Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Sep 28, 2026, 9:38 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 140 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Langerhans-Cell Histiocytosis Clear all
Not yet recruiting Phase 2 Interventional

Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

NCT07660783
View directory record Official record
Conditions
Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia, Immune Dysregulation, Hemophagocytic Lymphohistiocytosis (HLH), Non Malignant Disorders
Interventions
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
6 Months to 50 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
2
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Sep 28, 2026, 9:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

NCT03471143
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 Sep 28, 2026, 9:38 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant for Immunologic or Histiocytic Disorders

NCT00176865
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Sep 28, 2026, 9:38 PM EDT
Active, not recruiting Not applicable Interventional Results available

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

NCT05236764
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 Sep 28, 2026, 9:38 PM EDT
Terminated Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

NCT00668564
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Sep 28, 2026, 9:38 PM EDT
Recruiting No phase listed Observational

Project: Every Child for Younger Patients With Cancer

NCT02402244
View directory record Official record
Conditions
Adrenal Gland Pheochromocytoma, Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma, Childhood Kidney Neoplasm, Childhood Langerhans Cell Histiocytosis, Childhood Mature Teratoma, Congenital Mesoblastic Nephroma, Desmoid Fibromatosis, Ganglioneuroma, Lymphoproliferative Disorder, Malignant Neoplasm, Malignant Solid Neoplasm, Melanocytic Neoplasm, Myeloproliferative Neoplasm, Neoplasm of Uncertain Malignant Potential, Neuroendocrine Neoplasm, Stromal Neoplasm
Interventions
Cytology Specimen Collection Procedure, Medical Chart Review
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 25 Years
Enrollment
75,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
246
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 177 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Sep 28, 2026, 9:38 PM EDT
Completed Phase 2 Interventional Results available

A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome

NCT02400463
View directory record Official record
Conditions
Hemophagocytic Syndrome (HPS)
Interventions
Ruxolitinib
Drug
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jan 24, 2021 · Synced Sep 28, 2026, 9:38 PM 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 Sep 28, 2026, 9:38 PM EDT
Not listed No phase listed Observational

Long-term Outcome After Vemurafenib / BRAF Inhibitors Interruption in Erdheim-chester Disease

NCT02089724
View directory record Official record
Conditions
Erdheim-Chester Disease
Interventions
Not listed
Lead sponsor
Groupe Hospitalier Pitie-Salpetriere
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 27, 2016 · Synced Sep 28, 2026, 9:38 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)

NCT03698994
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Histiocytic and Dendritic Cell Neoplasm, 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 Malignant Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Glioma, Refractory Hepatoblastoma, Refractory Histiocytic and Dendritic Cell Neoplasm, 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 Malignant Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Wilms Tumor
Interventions
Pharmacokinetic Study, Ulixertinib
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
105
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 82 more
Source: ClinicalTrials.gov public record
Updated Sep 20, 2026 · Synced Sep 28, 2026, 9:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis

NCT04551131
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Sep 28, 2026, 9:38 PM 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 Sep 28, 2026, 9:38 PM EDT
Terminated Phase 2 Interventional Results available

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 17, 2019 · Synced Sep 28, 2026, 9:38 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

NCT03210714
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor
Interventions
Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Erdafitinib, 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
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
114
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Sep 28, 2026, 9:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

NCT02292654
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 22, 2022 · Synced Sep 28, 2026, 9:38 PM EDT
Withdrawn Phase 2 Interventional

Dabrafenib and Trametinib in Treating Patients With Erdheim Chester Disease With BRAF V600 Mutations

NCT03794297
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 Sep 28, 2026, 9:38 PM EDT
Completed No phase listed Observational

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

NCT01417520
View directory record Official record
Conditions
Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS), Cancer
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 25, 2019 · Synced Sep 28, 2026, 9:38 PM 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 Sep 28, 2026, 9:38 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
Up to 4 Weeks
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2029
U.S. locations
9
States / cities
Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 28, 2026, 9:38 PM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 6, 2024 · Synced Sep 28, 2026, 9:38 PM EDT
Completed Phase 1 Interventional

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

NCT00316498
View directory record Official record
Conditions
Niemann Pick Diseases
Interventions
OGT918
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 28, 2026, 9:38 PM EDT
Completed No phase listed Observational

Uptake and Biodistribution of 18F-fluorocholine in Histiocytic Disorders by PET Imaging and Biopsy Measurement

NCT02608619
View directory record Official record
Conditions
Erdheim-Chester Disease, Langerhans Cell Histiocytosis, Histiocytic Disorders
Interventions
F-choline PET Scan, FDG-PET, biopsy
Radiation · Device · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced Sep 28, 2026, 9:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Sep 28, 2026, 9:38 PM EDT
Recruiting No phase listed Observational

Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study

NCT06339177
View directory record Official record
Conditions
Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis, Macrophage Activation Syndrome, Hyperinflammatory Syndromes
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 120 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
2
States / cities
Bethesda, Maryland • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Sep 28, 2026, 9:38 PM EDT