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Showing 1–24 of 65 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Gaucher Disease, Type 2 Clear all
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 Jul 21, 2026, 6:11 PM EDT
Completed Phase 2 Interventional

Monoclonal Antibody Therapy in Treating Children With Metastatic Neuroblastoma in Second Remission

NCT00002458
View directory record Official record
Conditions
Neuroblastoma
Interventions
monoclonal antibody 3F8
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1987 – 2001
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2013 · Synced Jul 21, 2026, 6:11 PM EDT
Completed Phase 1 Interventional

Monoclonal Antibody Therapy With Sargramostim and Interleukin-2 in Treating Children With Neuroblastoma

NCT00005576
View directory record Official record
Conditions
Disseminated Neuroblastoma, Recurrent Neuroblastoma, Regional Neuroblastoma
Interventions
monoclonal antibody Ch14.18, isotretinoin, aldesleukin, sargramostim
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Arcadia, California
Source: ClinicalTrials.gov public record
Updated Jan 15, 2013 · Synced Jul 21, 2026, 6:11 PM EDT
Recruiting Phase 1 Interventional

Allogeneic Expanded Gamma Delta T Cells With GD2 Chemoimmunotherapy in Relapsed /Refractory Neuroblastoma or Refractory/ Relapsed Osteosarcoma

NCT05400603
View directory record Official record
Conditions
Neuroblastoma, Refractory Neuroblastoma, Relapsed Neuroblastoma, Relapsed Osteosarcoma, Refractory Osteosarcoma
Interventions
Ex Vivo Expanded Allogeneic γδ T Cells in Combination with Dinutuximab, Temozolomide, Irinotecan and Zoledronate
Combination Product
Lead sponsor
Emory University
Other
Eligibility
12 Months and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 25, 2025 · Synced Jul 21, 2026, 6:11 PM EDT
Active, not recruiting 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 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Jul 21, 2026, 6:11 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 Jul 21, 2026, 6:11 PM EDT
Completed Phase 1 Interventional

Biological Therapy in Treating Children With Refractory or Recurrent Neuroblastoma or Other Tumors

NCT00003750
View directory record Official record
Conditions
Melanoma (Skin), Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
hu14.18-IL2 fusion protein
Biological
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
54
States / cities
Little Rock, Arkansas • Duarte, California • La Jolla, California + 44 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2014 · Synced Jul 21, 2026, 6:11 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

NCT06193304
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jan 4, 2024 · Synced Jul 21, 2026, 6:11 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety and Clinical Activity of Nivatrotamab in Relapsed/Recurrent Metastatic Small-cell Lung Cancer

NCT04750239
View directory record Official record
Conditions
SCLC
Interventions
Nivatrotamab
Drug
Lead sponsor
Y-mAbs Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
7
States / cities
Tampa, Florida • Atlanta, Georgia • Detroit, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2023 · Synced Jul 21, 2026, 6:11 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 Jul 21, 2026, 6:11 PM EDT
Suspended Phase 1Phase 2 Interventional

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

NCT01463215
View directory record Official record
Conditions
Type I Gaucher Disease
Interventions
Ambroxol
Drug
Lead sponsor
Exsar Corporation
Industry
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Monmouth Junction, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 14, 2013 · Synced Jul 21, 2026, 6:11 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 – 2028
U.S. locations
4
States / cities
Oakland, California • Minneapolis, Minnesota • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Jul 21, 2026, 6:11 PM EDT
Recruiting Phase 1 Interventional

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Jul 21, 2026, 6:11 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 Jul 21, 2026, 6:11 PM EDT
Active, not recruiting No phase listed Observational

Feasibility of Screening for Early Late Effects of Contemporary Therapy in High-Risk Neuroblastoma Survivors

NCT06480526
View directory record Official record
Conditions
High-risk Neuroblastoma
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
5 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Jul 21, 2026, 6:11 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 Jul 21, 2026, 6:11 PM EDT
Recruiting Phase 1 Interventional

GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas (DIPG) & Spinal Diffuse Midline Glioma(DMG)

NCT04196413
View directory record Official record
Conditions
Glioma of Spinal Cord, Glioma of Brainstem
Interventions
GD2 CAR T cells, Fludarabine, Cyclophosphamide, Rituximab
Drug
Lead sponsor
Stanford University
Other
Eligibility
2 Years to 60 Years
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2043
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Jul 21, 2026, 6:11 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 Jul 21, 2026, 6:11 PM EDT
Completed Phase 1 Interventional

Blood T-Cells and EBV Specific CTLs Expressing GD-2 Specific Chimeric T Cell Receptors to Neuroblastoma Patients

NCT00085930
View directory record Official record
Conditions
Neuroblastoma
Interventions
EBV specific CTLs
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2025
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Jul 21, 2026, 6:11 PM EDT
Completed Phase 1 Interventional

Beta-Glucan and Monoclonal Antibody 3F8 in Treating Patients With Metastatic Neuroblastoma

NCT00492167
View directory record Official record
Conditions
Neuroblastoma
Interventions
beta-glucan, monoclonal antibody 3F8, immunohistochemistry staining method, laboratory biomarker analysis
Biological · Other
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
0 Years to 120 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2022
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 6, 2022 · Synced Jul 21, 2026, 6:11 PM EDT
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 Jul 21, 2026, 6:11 PM EDT
Active, not recruiting Phase 1 Interventional

iC9-GD2-CAR-VZV-CTLs/Refractory or Metastatic GD2-positive Sarcoma and Neuroblastoma

NCT01953900
View directory record Official record
Conditions
Osteosarcoma, Neuroblastoma
Interventions
GD2 T cells, VZV vaccine, Fludarabine, Cyclophosphamide
Genetic · Biological · Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jul 21, 2026, 6:11 PM EDT
Active, not recruiting Phase 1 Interventional

Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma

NCT01711554
View directory record Official record
Conditions
Recurrent Neuroblastoma, Refractory Neuroblastoma
Interventions
Dinutuximab, Isotretinoin, Laboratory Biomarker Analysis, Lenalidomide, Pharmacological Study
Biological · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2027
U.S. locations
14
States / cities
Los Angeles, California • Palo Alto, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jul 21, 2026, 6:11 PM EDT
Not listed No phase listed Observational

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

NCT02605603
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 12, 2019 · Synced Jul 21, 2026, 6:11 PM EDT