anti-GD2 ADC M3554 in Advanced Solid Tumors

NCT06641908
View directory record Official record
Conditions
Advanced Solid Tumor
Interventions
M3554
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Anti-GD2 3F8 Antibody and Allogeneic Natural Killer Cells for High-Risk Neuroblastoma

NCT00877110
View directory record Official record
Conditions
Neuroblastoma, Bone Marrow, Sympathetic Nervous System
Interventions
cyclophosphamide, vincristine, topotecan ,allogeneic NK cells & 3F8
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 9, 2019 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1Phase 2 Interventional

Bivalent Vaccine With Escalating Doses of the Immunological Adjuvant OPT-821, in Combination With Oral β-glucan for High-Risk Neuroblastoma

NCT00911560
View directory record Official record
Conditions
Neuroblastoma
Interventions
adjuvant OPT-821 in a vaccine containing two antigens (GD2L and GD3L) covalently linked to KLH, oral β-glucan
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
374 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

NCT03812042
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 21, 2019 · Synced Sep 22, 2026, 10:06 PM EDT
Recruiting Phase 1Phase 2 Interventional

Gemcitabine and Ex Vivo Expanded Allogenic Universal Donor, TGFβi Natural Killer (NK) Cells With or Without Naxitamab (Danyelza) for the Treatment of Patients With Metastatic, GD2 Expressing, HER2 Negative Breast Cancer

NCT06026657
View directory record Official record
Conditions
Anatomic Stage IV Breast Cancer AJCC v8, HER2-Negative Breast Carcinoma
Interventions
Biospecimen Collection, Computed Tomography, Gemcitabine, Magnetic Resonance Imaging, Naxitamab, Universal Donor Expanded TGF-beta-imprinted NK Cells
Procedure · Drug · Biological
Lead sponsor
Margaret Gatti-Mays
Other
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Completed No phase listed Observational

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

NCT00244582
View directory record Official record
Conditions
Type 2 Gaucher Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Up to 5 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 22, 2026, 10:06 PM EDT
Recruiting Phase 1 Interventional

C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B)

NCT04099797
View directory record Official record
Conditions
Diffuse Intrinsic Pontine Glioma, High Grade Glioma, Embryonal Tumor, Ependymal Tumor
Interventions
C7R-GD2.CART cells (IV and ICV infusion)
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
12 Months to 25 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2041
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 2, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

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

NCT04411654
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

A Phase I Trial of T Cells Expressing an Anti-GD2 Chimeric Antigen Receptor in Children and Young Adults With GD2+ Solid Tumors

NCT02107963
View directory record Official record
Conditions
Sarcoma, Osteosarcoma, Neuroblastoma, Melanoma
Interventions
Anti-GD2-CAR engineered T cells, AP1903, Cyclophosphamide
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 15, 2024 · Synced Sep 22, 2026, 10:06 PM EDT
Recruiting Phase 2 Interventional

NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial

NCT06450041
View directory record Official record
Conditions
Neuroblastoma
Interventions
Universal Donor (UD) TGFβi NK Cells, Temozolomide, Irinotecan, Dinutuximab, GM-CSF
Drug
Lead sponsor
New Approaches to Neuroblastoma Therapy Consortium
Other
Eligibility
1 Year to 31 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2038
U.S. locations
13
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 2 Interventional Results available

Trivalent Ganglioside Vaccine With Immunological Adjuvant or Immunological Adjuvant Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free

NCT01141491
View directory record Official record
Conditions
Sarcoma
Interventions
Trivalent ganglioside vaccine, OPT-821
Biological
Lead sponsor
MabVax Therapeutics, Inc.
Industry
Eligibility
16 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
13
States / cities
Los Angeles, California • Denver, Colorado • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2017 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Monoclonal Antibody A1G4 Plus BCG in Treating Patients With Cancer

NCT00003023
View directory record Official record
Conditions
Neuroblastoma, Sarcoma
Interventions
BCG vaccine, monoclonal antibody A1G4 anti-idiotype vaccine
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced Sep 22, 2026, 10:06 PM EDT
Active, not recruiting Phase 1 Interventional

3rd Generation GD-2 Chimeric Antigen Receptor and iCaspase Suicide Safety Switch, Neuroblastoma, GRAIN

NCT01822652
View directory record Official record
Conditions
Neuroblastoma
Interventions
iC9-GD2 T Cells - frozen, iC9-GD2 T Cells - fresh, Cytoxan, Fludara, Keytruda, iC9-GD2 T cells
Genetic · Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Combination Chemotherapy, Monoclonal Antibody, and Natural Killer Cells in Treating Young Patients With Recurrent or Refractory Neuroblastoma

NCT01576692
View directory record Official record
Conditions
Neuroblastoma
Interventions
Humanized anti-GD2 antibody, Chemotherapy, Cytokines, Natural killer cells, CliniMACS
Biological · Drug · Other + 1 more
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 14, 2018 · Synced Sep 22, 2026, 10:06 PM EDT
Recruiting Phase 1 Interventional

GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma

NCT03294954
View directory record Official record
Conditions
Neuroblastoma
Interventions
GINAKIT Cells, GINAKIT cells + Etanercept
Genetic · Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 21 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2040
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 18, 2025 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Beta-Glucan and Monoclonal Antibody in Treating Patients With Metastatic Neuroblastoma

NCT00037011
View directory record Official record
Conditions
Neuroblastoma
Interventions
beta-glucan, monoclonal antibody 3F8
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 49 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Humanized 3F8 Monoclonal Antibody (Hu3F8) When Combined With Interleukin-2 in Patients With High-Risk Neuroblastoma and GD2-positive Solid Tumors

NCT01662804
View directory record Official record
Conditions
Neuroblastoma
Interventions
3F8 Monoclonal Antibody Combined with Interleukin-2
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
13 Months and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Vaccine Enriched, Autologous, Activated T-Cells Directed to Tumor in Patients With Relapsed/Refractory Melanoma

NCT02482532
View directory record Official record
Conditions
Melanoma
Interventions
tvs-CTL Vaccine
Biological
Lead sponsor
Gary Doolittle
Other
Eligibility
18 Years to 66 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Fairway, Kansas
Source: ClinicalTrials.gov public record
Updated Feb 3, 2022 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 2 Interventional

Biological Therapy in Treating Patients With Neuroblastoma That Has Not Responded to Previous Treatment

NCT00089258
View directory record Official record
Conditions
Neuroblastoma
Interventions
beta-glucan, monoclonal antibody 3F8, sargramostim, isotretinoin
Biological · Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 16, 2013 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 1 Interventional

Monoclonal Antibody Therapy in Treating Patients With Leptomeningeal Cancer

NCT00003022
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Intraocular Melanoma, Lung Cancer, Melanoma (Skin), Neuroblastoma, Retinoblastoma, Sarcoma
Interventions
iodine I 131 monoclonal antibody 3F8
Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
3 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2005
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2013 · Synced Sep 22, 2026, 10:06 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Investigating Lysosomal Storage Diseases in Minority Groups

NCT02120235
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 3, 2017 · Synced Sep 22, 2026, 10:06 PM EDT
Completed Phase 4 Interventional Results available

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT00364858
View directory record Official record
Conditions
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2007
U.S. locations
18
States / cities
Washington D.C., District of Columbia • Coral Springs, Florida • Atlanta, Georgia + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced Sep 22, 2026, 10:06 PM EDT