non-Langerhans cell histiocytosis
non-Langerhans cell histiocytosis
Explore linked clinical trials. Standard reference: MONDO:001553166 results · Page 1 of 3
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion | Bone 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 histiocytosis | Active, not recruiting | Not applicable | 2026-10-08 | NCT05236764 | |
| Retrospective Study of Immunotherapy Related Toxicities and Factors Impacting Outcomes in Children and Adults With Cancer | Hemic and Lymphatic Diseases, Lymphohistiocytosis, Hemophagocytic, histiocytosis, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoproliferative syndrome, macrophage activation syndrome, non-Langerhans cell histiocytosis | Active, not recruiting | Not stated | 2026-10-08 | NCT03827343 | |
| 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 lymphohistiocytosis | Not yet recruiting | Phase 3 | 2026-10-06 | NCT07861776 | |
| Administration of Donor T Cells With the Caspase-9 Suicide Gene | Chronic 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 disease | Active, not recruiting | Phase 1 | 2026-10-02 | NCT01494103 | |
| Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C | 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, sphingolipidosis | Recruiting | Not stated | 2026-10-02 | NCT00344331 | |
| 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, sphingolipidosis | Recruiting | Phase 3 | 2026-09-28 | NCT07082725 | |
| 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 | Brain 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, sphingolipidosis | Recruiting | Phase 3 | 2026-09-24 | NCT07054515 | |
| 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 histiocytosis | Recruiting | Phase 2 | 2026-09-21 | NCT06160791 | |
| Mirdametinib in Histiocytic Disorders | Hemic 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 disorder | Recruiting | Phase 2 | 2026-09-21 | NCT06153173 | |
| 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, sphingolipidosis | Recruiting | Not stated | 2026-09-16 | NCT06192576 | |
| Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study | Hemic and Lymphatic Diseases, Inflammation, Lymphohistiocytosis, Hemophagocytic, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Shock, Skin and Connective Tissue Diseases, Systemic Inflammatory Response Syndrome, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, cytokine release syndrome, histiocytosis, immune system disorder, immunoproliferative disorder, iron metabolism disease, isolated hyperferritinemia, juvenile idiopathic arthritis, lymphatic system disorder, lymphoproliferative syndrome, macrophage activation syndrome, metabolic disease, musculoskeletal system disorder, non-Langerhans cell histiocytosis, rheumatic disorder, secondary hemophagocytic lymphohistiocytosis | Recruiting | Not stated | 2026-09-10 | NCT06339177 | |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Neurobehavioral Manifestations, Neurologic Manifestations, Neuronal Ceroid-Lipofuscinoses, Niemann-Pick disease, Niemann-Pick disease type A, Nutritional and Metabolic Diseases, Pelizaeus-Merzbacher spectrum disorder, Peroxisomal Disorders, Sandhoff disease, Skin and Connective Tissue Diseases, Sulfatidosis, Tay-Sachs disease, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, alpha-mannosidosis, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, demyelinating disease, endocrine system disorder, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, juvenile neuronal ceroid lipofuscinosis, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorder, neurodegenerative disease, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 1 | 2026-09-03 | NCT02254863 | |
| Establishing a National Egyptian HLH Registry (NEHR) | Hemic and Lymphatic Diseases, Lymphohistiocytosis, Hemophagocytic, hemophagocytic syndrome, histiocytosis, lymphatic system disorder, non-Langerhans cell histiocytosis | Recruiting | Not stated | 2026-09-02 | NCT07800234 | |
| Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C | 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, sphingolipidosis | Recruiting | Not stated | 2026-08-31 | NCT05588167 | |
| DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers. | Adnexal Diseases, Blood Protein Disorders, Cancer of Gastrointestinal Tract, Digestive System Diseases, Digestive System Neoplasms, Erdheim-Chester disease, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Non-Melanoma Skin Neoplasms, Otorhinolaryngologic Neoplasms, Thoracic Neoplasms, Thyroid Cancer, Papillary, Urogenital Diseases, Urogenital Neoplasms, adenocarcinoma, bronchial neoplasm, bronchogenic carcinoma, cancer, carcinoma, cardiovascular disorder, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endocrine system disorder, epithelial neoplasm, female reproductive system disorder, female reproductive system neoplasm, glioma, gonadal disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, hemorrhagic disease, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, intestinal disorder, laryngeal disorder, laryngeal neoplasm, lung disorder, lung neoplasm, lymphatic system disorder, lymphoproliferative syndrome, malignant endocrine neoplasm, monoclonal gammopathy, neoplasm, neuroepithelial neoplasm, non-Langerhans cell histiocytosis, non-small cell lung carcinoma, otorhinolaryngologic disease, ovarian cancer, ovarian disorder, pancreas disorder, papillary adenocarcinoma, plasma cell neoplasm, primitive neuroectodermal tumor, rectal disorder, respiratory system disorder, respiratory tract neoplasm, thyroid gland disorder, thyroid gland undifferentiated (anaplastic) carcinoma, thyroid tumor, vascular disorder, vascular hemostatic disease | Recruiting | Phase 2 / Phase 3 | 2026-08-17 | NCT07440290 | |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ceroid Lipofuscinosis, Neuronal, 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, GM1 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, Wolman disease, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrotendinous xanthomatosis, cerebrovascular disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis, vascular disorder, xanthomatosis | Enrolling by invitation | Not stated | 2026-07-28 | NCT05368038 | |
| 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) | 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, Skin and Connective Tissue Diseases, 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, histiocytic sarcoma, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, juvenile xanthogranuloma, kidney cancer, kidney disorder, lung disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, malignant glioma, malignant histiocytosis, medulloblastoma, mixed neoplasm, myomatous neoplasm, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, non-Langerhans cell histiocytosis, osteosarcoma, primitive neuroectodermal tumor, respiratory system disorder, rhabdoid tumor, rhabdomyosarcoma, sarcoma, skin disorder, urinary system disorder | Active, not recruiting | Phase 2 | 2026-07-24 | NCT03155620 | |
| Lenalidomide for Adult Histiocyte Disorders | Erdheim-Chester disease, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Neoplasms by Histologic Type, histiocytic sarcoma, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, malignant histiocytosis, neoplasm, non-Langerhans cell histiocytosis, respiratory system disorder | Active, not recruiting | Phase 2 | 2026-07-23 | NCT02523040 | |
| Supportive Care Needs of Caregivers of People With Erdheim-Chester Disease and Other Histiocytic Diseases | Erdheim-Chester disease, Hemic and Lymphatic Diseases, histiocytosis, lymphatic system disorder, non-Langerhans cell histiocytosis | Recruiting | Not stated | 2026-07-02 | NCT03990428 | |
| Early Check: Expanded Screening in Newborns | 3-hydroxy-3-methylglutaric aciduria, 3-hydroxyacyl-CoA dehydrogenase deficiency, 3-methylcrotonyl-CoA carboxylase 1 deficiency, 3-methylcrotonyl-CoA carboxylase 2 deficiency, 3-phosphoglycerate dehydrogenase deficiency, AGAT deficiency, Abnormalities, Multiple, Adrenal hyperplasia, congenital, type 5, Adrenoleukodystrophy, Neonatal, Angelman syndrome, Aortic Stenosis, Supravalvular, Arrhythmias, Cardiac, Barth syndrome, Bone Diseases, Developmental, Bone Diseases, Endocrine, Bone Diseases, Metabolic, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Canavan disease, Cardiac Conduction System Disease, Cardiovascular Abnormalities, Chronic Disease, Citrullinemia 1, Collagen Diseases, Congenital Abnormalities, Congenital Hyperinsulinism, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Digestive System Diseases, Disease Attributes, Duchenne muscular dystrophy, Dwarfism, Dyslipidemias, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Eye Diseases, Hereditary, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Fructose Metabolism, Inborn Errors, G6PD deficiency, Glycogen Storage Disease, Type IXA2, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Hermansky-Pudlak syndrome, Hermansky-Pudlak syndrome 1, Hermansky-Pudlak syndrome 4, Hyperphenylalaninemia, BH4-Deficient, B, Hypophosphatemia, Familial, Imprinting Disorders, Infant, Newborn, Diseases, Jervell and Lange-Nielsen syndrome, Jervell and Lange-Nielsen syndrome 1, Jervell and Lange-Nielsen syndrome 2, Krabbe disease, Leber congenital amaurosis 2, Leukoencephalopathies, Liddle syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, MPI-congenital disorder of glycosylation, Male Urogenital Diseases, Maple syrup urine disease, type 1A, Maple syrup urine disease, type 1B, Maple syrup urine disease, type 2, Menkes disease, Methylmalonic aciduria cblB type, Mucopolysaccharidosis I, Mucopolysaccharidosis III, Mucopolysaccharidosis Type IV A, Muscular Disorders, Atrophic, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type A, Niemann-Pick disease type C, Niemann-Pick disease, type C1, Niemann-Pick disease, type C2, Nutritional and Metabolic Diseases, Overweight, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Pitt-Hopkins syndrome, Prader-Willi syndrome, Primary Immunodeficiency Diseases, Rett syndrome, Schnyder corneal dystrophy, Sex Chromosome Disorders, Skin and Connective Tissue Diseases, Smith-Lemli-Opitz syndrome, Stickler syndrome type 1, Stickler syndrome type 2, Stress Disorders, Traumatic, Sulfatidosis, Tyrosinemia, Type I, Urogenital Abnormalities, Urogenital Diseases, Usher syndrome type 1B, Usher syndrome type 1C, Usher syndrome, type 1C, Usher syndrome, type 1F, Ventricular Outflow Obstruction, Waardenburg Syndrome, Type 2E, Waardenburg syndrome, Waardenburg syndrome type 1, Waardenburg syndrome type 2, Waardenburg syndrome type 2A, Wilson disease, Wolman disease, X-Linked Combined Immunodeficiency Diseases, X-linked Alport syndrome, X-linked disease, X-linked hypophosphatemic rickets, acrodermatitis enteropathica, acute stress disorder, adenine phosphoribosyltransferase deficiency, albinism, anemia, aortic valve disorder, aortic valve stenosis, apparent mineralocorticoid excess, arginase deficiency, argininosuccinic aciduria, autosomal recessive Alport syndrome, basal ganglia disorder, beta thalassemia, beta-ketothiolase deficiency, biotin-responsive basal ganglia disease, biotinidase deficiency, blood coagulation disease, blood platelet disease, bone disorder, brain disorder, calcium metabolic disease, carbamoyl phosphate synthetase I deficiency disease, cardiovascular disorder, carnitine palmitoyl transferase 1A deficiency, carnitine palmitoyltransferase II deficiency, carnitine-acylcarnitine translocase deficiency, central nervous system disorder, cerebrotendinous xanthomatosis, cholesteryl ester storage disease, chromosomal disorder, chronic granulomatous disease, citrullinemia, coagulation protein disease, combined immunodeficiency due to ZAP70 deficiency, congenital bile acid synthesis defect 1, congenital bile acid synthesis defect 2, congenital heart disease, congenital hypothyroidism, congenital isolated adrenocorticotropic hormone deficiency, connective tissue disorder, creatine transporter deficiency, cutaneous mucinosis, cystic fibrosis, cystinosis, demyelinating disease, diabetes mellitus, disease, disorder of glycogen metabolism, disorder of pilosebaceous unit, dopa-responsive dystonia due to sepiapterin reductase deficiency, encephalopathy due to GLUT1 deficiency, endocrine system disorder, epilepsy, epilepsy, early-onset, vitamin B6-dependent, epithelial neoplasm, eye disorder, factor VII deficiency, factor X deficiency, familial hemolytic anemia, familial isolated deficiency of vitamin E, fragile X syndrome, fructose-1,6-bisphosphatase deficiency, galactosemia, glucose metabolism disease, glutaryl-CoA dehydrogenase deficiency, glycogen storage disease I, glycogen storage disease II, glycogen storage disease IXb, glycogen storage disease IXc, glycogen storage disease Ib, guanidinoacetate methyltransferase deficiency, heart disorder, heart valve disorder, hematologic disorder, hemolytic anemia, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, hereditary fructose intolerance, hereditary neoplastic syndrome, hereditary nephritis, hereditary skin disorder, histiocytosis, holocarboxylase synthetase deficiency, homocystinuria, homocystinuria due to methylene tetrahydrofolate reductase deficiency, hyperammonemia due to N-acetylglutamate synthase deficiency, hyperhomocysteinemia, hyperinsulinemic hypoglycemia, familial, 2, hyperinsulinism, hyperinsulinism-hyperammonemia syndrome, hypoglycemia, hypophosphatasia, hypophosphatemia, hypophosphatemic rickets, hypopigmentation of the skin, hypothyroidism, hypothyroidism due to TSH receptor mutations, immune system disorder, inborn carbohydrate metabolic disorder, inborn disorder of amino acid metabolism, inborn error of immunity, inborn errors of metabolism, inborn metal metabolism disorder, inborn mitochondrial metabolism disorder, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, isovaleric acidemia, kidney disorder, leukocyte disorder, liver disorder, lung disorder, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, malignant endocrine neoplasm, maturity-onset diabetes of the young type 4, medium chain acyl-CoA dehydrogenase deficiency, metabolic disease, metachromatic leukodystrophy, methylcobalamin deficiency type cblE, methylmalonic aciduria and homocystinuria, methylmalonic aciduria and homocystinuria type cblC, methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency, methylmalonic aciduria, cblA type, mitochondrial trifunctional protein deficiency, motor neuron disorder, movement disorder, mucopolysaccharidosis, mucopolysaccharidosis type 1, mucopolysaccharidosis type 2, mucopolysaccharidosis type 3A, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, multiple carboxylase deficiency, multiple endocrine neoplasia, multiple endocrine neoplasia type 2B, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, neonatal severe primary hyperparathyroidism, neoplasm, nephritis, nervous system disorder, neurodegenerative disease, neuroepithelial neoplasm, neuromuscular disease, non-Langerhans cell histiocytosis, nutritional deficiency disease, nutritional disorder, obesity disorder, ocular cancer, oculocutaneous albinism, optic choroid disorder, ornithine aminotransferase deficiency, ornithine carbamoyltransferase deficiency, ornithine translocase deficiency, overnutrition, pancreas disorder, pancreatic agenesis 1, permanent neonatal diabetes mellitus, phagocyte bactericidal dysfunction, phenylketonuria, phosphorus metabolism disease, pituitary hormone deficiency, combined, 1, platelet storage pool deficiency, post-traumatic stress disorder, primary hyperoxaluria type 1, primary hyperoxaluria type 2, primary hyperoxaluria type 3, primitive neuroectodermal tumor, propionic acidemia, pseudohypoaldosteronism, pseudohypoaldosteronism type 1, psychiatric disorder, pyridoxal phosphate-responsive seizures, pyridoxine-dependent epilepsy, renal tubular transport disease, respiratory system disorder, retinal cancer, retinal disorder, retinoblastoma, riboflavin transporter deficiency, rickets, severe combined immunodeficiency, severe combined immunodeficiency due to DCLRE1C deficiency, severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency, sickle cell disease, skin disorder, skin pigmentation disorder, sphingolipidosis, spinal cord disorder, spinal muscular atrophy, steroid inherited metabolic disorder, sulfite oxidase deficiency due to molybdenum cofactor deficiency type A, thalassemia, thyroid dyshormonogenesis 2A, thyroid dyshormonogenesis 3, thyroid dyshormonogenesis 5, thyroid dyshormonogenesis 6, thyroid gland disorder, transcobalamin II deficiency, transient neonatal diabetes mellitus, tuberous sclerosis 1, tuberous sclerosis 2, tyrosine hydroxylase deficiency, tyrosinemia, urea cycle disorder, urinary system disorder, uveal disorder, very long chain acyl-CoA dehydrogenase deficiency, vitamin D deficiency, vitamin deficiency disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3, xanthomatosis | Active, not recruiting | Not stated | 2026-07-01 | NCT03655223 | |
| CASPALLO: Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene | Chronic Disease, 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, bone marrow disorder, hematologic disorder, histiocytic sarcoma, histiocytosis, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, myelodysplastic syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphoma, non-Langerhans cell histiocytosis | Active, not recruiting | Phase 1 | 2026-07-01 | NCT00710892 | |
| Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases | Bone Marrow Failure, Bone Marrow Failure Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lymphohistiocytosis, Hemophagocytic, Primary Immunodeficiency Diseases, bone marrow disorder, hematologic disorder, hereditary disease, histiocytosis, immune system disorder, inborn error of immunity, inherited hemoglobinopathy, lymphatic system disorder, non-Langerhans cell histiocytosis | Not yet recruiting | Phase 2 | 2026-06-22 | NCT07660783 | |
| A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, 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, sphingolipidosis | Recruiting | Phase 2 | 2026-06-18 | NCT07399704 | |
| Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | Bare Lymphocyte Syndrome, Blood Protein Disorders, Chediak-Higashi syndrome, Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, DNA repair disease, Disease Attributes, Eye Diseases, Hereditary, Griscelli syndrome, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Infant, Newborn, Diseases, Lymphohistiocytosis, Hemophagocytic, Nutritional and Metabolic Diseases, Omenn syndrome, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-Linked Lymphoproliferative Disorder, X-linked disease, albinism, blood coagulation disease, chronic granulomatous disease, common variable immunodeficiency, dysgammaglobulinemia, eye disorder, hematologic disorder, hemorrhagic disease, hereditary disease, histiocytosis, hyper-IgM syndrome, immune system disorder, immunoproliferative disorder, inborn error of immunity, inherited blood coagulation disorder, interstitial lung disease, leukocyte disorder, leukopenia, lung disorder, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, metabolic disease, non-Langerhans cell histiocytosis, phagocyte bactericidal dysfunction, respiratory system disorder, reticular dysgenesis, severe combined immunodeficiency | Active, not recruiting | Not applicable | 2026-06-17 | NCT01652092 | |
| Histiocytic Disorder Follow-up Study | Erdheim-Chester disease, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Histiocytosis, Malignant, Neoplasms by Histologic Type, Neoplasms, Second Primary, histiocytic sarcoma, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, malignant histiocytosis, neoplasm, non-Langerhans cell histiocytosis, respiratory system disorder, sinus histiocytosis with massive lymphadenopathy, xanthogranuloma | Recruiting | Not stated | 2026-06-12 | NCT05915208 |