- Conditions
- Angioedema, Angioedemas, Hereditary, Urticaria, Mastocytosis, ACE Inhibitor-Induced Angioedema, C1 Inhibitor Deficiency, Systemic Mastocytoses, Indolent Systemic Mastocytosis
- Interventions
- Venipuncture
- Diagnostic Test
- Lead sponsor
- Foundation For Rare Disease Research
- Other
- Eligibility
- 12 Years to 75 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2027
- U.S. locations
- 1
- States / cities
- Wheaton, Maryland
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 73–96
of 396
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic 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 Langerhans Cell Histiocytosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Aggressive NK-cell Leukemia, AIDS-related Diffuse Large Cell Lymphoma, AIDS-related Diffuse Mixed Cell Lymphoma, AIDS-related Diffuse Small Cleaved Cell Lymphoma, AIDS-related Immunoblastic Large Cell Lymphoma, AIDS-related Lymphoblastic Lymphoma, AIDS-related Malignancies, AIDS-related Small Noncleaved Cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Clear Cell Renal Cell Carcinoma, Cutaneous B-cell Non-Hodgkin Lymphoma, de Novo Myelodysplastic Syndromes, Essential Thrombocythemia, Extramedullary Plasmacytoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, HIV Infection, HIV-associated Hodgkin Lymphoma, Intraocular Lymphoma, Isolated Plasmacytoma of Bone, Light Chain Deposition Disease, Mast Cell Leukemia, Myelodysplastic Syndrome With Isolated Del(5q), Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Myeloid/NK-cell Acute Leukemia, Nodal Marginal Zone B-cell Lymphoma, Noncutaneous Extranodal Lymphoma, Osteolytic Lesions of Multiple Myeloma, Peripheral T-cell Lymphoma, Plasma Cell Neoplasm, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Primary Systemic Amyloidosis, Progressive Hairy Cell Leukemia, Initial Treatment, Prolymphocytic Leukemia, 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 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 Renal Cell Cancer, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Relapsing Chronic Myelogenous Leukemia, Stage IV Renal Cell Cancer, T-cell Large Granular Lymphocyte Leukemia, Testicular Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific, Waldenström Macroglobulinemia
- Interventions
- sunitinib malate, pharmacological study, laboratory biomarker analysis
- Drug · Other
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2009
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
- Conditions
- Hereditary Angioedema
- Interventions
- rhC1INH
- Drug
- Lead sponsor
- Pharming Technologies B.V.
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 152 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Hereditary Angioedema
- Interventions
- C1 esterase inhibitor [human] (C1INH-nf), Placebo (saline)
- Biological · Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 37
- States / cities
- Hoover, Alabama • Scottsdale, Arizona • Los Angeles, California + 34 more
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 100 Years
- Enrollment
- 999 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2099
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hereditary Angioedema (HAE)
- Interventions
- navenibart, Placebo
- Drug
- Lead sponsor
- Astria Therapeutics, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 157 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 15 more
- Conditions
- Ataxia Telangiectasia
- Interventions
- Dexamethasone sodium phosphate, Placebo
- Drug · Other
- Lead sponsor
- Quince Therapeutics S.p.A.
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 105 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
- Conditions
- Chronic Granulomatous Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 3 Years and older
- Enrollment
- 137 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2022
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- Stem cell transplant, Filgrastim, Alemtuzumab, Miltenyi CliniMACS
- Procedure · Drug · Device
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Up to 2 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2007
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Ataxia Telangiectasia
- Interventions
- Antisense oligonucleotide targeting the ATM gene
- Drug
- Lead sponsor
- Timothy Yu
- Other
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2036
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Hereditary Angioedema
- Interventions
- Factor XIIa antagonist monoclonal antibody, Placebo
- Biological · Drug
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 7
- States / cities
- Orange, California • Walnut Creek, California • Centennial, Colorado + 4 more
- Conditions
- Primary Immunodeficiency Disease
- Interventions
- Kedrion IVIG 10%
- Biological
- Lead sponsor
- Kedrion S.p.A.
- Industry
- Eligibility
- 2 Years to 70 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 11
- States / cities
- North Palm Beach, Florida • Chicago, Illinois • Overland Park, Kansas + 7 more
- Conditions
- Hereditary Angioedema
- Interventions
- Donidalorsen
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 154 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 16
- States / cities
- Paradise Valley, Arizona • La Jolla, California • Santa Monica, California + 13 more
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 13 Years to 17 Years · Female only
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hereditary Angioedema
- Interventions
- STAR-0215 (SC), Placebo (SC), STAR-0215 (IV), Placebo (IV)
- Drug
- Lead sponsor
- Astria Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 41 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- West Bend, Wisconsin
- Conditions
- Chediak-Higashi Syndrome
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 70 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Primary Immunodeficiency Diseases (PID)
- Interventions
- TAK-881, HYQVIA
- Biological
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 15
- States / cities
- Irvine, California • Walnut Creek, California • Denver, Colorado + 12 more
- Conditions
- Psuedomas Infection, Cystic Fibrosis
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 9 Years to 80 Years
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2018
- U.S. locations
- 4
- States / cities
- Bethesda, Maryland • Seattle, Washington • Madison, Wisconsin + 1 more
- Conditions
- Waning Donor Chimerism, Waning Immune Function, Primary Immunodeficiency Disease(s), Bone Marrow Failure
- Interventions
- CD34+
- Biological
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- Up to 35 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2018
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Hereditary Angioedema (HAE)
- Interventions
- DX-2930, Placebo
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 12
- States / cities
- San Diego, California • Walnut Creek, California • Tampa, Florida + 9 more
- Conditions
- Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
- Interventions
- autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
- Biological
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- Not listed
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2040
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- x Linked Combined Immunodeficiency
- Interventions
- CliniMACs
- Device
- Lead sponsor
- University of Miami
- Other
- Eligibility
- Up to 65 Years
- U.S. locations
- 1
- States / cities
- Miami, Florida
- Conditions
- Primary Immunodeficiency, DOCK8, Virus Susceptibility
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 2 Years to 85 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2009
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland