Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 10, 2026, 3:01 AM EDT

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Showing 49–72 of 140 matching trials from the live ClinicalTrials.gov search.
Conditions
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, melphalan, methylprednisolone, radiation therapy
Biological · Drug · Radiation
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Hershey, Pennsylvania
Conditions
Cancer
Interventions
Trametinib, Dabrafenib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Month to 17 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
10
States / cities
Phoenix, Arizona • San Francisco, California • Washington D.C., District of Columbia + 7 more
Conditions
Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, filgrastim, methotrexate, allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Sebelipase alfa (SBC-102)
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 24 Months
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Irvine, California
Conditions
Erdheim-Chester Disease
Interventions
Dabrafenib Mesylate, Quality-of-Life Assessment, Questionnaire Administration, Trametinib Dimethyl Sulfoxide
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Houston, Texas
Conditions
Niemann-Pick Type C Disease
Interventions
Nizubaglustat, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Conditions
Neimann-Pick Disease
Interventions
Vorinostat
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
Interventions
Hematopoietic Stem Cell Transplant
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
4 Months to 45 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
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
Sphingomyelin Lipidosis
Interventions
placebo (saline), Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2023
U.S. locations
3
States / cities
San Francisco, California • Decatur, Georgia • The Bronx, New York
Conditions
Niemann-Pick Diseases
Interventions
Not listed
Lead sponsor
National Niemann-Pick Disease Foundation
Other
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
New York, New York
Conditions
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
Up to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Niemann-Pick Disease, Type C
Interventions
arimoclomol, Placebo
Drug
Lead sponsor
ZevraDenmark
Industry
Eligibility
2 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Conditions
Cancer
Interventions
Sirolimus, Celecoxib, Etoposide, Cyclophosphamide
Drug
Lead sponsor
Emory University
Other
Eligibility
12 Months to 30 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
6
States / cities
Phoenix, Arizona • Wilmington, Delaware • Atlanta, Georgia + 2 more
Active, not recruiting Phase 2 Interventional Results available

Lenalidomide for Adult Histiocyte Disorders

NCT02523040
Conditions
Langerhans Cell Histiocytosis (LCH), Histiocytoses Erdheim-chester Disease, Histiocytic Sarcoma (HS)
Interventions
Lenalidomide
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Completed Phase 2Phase 3 Interventional Results available

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT02534844
Conditions
Niemann-Pick Disease, Type C
Interventions
Parts A/B: Adrabetadex, Parts A/B: Sham Control
Drug · Other
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
14
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 11 more
Conditions
Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
Interventions
Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Leukemia
Interventions
Alemtuzumab, Etoposide, Dexamethasone, Methotrexate, Phone Call, Tocilizumab
Drug · Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Houston, Texas
Conditions
Niemann-Pick Disease, Type C
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease, Lip and Oral Cavity Carcinoma, Lymphangioleiomyomatosis, Malignant Endocrine Neoplasm, Malignant Female Reproductive System Neoplasm, Malignant Male Reproductive System Neoplasm, Malignant Neoplasm, Malignant Respiratory Tract Neoplasm, Malignant Thoracic Neoplasm, Malignant Urinary System Neoplasm, Mesothelial Neoplasm, Metastatic Malignant Neoplasm, Metastatic Urothelial Carcinoma, Neurofibromatosis Type 2, Recurrent Adult Soft Tissue Sarcoma, Recurrent Breast Carcinoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Digestive System Carcinoma, Recurrent Female Reproductive System Carcinoma, Recurrent Male Reproductive System Carcinoma, Recurrent Malignant Neoplasm, Recurrent Pharyngeal Carcinoma, Recurrent Thyroid Gland Carcinoma, Refractory Malignant Neoplasm, Soft Tissue Neoplasm, Stage III Breast Cancer AJCC v7, Stage III Pharyngeal Cancer, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Stage IV Breast Cancer AJCC v6 and v7, Stage IV Pharyngeal Cancer, Stage IVA Pharyngeal Cancer, Stage IVB Pharyngeal Cancer, Stage IVC Pharyngeal Cancer, Thyroid Gland Neoplasm
Interventions
Bevacizumab, Cetuximab, Laboratory Biomarker Analysis, Pharmacological Study, Temsirolimus, Valproic Acid
Biological · Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Houston, Texas
Conditions
Histiocytosis, Langerhans-Cell
Interventions
Biospecimen Collection, Laboratory Biomarker Analysis, Questionnaire Administration
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 25 Years
Enrollment
647 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Houston, Texas
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Industry
Eligibility
2 Years to 25 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl-L-Leucine, Placebo
Drug · Other
Lead sponsor
IntraBio Inc
Industry
Eligibility
4 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota