- Conditions
- Leukemia, Leukemia, Lymphoblastic, Lymphoma, Large B-Cell, Diffuse, Follicular Lymphoma, Lymphoma, B-Cell, Lymphoma, Follicular, Lymphoma, Non-Hodgkin, HIV Infections, HIV Associated Lymphoma, HIV Disease; Lymphoma
- Interventions
- Not listed
- Lead sponsor
- AIDS Malignancy Consortium
- Network
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
Search Results
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Showing 1–24
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Primary Immune Deficiency
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 1 Month to 100 Years
- Enrollment
- 5,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2007
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Ann Arbor, Michigan
- Conditions
- AIDS-Related Diffuse Large B-cell Lymphoma, AIDS-Related Non-Hodgkin Lymphoma, HIV Infection, Recurrent Diffuse Large B-Cell Lymphoma, Recurrent Grade 3b Follicular Lymphoma, Recurrent High Grade B-Cell Lymphoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Primary Mediastinal (Thymic) Large B-Cell Lymphoma, Recurrent Transformed B-Cell Non-Hodgkin Lymphoma, Refractory Diffuse Large B-Cell Lymphoma, Refractory Grade 3b Follicular Lymphoma, Refractory High Grade B-Cell Lymphoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Mediastinal (Thymic) Large B-Cell Lymphoma, Refractory Transformed B-Cell Non-Hodgkin Lymphoma
- Interventions
- Axicabtagene Ciloleucel, Cyclophosphamide, Fludarabine
- Biological · Drug
- Lead sponsor
- AIDS Malignancy Consortium
- Network
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 7
- States / cities
- Duarte, California • Chicago, Illinois • New York, New York + 4 more
- Conditions
- Primary Immune Deficiency Disorders, Metabolic Disease
- Interventions
- Haploidentical Hematopoietic Cell Transplantation
- Biological
- Lead sponsor
- Johns Hopkins All Children's Hospital
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- St. Petersburg, Florida
- Conditions
- Ataxia-Telangiectasia (A-T)
- Interventions
- A-T iPS cell line, Carrier patients iPS cell line
- Other
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 3 Years to 100 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Aplastic Anemia, Burkitt Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Juvenile Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Paroxysmal Nocturnal Hemoglobinuria, Peripheral T-cell Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Severe Combined Immunodeficiency, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
- Interventions
- fludarabine phosphate, melphalan, total-body irradiation, tacrolimus, mycophenolate mofetil, methotrexate, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
- Drug · Radiation · Other + 1 more
- Lead sponsor
- Roswell Park Cancer Institute
- Other
- Eligibility
- 3 Years to 75 Years
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- T-Cell Immune Deficiency Diseases, Severe Combined Immunodeficiency
- Interventions
- Busulfan, Fludarabine and ATG
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- Not listed
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Ends 2006
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Cytomegalovirus Infections, Primary Immune Deficiency Disorder
- Interventions
- viral specific cytotoxic t-lymphocytes
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 1 Month to 79 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2027
- U.S. locations
- 9
- States / cities
- Los Angeles, California • San Francisco, California • Indianapolis, Indiana + 6 more
- 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
- Conditions
- Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
- Interventions
- CD3/CD19 negative allogeneic hematopoietic stem cells
- Biological
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 5 Years to 45 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Immunodeficiencies, Immune Dysregulation Syndromes
- Interventions
- Apha/beta T and CD19+ cell depletion using CliniMACS device
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 25 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
- Interventions
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
- Drug · Other · Procedure + 1 more
- Lead sponsor
- Roswell Park Cancer Institute
- Other
- Eligibility
- 1 Year to 75 Years
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Blastic Plasmacytoid Dendritic Cell Neoplasm, Hepatosplenic T-Cell Lymphoma, HTLV-1 Infection, NK-Cell Lymphoma, Unclassifiable, Primary Systemic Anaplastic Large Cell Lymphoma, ALK-Negative, Recurrent Adult T-Cell Leukemia/Lymphoma, Recurrent Anaplastic Large Cell Lymphoma, Recurrent Angioimmunoblastic T-cell Lymphoma, Recurrent Enteropathy-Associated T-Cell Lymphoma, Recurrent Mycosis Fungoides, Refractory Adult T-Cell Leukemia/Lymphoma, Refractory Anaplastic Large Cell Lymphoma, Refractory Angioimmunoblastic T-cell Lymphoma, Refractory Enteropathy-Associated T-Cell Lymphoma, Refractory Mycosis Fungoides, Refractory Nasal Type Extranodal NK/T-Cell Lymphoma, Refractory Peripheral T-Cell Lymphoma, Not Otherwise Specified
- Interventions
- Laboratory Biomarker Analysis, Nivolumab
- Other · Biological
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Cytomegalovirus Infections, Adenovirus Infection, EBV Infection
- Interventions
- Virus Specific T-cell (VST) infusion
- Biological
- Lead sponsor
- Pediatric Transplantation & Cellular Therapy Consortium
- Other
- Eligibility
- Not listed
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 30
- States / cities
- Phoenix, Arizona • Duarte, California • Los Angeles, California + 24 more
- Conditions
- Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD), EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications, EBV+ Sarcomas, Leiomyosarcoma
- Interventions
- Tabelecleucel
- Biological
- Lead sponsor
- Pierre Fabre Medicament
- Industry
- Eligibility
- Not listed
- Enrollment
- 190 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 23
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 18 more
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- Gene-Transduced Autologous CD34+ Stem Cells
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Months to 20 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
- Interventions
- Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 1 Month to 25 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
- Interventions
- BPX-501 and Rimiducid
- Biological
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 4 Months to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
- Interventions
- Cultured Thymus Tissue for Implantation (CTTI)
- Biological
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1991 – 2017
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
- Interventions
- Campath -1H, Fludarabine, Anti-CD45, Stem cell infusion
- Biological · Drug · Procedure
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- X-linked Agammaglobulinemia, XLA, Primary Immune Deficiency, CVID, Common Variable Immunodeficiency, Primary Antibody Deficiencies, Secondary Hypogammaglobulinemia
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- 6 Months and older
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2026
- U.S. locations
- 3
- States / cities
- St. Petersburg, Florida • Chapel Hill, North Carolina • Durham, North Carolina
- Conditions
- Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
- Interventions
- Norovirus -specific T-cell (NST) therapy
- Biological
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 3 Months to 80 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 3
- States / cities
- Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland
- Conditions
- Ebola Virus Disease, Marburg Virus Disease
- Interventions
- cAd3-Marburg, cAd3-EBO-S
- Biological
- Lead sponsor
- Albert B. Sabin Vaccine Institute
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021
- U.S. locations
- 1
- States / cities
- Oklahoma City, Oklahoma