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ClinicalTrials.gov public records Last synced May 21, 2026, 6:39 PM EDT

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Showing 1–24 of 139 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Langerhans-Cell Histiocytosis Clear all
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
Interventions
15 O Water
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
123 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Phase 2 Interventional Results available
View directory record Official record
Conditions
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Histiocytic and Dendritic Cell Neoplasm, Recurrent Langerhans Cell Histiocytosis, Recurrent Lymphoma, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Histiocytic and Dendritic Cell Neoplasm, Refractory Langerhans Cell Histiocytosis, Refractory Lymphoma, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor
Interventions
Computed Tomography, Magnetic Resonance Imaging, Positron Emission Tomography, Radionuclide Imaging, Selpercatinib, X-Ray Imaging
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
172
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 9, 2025 · Synced May 21, 2026, 6:39 PM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Histiocytosis
Interventions
Trail Making Test, Parts A & B, Brief Test of Attention, Symbol Span, Controlled Oral Word Association Test, The Hopkins Verbal Learning Test-Revised, Brief Visuospatial Memory Test-Revised, Hospital Anxiety and Depression Scale, McGill Quality of Life Scale, MR Brain Imaging, Resting state functional MRI, Functional Assessment of Cancer Therapy Cognitive Scale (FACT-Cog)
Behavioral · Diagnostic Test
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
4
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Commack, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced May 21, 2026, 6:39 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Childhood Langerhans Cell Histiocytosis
Interventions
leucovorin calcium, methotrexate, prednisolone, vinblastine sulfate
Drug
Lead sponsor
Histiocyte Society
Other
Eligibility
Up to 17 Years
Enrollment
376 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
4
States / cities
Minneapolis, Minnesota • Cincinnati, Ohio • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 9, 2014 · Synced May 21, 2026, 6:39 PM EDT
Completed No phase listed Observational Results available
View directory record Official record
Conditions
Pulmonary Langerhans Cell Histiocytosis
Interventions
Not listed
Lead sponsor
University of Cincinnati
Other
Eligibility
18 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 30, 2024 · Synced May 21, 2026, 6:39 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 13, 2021 · Synced May 21, 2026, 6:39 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 6:39 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Adult Langerhans Cell Histiocytosis, Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
Peripheral Blood Stem Cell Transplantation
Procedure
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 65 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 5, 2014 · Synced May 21, 2026, 6:39 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Langerhans Cell Histiocytosis
Interventions
Methotrexate, 6-Thioguanine, Leucovorin Calcium
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 3, 2015 · Synced May 21, 2026, 6:39 PM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Langerhans Cell Histiocytosis
Interventions
Cytarabine, Vinblastine/prednisone
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
11
States / cities
Palo Alto, California • San Diego, California • Minneapolis, Minnesota + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Not applicable Interventional Results available
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
Interventions
CliniMACS
Device
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced May 21, 2026, 6:39 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
VTS-270
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 6 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 4, 2026 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Hyper-IgM, DiGeorge Syndrome, Chediak-Higashi Syndrome, Common Variable Immune Deficiency, Immune Dysregulatory Disorders, Hemophagocytic Lymphohistiocytosis, IPEX, Autoimmune Lymphoproliferative Syndrome, X-linked Lymphoproliferative Syndrome
Interventions
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Acute Myeloid Leukemia, Chronic Myelogenous Leukemia, Non Hodgkin Lymphoma, Hemophagocytic Lymphohistiocytosis, Familial Hemophagocytic Lymphohistiocytosis, Hemophagocytic Syndrome, Epstein Barr Virus Infection, X-linked Lymphoproliferative Disease
Interventions
iCaspase9-transduced T cells, AP1903
Biological · Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2029
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 10, 2025 · Synced May 21, 2026, 6:39 PM EDT
Terminated Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions
Stem Cell Transplant, Myeloablative conditioning regimen
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 22, 2018 · Synced May 21, 2026, 6:39 PM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros A.G.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced May 21, 2026, 6:39 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Kidney Wilms Tumor, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma
Interventions
Laboratory Biomarker Analysis, Palbociclib, Pharmacological Study
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
115
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 93 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced May 21, 2026, 6:39 PM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced May 21, 2026, 6:39 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Langerhans Cell Histiocytosis
Interventions
Prednisone, Vinblastine, mercaptopurine, INDOMETHACIN, Methotrexate, Cytosine Arabinoside, 2-chlorodeoxyadenosine, hematopoietic stem cell transplantation (RIC-HSCT), Intravenous immunoglobulin
Drug · Procedure · Biological
Lead sponsor
North American Consortium for Histiocytosis
Other
Eligibility
Up to 18 Years
Enrollment
1,400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
42
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 36 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced May 21, 2026, 6:39 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced May 21, 2026, 6:39 PM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced May 21, 2026, 6:39 PM EDT