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Showing 409–425 of 425 matching trials from the live ClinicalTrials.gov search.
Conditions
Primary Immune Deficiency
Interventions
Asceniv™
Biological
Lead sponsor
ADMA Biologics, Inc.
Industry
Eligibility
2 Years to 11 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
3
States / cities
Centennial, Colorado • Charleston, South Carolina • Salt Lake City, Utah
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
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
HIV, AIDS, Fetal Alcohol Syndrome, Alcohol Related Neurodevelopmental Disorder
Interventions
Opt-in or Opt-out testing, Focus Group
Behavioral
Lead sponsor
University of Oklahoma
Other
Eligibility
18 Years to 44 Years · Female only
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Completed No phase listed Observational Accepts healthy volunteers

Improving the Diagnosis of Common Variable Immune Deficiency

NCT03335605
Conditions
CVI - Common Variable Immunodeficiency
Interventions
Not listed
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Advanced Solid Tumors
Interventions
Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
Conditions
Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
Interventions
ADX-324 Dose Level 1, ADX-324 Dose Level 2
Drug
Lead sponsor
ADARx Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
15
States / cities
Litchfield Park, Arizona • Little Rock, Arkansas • San Diego, California + 12 more
Conditions
Hereditary Angioedema (HAE), Angioedema
Interventions
Lanadelumab 150 mg, Lanadelumab 300 mg
Drug
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
U.S. locations
14
States / cities
Scottsdale, Arizona • Santa Monica, California • Walnut Creek, California + 10 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

COVID-19 Vaccine Responses in PIDD Subjects

NCT05321407
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
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
Recruiting No phase listed Observational Accepts healthy volunteers

Evaluation of Patients With Immune Function Abnormalities

NCT00128973
Conditions
Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
3,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
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
Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
Interventions
Increlex
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 20 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Hereditary Angioedema
Interventions
C1 Esterase Inhibitor, Placebo
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
6 Years and older
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
17
States / cities
Granada Hills, California • Weston, Florida • Atlanta, Georgia + 14 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
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
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Conditions
Severe Combined Immunodeficiency Syndrome
Interventions
CD34+ cells transduced with ADA retrovir
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland