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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Long-term Follow-up Study of Lentiviral-based Gene-edited Immune Cell TherapyB-cell neoplasm, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, diffuse large B-cell lymphoma, histiocytosis, immune system disorder, immunoproliferative disorder, interdigitating dendritic cell sarcoma, large B-cell lymphoma, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, neoplasmRecruitingNot stated2026-10-09NCT05377307
Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment TrialCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Male Urogenital Diseases, Myosarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, connective tissue neoplasm, ependymoma, epithelial neoplasm, glioma, hepatoblastoma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, kidney cancer, kidney disorder, lung disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, medulloblastoma, mixed neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, respiratory system disorder, rhabdoid tumor, rhabdomyosarcoma, sarcoma, urinary system disorderActive, not recruitingPhase 22026-10-09NCT04320888
A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell HistiocytosisHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorderRecruitingPhase 22026-10-08NCT05828069
Low Dose Radiation as Bridging Therapy in Relapsed B-Cell Non-Hodgkin LymphomaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, follicular lymphoma, high grade B-cell lymphoma, histiocytosis, immune system disorder, immunoproliferative disorder, indolent B-cell non-Hodgkin lymphoma, interdigitating dendritic cell sarcoma, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, neoplasm, primary mediastinal large B-cell lymphomaActive, not recruitingPhase 12026-10-08NCT05621096
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 DepletionBone Marrow Failure Disorders, Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Lymphohistiocytosis, Hemophagocytic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Primary Immunodeficiency Diseases, acute myeloid leukemia, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, histiocytosis, immune system disorder, immunoproliferative disorder, inborn error of immunity, inherited hemoglobinopathy, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myelodysplastic syndrome, myeloid leukemia, neoplasm, non-Langerhans cell histiocytosisActive, not recruitingNot applicable2026-10-08NCT05236764
Retrospective Study of Immunotherapy Related Toxicities and Factors Impacting Outcomes in Children and Adults With CancerHemic and Lymphatic Diseases, Lymphohistiocytosis, Hemophagocytic, histiocytosis, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoproliferative syndrome, macrophage activation syndrome, non-Langerhans cell histiocytosisActive, not recruitingNot stated2026-10-08NCT03827343
Optimization of the Time and Dosage of Trametinib in BRAF Negative Juvenile PatientsHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorderActive, not recruitingPhase 22026-10-07NCT04943224
Determination of Molecular Status, the Efficacy and Safety of Fluorodeoxyglucose in PET-CT ImagingHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorderActive, not recruitingPhase 32026-10-07NCT04943211
Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory HistiocytosisHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorderActive, not recruitingPhase 22026-10-07NCT04943198
Efficacy of Short Course of Intravenous Dexamethasone and Intravenous Immunoglobulin Therapy in Infection-Related Secondary Hemophagocytic Lymphohistiocytosis (isHLH)Hemic and Lymphatic Diseases, Lymphohistiocytosis, Hemophagocytic, histiocytosis, lymphatic system disorder, non-Langerhans cell histiocytosis, secondary hemophagocytic lymphohistiocytosisNot yet recruitingPhase 32026-10-06NCT07861776
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Male Urogenital Diseases, Myosarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, central nervous system cancer, connective tissue neoplasm, ependymoma, epithelial neoplasm, glioma, hepatoblastoma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, kidney cancer, kidney disorder, lung disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, medulloblastoma, mixed neoplasm, myomatous neoplasm, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, respiratory system disorder, rhabdoid tumor, rhabdomyosarcoma, sarcoma, urinary system disorderActive, not recruitingPhase 22026-10-06NCT03213652
Comparison of Point-of-care Produced CAR T-cell With Commercial CAR T-cells in Patients With R/R LBCLHemic and Lymphatic Diseases, Neoplasms by Histologic Type, histiocytosis, interdigitating dendritic cell sarcoma, lymphatic system disorder, malignant histiocytosis, neoplasm, non-Hodgkin lymphomaActive, not recruitingPhase 22026-10-05NCT05641428
