Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 7, 2026, 3:32 AM EDT

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Showing 49–64 of 64 matching trials from the live ClinicalTrials.gov search.
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
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
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
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
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
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
Conditions
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
2
States / cities
New York, New York
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
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
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
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
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
Not listed No phase listed Observational Accepts healthy volunteers

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

NCT02000310
Conditions
Lysosomal Storage Disorders
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
80 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2022
U.S. locations
1
States / cities
Fairfax, Virginia
Not listed No phase listed Observational Accepts healthy volunteers

Investigating Lysosomal Storage Diseases in Minority Groups

NCT02120235
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
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
Conditions
GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Interventions
Not listed
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
0 Months and older
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
3
States / cities
Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia