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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesBare 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 immunodeficiencyActive, not recruitingNot applicable2026-06-17NCT01652092
Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World StudyChemically-Induced Disorders, Chronic Pain, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Eye Diseases, Hereditary, Hemic and Lymphatic Diseases, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Sensation Disorders, Signs and Symptoms, Wiskott-Aldrich syndrome, X-linked disease, anxiety disorder, blood coagulation disease, cannabis dependence, color vision disorder, cone dystrophy, eye disorder, hematologic disorder, hemorrhagic disease, hereditary disease, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopenia, nervous system disorder, psychiatric disorder, substance-related disorder, vision disorderEnrolling by invitationNot stated2026-03-30NCT07362407
Immune Disorder HSCT Protocol22q11.2 deletion syndrome, Abnormalities, Multiple, Blood Protein Disorders, Bruton-type agammaglobulinemia, Cardiovascular Abnormalities, Chediak-Higashi syndrome, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Cytopenia, DNA repair disease, DiGeorge syndrome, Disease Attributes, Eye Diseases, Hereditary, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Lymphatic Abnormalities, Lymphohistiocytosis, Hemophagocytic, Musculoskeletal Abnormalities, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, albinism, autoimmune disease, autoimmune lymphoproliferative syndrome, blood coagulation disease, cardiovascular disorder, chromosomal disorder, chronic granulomatous disease, common variable immunodeficiency, congenital heart disease, dysgammaglobulinemia, endocrine system disorder, eye disorder, heart disorder, hematologic disorder, hemorrhagic disease, hereditary disease, histiocytosis, hyper-IgM syndrome, hypoparathyroidism, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, metabolic disease, musculoskeletal system disorder, non-Langerhans cell histiocytosis, parathyroid gland disorder, phagocyte bactericidal dysfunction, severe combined immunodeficiencyActive, not recruitingPhase 22026-02-19NCT01821781
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICBlood Protein Disorders, Bone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dysgammaglobulinemia, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, histiocytosis, hyper-IgM syndrome, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, leukocyte adhesion deficiency 1, leukocyte disorder, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, rheumatoid arthritis, severe combined immunodeficiency, sickle cell disease, sphingolipidosis, syndromic disease, thalassemiaRecruitingNot stated2026-01-13NCT04528355
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTBone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dyskeratosis congenita, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, hereditary skin disorder, histiocytosis, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, juvenile idiopathic arthritis, leukocyte adhesion deficiency 1, leukocyte disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, severe combined immunodeficiency, sickle cell disease, skin disorder, sphingolipidosis, syndromic disease, thalassemiaRecruitingPhase 22025-12-15NCT01962415
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency DiseasesChronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, DNA repair disease, Disease Attributes, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, blood coagulation disease, chronic granulomatous disease, common variable immunodeficiency, hematologic disorder, hemorrhagic disease, hereditary disease, hyper-IgE recurrent infection syndrome 1, autosomal dominant, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopenia, metabolic disease, phagocyte bactericidal dysfunction, severe chronic neutropenia, severe combined immunodeficiencyEnrolling by invitationPhase 1 / Phase 22025-12-15NCT01852370
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, blood coagulation disease, hematologic disorder, hemorrhagic disease, hereditary disease, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopeniaActive, not recruitingPhase 32025-09-29NCT03837483
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, blood coagulation disease, hematologic disorder, hemorrhagic disease, hereditary disease, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopeniaActive, not recruitingPhase 1 / Phase 22021-06-03NCT02333760