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ClinicalTrials.gov public records Last synced May 21, 2026, 7:19 PM EDT

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

Showing 1–24 of 65 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Gaucher Disease, Type 2 Clear all
Recruiting No phase listed Observational
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced May 21, 2026, 7:19 PM EDT
Not listed No phase listed Observational
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 May 21, 2026, 7:19 PM EDT
Withdrawn Phase 1 Interventional
View directory record Official record
Conditions
Neuroblastoma
Interventions
cyclophosphamide, Fludarabine, GINAKIT Cells
Drug · Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 13, 2017 · Synced May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Neuroblastoma
Interventions
GD2 CAR modified Tri-virus specific cytotoxic t-cells
Biological
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
18 Months to 17 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 22, 2019 · Synced May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
SCLC, Malignant Melanoma, Sarcoma, High Risk Neuroblastoma
Interventions
GD2-SADA:177Lu-DOTA Complex
Drug
Lead sponsor
Y-mAbs Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Scottsdale, Arizona • Duarte, California • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2024 · Synced May 21, 2026, 7:19 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Neuroblastoma
Interventions
anti-GD2 murine IgG3 monoclonal antibody 3F8
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
291 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 15, 2022 · Synced May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 17, 2024 · Synced May 21, 2026, 7:19 PM EDT
Recruiting Phase 1Phase 2 Interventional
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 – 2026
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 21, 2026, 7:19 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Neuroblastoma
Interventions
Anti-GD2 3F8 Monoclonal Antibody, GM-CSF (granulocyte-macrophage colony-stimulating factor), oral isotretinoin
Biological · Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT
Recruiting Phase 1 Interventional
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
52 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 May 7, 2026 · Synced May 21, 2026, 7:19 PM EDT
Suspended Phase 1 Interventional
View directory record Official record
Conditions
Recurrent Childhood Neuroblastoma, Recurrent Childhood Osteosarcoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Childhood Neuroblastoma, Refractory Childhood Osteosarcoma, Refractory Neuroblastoma, Refractory Osteosarcoma
Interventions
Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Cyclophosphamide, Echocardiography Test, Fludarabine Phosphate, GD2-CAR-expressing Autologous T-lymphocytes, Imaging Procedure, Magnetic Resonance Imaging of the Heart, Multigated Acquisition Scan, Questionnaire Administration
Procedure · Drug · Biological + 1 more
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 40 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2040
U.S. locations
6
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
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 May 21, 2026, 7:19 PM EDT
Active, not recruiting Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Neuroblastoma
Interventions
Humanized 3F8 Monoclonal Antibody (Hu3F8)
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
2 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 3, 2023 · Synced May 21, 2026, 7:19 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Neuroblastoma, Osteosarcoma, Other Solid Tumor Cancers
Interventions
Humanized 3F8 Bispecific Antibody, Blood draw
Biological · Other
Lead sponsor
Y-mAbs Therapeutics
Industry
Eligibility
1 Year to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced May 21, 2026, 7:19 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced May 21, 2026, 7:19 PM EDT
Suspended Phase 1Phase 2 Interventional
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 May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Neuroblastoma, High-Risk
Interventions
cyclophosphamide, NK cells, hu3F8, rIL-2
Drug · Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced May 21, 2026, 7:19 PM EDT
Active, not recruiting Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 7:19 PM EDT