Clinical Trials

46 trials
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Genetic Diseases, Inborn × Clear all
NCT04880356 2024-11-19

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Recruiting
100 enrolled

2-Hydroxyglutaricaciduria, Fahr's disease, Polycystic lipomembranous osteodysplasia with sclerosing leukoencephalopathy, Methylmalonic acidemia with homocystinuria, Undiagnosed Diseases, Adrenoleukodystrophy, Cysts, Hepatolenticular Degeneration, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Metabolic Diseases, Nervous System Diseases, Sphingolipidoses, Xanthomatosis, Cerebrotendinous, Pelizaeus-Merzbacher Disease, Genetic Diseases, Inborn, Rare Diseases, Alexander Disease, Leukoencephalopathies

NCT00019630 2024-03-04

Liposomal Doxorubicin in Treating Children With Refractory Solid Tumors

National Cancer Institute (NCI)

Phase 1 Completed
CNSGastrointestinalGenitourinaryGynecologicSarcoma

Familial ependymoma, Ovarian Germ Cell Cancer, Astrocytoma, Bone Neoplasms, Brain Neoplasms, Carcinoma, Renal Cell, Endocrine Gland Neoplasms, Gastrointestinal Neoplasms, Carcinoma, Hepatocellular, Medulloblastoma, Meningioma, Neoplasms, Wilms Tumor, Neuroblastoma, Oligodendroglioma, Pinealoma, Osteosarcoma, Teratoma, Testicular Neoplasms, Urologic Neoplasms, Central Nervous System Neoplasms, Choroid Plexus Neoplasms, Neuroectodermal Tumors, Primitive, Peripheral, Muscle Neoplasms, Optic Nerve Glioma, Genetic Diseases, Inborn, Histiocytoma, Malignant Fibrous

NCT00020267 2024-03-04

Vaccine Therapy in Treating Patients With Metastatic Cancer

National Cancer Institute (NCI)

Phase 1 Completed
BreastDermatologicGastrointestinalSarcomaThoracic

Uveal Melanoma, Bone Neoplasms, Breast Neoplasms, Carcinoma, Non-Small-Cell Lung, Colonic Neoplasms, Eye Neoplasms, Gastrointestinal Neoplasms, Lung Neoplasms, Melanoma, Neoplasms, Rectal Neoplasms, Recurrence, Sarcoma, Osteosarcoma, Skin Neoplasms, Colorectal Neoplasms, Neuroectodermal Tumors, Primitive, Peripheral, Breast Neoplasms, Male, Muscle Neoplasms, Genetic Diseases, Inborn, Histiocytoma, Malignant Fibrous

NCT01200017 2021-05-27

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

University of California, San Francisco

No longer available
Classical hematologyLymphoidMyeloidOther hematologic neoplasmOther neoplasm

Bone Marrow Failure Disorders, Cytopenia, Bone Diseases, Bone Diseases, Developmental, Bone Marrow Diseases, Graft vs Host Disease, Hematologic Diseases, Hemoglobinopathies, Immunologic Deficiency Syndromes, Immune System Diseases, Immunoproliferative Disorders, Leukemia, Leukemia, Lymphoid, Leukemia, Myeloid, Leukocyte Disorders, Lymphatic Diseases, Lymphoma, Lymphoproliferative Disorders, Musculoskeletal Diseases, Myelodysplastic Syndromes, Myeloproliferative Disorders, Neoplasms, Neoplasms by Histologic Type, Osteochondrodysplasias, Osteopetrosis, Osteosclerosis, Pancytopenia, Precancerous Conditions, Preleukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Genetic Diseases, Inborn, Precursor Cell Lymphoblastic Leukemia-Lymphoma

NCT00005900 2005-06-24

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Office of Rare Diseases (ORD)

Unknown
10 enrolled

Adrenoleukodystrophy, Fucosidosis, Gaucher Disease, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Mucopolysaccharidosis I, Metabolism, Inborn Errors, Mucolipidoses, Mucopolysaccharidoses, Mucopolysaccharidosis VI, Niemann-Pick Diseases, Sphingolipidoses, Wolman Disease, Genetic Diseases, Inborn, Rare Diseases, Mannosidase Deficiency Diseases