Administration of Donor T Cells With the Caspase-9 Suicide GeneChronic Disease, DNA Virus Infections, Disease Attributes, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Lymphohistiocytosis, Hemophagocytic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Tumor Virus Infections, X-Linked Lymphoproliferative Disorder, acute myeloid leukemia, bone marrow disorder, hematologic disorder, histiocytosis, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myelodysplastic syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphoma, non-Langerhans cell histiocytosis, viral infectious diseaseActive, not recruitingPhase 12026-10-02NCT01494103
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type CBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingNot stated2026-10-02NCT00344331
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 (NPC)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-28NCT07082725
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 GangliosidosisBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-24NCT07054515
Prevention of Anthracycline-Induced Cardiac Dysfunction With Dexrazoxane in Patients With Diffuse Large-B Cell LymphomaB-cell neoplasm, Cardiotoxicity, Chemically-Induced Disorders, Drug-Related Side Effects and Adverse Reactions, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, cardiovascular disorder, diffuse large B-cell lymphoma, heart disorder, heart failure, histiocytosis, immune system disorder, immunoproliferative disorder, injury, interdigitating dendritic cell sarcoma, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, neoplasm, radiation injuryRecruitingPhase 32026-09-23NCT06220032
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)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, central nervous system cancer, childhood ependymoma, ependymoma, epithelial neoplasm, glioma, hepatoblastoma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, kidney cancer, kidney disorder, lung disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, medulloblastoma, mixed neoplasm, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumor, respiratory system disorder, rhabdoid tumor, urinary system disorderActive, not recruitingPhase 22026-09-22NCT03210714
Ruxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)Hemic and Lymphatic Diseases, Lymphohistiocytosis, Hemophagocytic, histiocytosis, lymphatic system disorder, non-Langerhans cell histiocytosisRecruitingPhase 22026-09-21NCT06160791
Mirdametinib in Histiocytic DisordersHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Skin and Connective Tissue Diseases, histiocytosis, interstitial lung disease, juvenile xanthogranuloma, lung disorder, lymphatic system disorder, non-Langerhans cell histiocytosis, respiratory system disorder, sinus histiocytosis with massive lymphadenopathy, skin disorderRecruitingPhase 22026-09-21NCT06153173
Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Male Urogenital Diseases, Myosarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, connective tissue neoplasm, ependymoma, epithelial neoplasm, glioma, hepatoblastoma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, kidney cancer, kidney disorder, lung disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, medulloblastoma, mixed neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, respiratory system disorder, rhabdoid tumor, rhabdomyosarcoma, sarcoma, urinary system disorderActive, not recruitingPhase 22026-09-21NCT03698994
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingNot stated2026-09-16NCT06192576
Phase 1/2 Trial of S241656 in Selected RAS/MAPK Mutation- Positive MalignanciesBrain Metastases, Digestive System Diseases, Digestive System Neoplasms, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Neoplasms by Site, Thoracic Neoplasms, brain disorder, bronchial neoplasm, bronchogenic carcinoma, central nervous system cancer, central nervous system disorder, colonic disorder, colorectal cancer, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endocrine system disorder, histiocytosis, intestinal disorder, lung disorder, lung neoplasm, lymphatic system disorder, malignant endocrine neoplasm, neoplasm, nervous system disorder, non-small cell lung carcinoma, rectal disorder, respiratory system disorder, respiratory tract neoplasm, thyroid gland carcinoma, thyroid gland disorder, thyroid tumorRecruitingPhase 1 / Phase 22026-09-16NCT05786924
CAR-T Followed by Bispecific AntibodiesHemic and Lymphatic Diseases, Neoplasms by Histologic Type, histiocytosis, interdigitating dendritic cell sarcoma, large B-cell lymphoma, lymphatic system disorder, malignant histiocytosis, neoplasmActive, not recruitingPhase 22026-09-14NCT04889716
Pirtobrutinib Plus Methotrexate-Based Chemoimmunotherapy for Systemic DLBCL With CNS InvolvementHemic and Lymphatic Diseases, Neoplasms by Histologic Type, histiocytosis, interdigitating dendritic cell sarcoma, lymphatic system disorder, malignant histiocytosis, neoplasmNot yet recruitingPhase 22026-09-11NCT07812493