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ClinicalTrials.gov public records Last synced Jul 23, 2026, 2:20 PM EDT

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Showing 1–24 of 122 matching trials from the live ClinicalTrials.gov search.
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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
Lymphoma, Hemophagocytic Lymphohistiocytoses
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
Non-Hodgkin Lymphoma, Multiple Myeloma, Histiocytic Neoplasms, Erdheim-Chester Disease, Rosai-Dorfman Disease
Interventions
[68Ga]-Pentixafor
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
New York, New York
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
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
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
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Deficiency Syndromes, Langerhans-Cell Histiocytosis
Interventions
Stem Cell Transplant, Fludarabine, Melphalan, Anti-thymocyte globulin (ATG), Campath 1H, Cyclosporin A, Mycophenolate mofetil, Intravenous immunoglobulin (IVIG)
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 35 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Kidney Wilms Tumor, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Melanoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdoid Tumor of the Kidney, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Carcinoma, Recurrent WHO Grade 2 Glioma, Refractory Adrenal Gland Pheochromocytoma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Medulloblastoma, Refractory Melanoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdoid Tumor of the Kidney, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Carcinoma, Refractory WHO Grade 2 Glioma
Interventions
Tipifarnib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
171
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more
Conditions
Erdheim-Chester Disease
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
New York, New York
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
Conditions
Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic, Arthritis, Juvenile, Adult Onset Still Disease
Interventions
Emapalumab
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
0 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, 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 Langerhans Cell Histiocytosis, 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
Ivosidenib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
171
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more
Conditions
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Wilms Tumor
Interventions
Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Ensartinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
122
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 95 more
Conditions
Niemann-Pick Type C Disease
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Little Rock, Arkansas • Loma Linda, California • Los Angeles, California + 13 more
Terminated Phase 2Phase 3 Interventional Results available

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

NCT04958642
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
Conditions
Hemophagocytic Lymphohistiocytosis
Interventions
Emapalumab
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
Not listed
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
5
States / cities
Grand Rapids, Michigan • Chapel Hill, North Carolina • Cincinnati, Ohio + 1 more
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
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin, Placebo
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Oakland, California • Jacksonville, Florida • Atlanta, Georgia + 4 more
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
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Conditions
Hemophagocytic Lymphohistiocytosis
Interventions
ATG, rabbit, Etoposide, Methotrexate, hydrocortisone, Dexamethasone
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Up to 18 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
14
States / cities
Phoenix, Arizona • San Francisco, California • Stanford, California + 11 more
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma, Ewing Sarcoma, Hepatoblastoma, Langerhans Cell Histiocytosis, Malignant Germ Cell Tumor, Malignant Glioma, Osteosarcoma, Peripheral Primitive Neuroectodermal Tumor, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Rhabdoid Tumor, Rhabdomyosarcoma, Soft Tissue Sarcoma, Wilms Tumor
Interventions
Laboratory Biomarker Analysis, Vemurafenib
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
114
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 90 more
Conditions
Hemophagocytic Lymphohistiocytosis
Interventions
Ruxolitinib, Dexamethasone, Etoposide
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
6 Weeks to 22 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
13
States / cities
Phoenix, Arizona • Orange, California • San Francisco, California + 10 more