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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Histiocytosis, Langerhans-Cell Clear all
Completed Phase 2 Interventional Results available

Dabrafenib and Trametinib in People With BRAF V600E Mutation Positive Lesions in Erdheim Chester Disease

NCT02281760
View directory record Official record
Conditions
BRAF V600E Mutation
Interventions
Dabrafenib Mesylate, Trametinib Dimethyl Sulfoxide
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 30, 2021 · Synced Jul 23, 2026, 5:04 AM EDT
Completed No phase listed Observational Results available

Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohistiocytosis

NCT03259230
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis
Interventions
Blood Draws, Data Collection
Other
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jul 23, 2026, 5:04 AM EDT
Not listed Phase 2 Interventional

Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases

NCT00084695
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 9, 2014 · Synced Jul 23, 2026, 5:04 AM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Completed Phase 2 Interventional Results available

Study of Sequential Administration of Oral 6-Thioguanine After Methotrexate in Patients With LCH

NCT00588536
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 Jul 23, 2026, 5:04 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders

NCT02425904
View directory record Official record
Conditions
Langerhans Cell Histiocytosis
Interventions
Clofarabine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
14
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Recruiting Phase 2 Interventional

Mirdametinib in Histiocytic Disorders

NCT06153173
View directory record Official record
Conditions
Langerhans Cell Histiocytosis (LCH), Juvenile Xanthogranuloma (JXG), Rosai-Dorfman Disease (RDD), Histiocytic Disorders
Interventions
Mirdametinib
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Jul 23, 2026, 5:04 AM EDT
Active, not recruiting No phase listed Observational

Retrospective Study of Immunotherapy Related Toxicities and Factors Impacting Outcomes in Children and Adults With Cancer

NCT03827343
View directory record Official record
Conditions
Macrophage Activation Syndrome, Primary Hemophagocytic Lymphohistiocytosis
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month to 120 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Completed Phase 2 Interventional Results available

Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)

NCT03233204
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma, Malignant Glioma, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Childhood Malignant Germ Cell Tumor, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Wilms Tumor
Interventions
Olaparib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
111
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Jul 23, 2026, 5:04 AM EDT
Active, not recruiting Phase 1 Interventional

Administration of Donor T Cells With the Caspase-9 Suicide Gene

NCT01494103
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 Jun 30, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Recruiting No phase listed Observational

ECD-Score: a Study on Erdheim-Chester Disease

NCT07454343
View directory record Official record
Conditions
Erdheim-Chester Disease (ECD)
Interventions
Not listed
Lead sponsor
Meyer Children's Hospital IRCCS
Other
Eligibility
7 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Bethesda, Maryland • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

NCT03887533
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 29, 2022 · Synced Jul 23, 2026, 5:04 AM EDT
Recruiting Phase 3 Interventional

Vinblastine/Prednisone Versus Single Therapy With Cytarabine for Langerhans Cell Histiocytosis (LCH)

NCT02670707
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 Jul 23, 2026, 5:04 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patient With Advanced Tumors

NCT04198818
View directory record Official record
Conditions
Advanced Tumors, Melanoma, Non-Small-Cell Lung Cancer, Erdheim-Chester Disease, Other RAS/RAF/MEK/ERK Mutated Tumors
Interventions
HH2710
Drug
Lead sponsor
Haihe Biopharma Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Lafayette, Indiana • Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 18, 2024 · Synced Jul 23, 2026, 5:04 AM EDT
Not listed Not applicable Interventional

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

NCT00006056
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 23, 2026, 5:04 AM EDT
Terminated Phase 2 Interventional Results available

Etoposide in Patients With COVID-19 Infection

NCT04356690
View directory record Official record
Conditions
COVID-19
Interventions
Etoposide
Drug
Lead sponsor
Boston Medical Center
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 14, 2023 · Synced Jul 23, 2026, 5:04 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

NCT03155620
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Histiocytic Sarcoma, Juvenile Xanthogranuloma, Langerhans Cell Histiocytosis, Malignant Glioma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Soft Tissue Sarcoma, Refractory Ewing Sarcoma, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Wilms Tumor
Interventions
Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Ensartinib, Erdafitinib, Ivosidenib, Laboratory Biomarker Analysis, Larotrectinib Sulfate, Magnetic Resonance Imaging, Mutation Carrier Screening, Olaparib, Palbociclib, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, Samotolisib, Selpercatinib, Selumetinib Sulfate, Tazemetostat, Tipifarnib, Ulixertinib, Vemurafenib, X-Ray Imaging
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
1,377 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
168
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 126 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Completed Phase 2 Interventional Results available

Alemtuzumab or Tocilizumab in Combination With Etoposide and Dexamethasone for the Treatment of Adult Patients With Hemophagocytic Lymphohistiocytosis

NCT02385110
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 16, 2024 · Synced Jul 23, 2026, 5:04 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

NCT00344331
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 120 Years
Enrollment
900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Completed Phase 2 Interventional Results available

Umbilical Cord Blood Transplant With Added Sugar and Chemotherapy and Radiation Therapy in Treating Patients With Leukemia or Lymphoma

NCT03096782
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia, Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Chemotherapy-Related Leukemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Hodgkin Lymphoma, Langerhans Cell Histiocytosis, Minimal Residual Disease, Myelodysplastic Syndrome, Myelodysplastic Syndrome With Excess Blasts, Non-Hodgkin Lymphoma, Recurrent Hodgkin Lymphoma, Refractory Acute Lymphoblastic Leukemia, Refractory Myelodysplastic Syndrome, Small Lymphocytic Lymphoma, Therapy-Related Myelodysplastic Syndrome
Interventions
Anti-Thymocyte Globulin, Busulfan, Clofarabine, Cyclophosphamide, Filgrastim-sndz, Fludarabine, Melphalan, Mycophenolate Mofetil, Rituximab, Tacrolimus, Total-Body Irradiation, Umbilical Cord Blood Transplantation
Biological · Drug · Radiation + 1 more
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
12 Years to 65 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 10, 2023 · Synced Jul 23, 2026, 5:04 AM EDT
Active, not recruiting Phase 1 Interventional

CASPALLO: Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene

NCT00710892
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Non-Hodgkin's Lymphoma, Myelodysplastic Syndrome, Chronic Myeloid Leukemia
Interventions
Allodepleted T Cells
Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2027
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Jul 23, 2026, 5:04 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2023 · Synced Jul 23, 2026, 5:04 AM EDT
Completed No phase listed Observational

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

NCT04469894
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 5, 2020 · Synced Jul 23, 2026, 5:04 AM EDT