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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
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Infection, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
ravuconazole
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nausea and Vomiting, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
Aprepitant, Cyclophosphamide, Dexamethasone, Granisetron hydrochloride
Drug
Lead sponsor
Barbara Ann Karmanos Cancer Institute
Other
Eligibility
18 Years to 120 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
Detroit, Michigan
Conditions
Cancer
Interventions
palivizumab, ribavirin
Biological · Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Cancer
Interventions
graft versus host disease prophylaxis/therapy, fluorescence in situ hybridization, immunoenzyme technique, immunohistochemistry staining method, laboratory biomarker analysis, in vitro-treated bone marrow transplantation, management of therapy complications
Biological · Genetic · Other + 1 more
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
8
States / cities
Cleveland, Ohio
Conditions
Cancer
Interventions
immunological diagnostic method, physiologic testing
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
Up to 120 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2009
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Conditions
Cancer
Interventions
infection prophylaxis and management
Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
12 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2002
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
alemtuzumab, methylprednisolone, tacrolimus
Biological · Drug
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
6
States / cities
New York, New York • Cleveland, Ohio • Portland, Oregon + 2 more
Conditions
Breast Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Testicular Germ Cell Tumor, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
busulfan, carboplatin, carmustine, cyclophosphamide, etoposide, melphalan, thiotepa, autologous bone marrow transplantation, bone marrow ablation with stem cell support, peripheral blood stem cell transplantation, radiation therapy
Drug · Procedure · Radiation
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years and older
Enrollment
451 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2013
U.S. locations
1
States / cities
Buffalo, New York
Conditions
Cancer
Interventions
itraconazole, voriconazole
Drug
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
12 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Cancer
Interventions
cytomegalovirus IE-1-specific cytotoxic T lymphocytes, cytomegalovirus pp65-specific cytotoxic T lymphocytes, therapeutic allogeneic lymphocytes, polymerase chain reaction, flow cytometry, immunological diagnostic method, laboratory biomarker analysis
Biological · Genetic · Other
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
2 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Hershey, Pennsylvania
Conditions
Adult Acute Lymphoblastic Leukemia, Adult Acute Myeloid Leukemia, Adult Burkitt Lymphoma, Adult Diffuse Large Cell Lymphoma, Adult Diffuse Mixed Cell Lymphoma, Adult Diffuse Small Cleaved Cell Lymphoma, Adult Hodgkin Lymphoma, Adult Immunoblastic Large Cell Lymphoma, Adult Lymphoblastic Lymphoma, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Breast Cancer, Chronic Eosinophilic Leukemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Cutaneous T-cell Non-Hodgkin Lymphoma, Disseminated Neuroblastoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Grade 1 Follicular Lymphoma, Grade 2 Follicular Lymphoma, Grade 3 Follicular Lymphoma, Malignant Testicular Germ Cell Tumor, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Multiple Myeloma, Mycosis Fungoides/Sezary Syndrome, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Neutropenia, Nodal Marginal Zone B-cell Lymphoma, Ovarian Epithelial Cancer, Ovarian Germ Cell Tumor, Plasma Cell Neoplasm, Poor Prognosis Metastatic Gestational Trophoblastic Tumor, Primary Myelofibrosis, Prolymphocytic Leukemia, Small Lymphocytic Lymphoma, Splenic Marginal Zone Lymphoma
Interventions
cefepime hydrochloride
Drug
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Neuroblastoma, Ovarian Cancer, Psychosocial Effects of Cancer and Its Treatment, Testicular Germ Cell Tumor
Interventions
psychosocial assessment and care
Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
21 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2004
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
flow cytometry, laboratory biomarker analysis, Data Collection
Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
1
States / cities
Nashville, Tennessee
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
recombinant interleukin-12
Biological
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
alemtuzumab, methylprednisolone, prednisone, flow cytometry, immunologic technique, pharmacological study
Biological · Drug · Other
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Cancer
Interventions
fluconazole, itraconazole
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
13 Years and older
Enrollment
578 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
2
States / cities
Seattle, Washington
Conditions
Cancer
Interventions
polymorphism analysis, laboratory biomarker analysis
Genetic · Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
Not listed
Enrollment
252 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
3
States / cities
Nashville, Tennessee
Conditions
Cancer
Interventions
acupuncture therapy, management of therapy complications, pain therapy
Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
Interventions
peripheral blood lymphocyte therapy
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Cancer
Interventions
captopril, cyclophosphamide, autologous bone marrow transplantation, peripheral blood stem cell transplantation, radiation therapy
Drug · Procedure · Radiation
Lead sponsor
Northwestern University
Other
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Breast Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Ovarian Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
PS-341, Doxil, Velcade
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2010
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Conditions
Hereditary Thyroid Gland Medullary Carcinoma, Locally Advanced Thyroid Gland Medullary Carcinoma, Multiple Endocrine Neoplasia Type 2A, Multiple Endocrine Neoplasia Type 2B, Recurrent Thyroid Gland Medullary Carcinoma, Sporadic Thyroid Gland Medullary Carcinoma, Stage III Thyroid Gland Medullary Carcinoma AJCC v7, Stage IV Thyroid Gland Medullary Carcinoma AJCC v7, Stage IVA Thyroid Gland Medullary Carcinoma AJCC v7, Stage IVB Thyroid Gland Medullary Carcinoma AJCC v7, Stage IVC Thyroid Gland Medullary Carcinoma AJCC v7
Interventions
Sorafenib Tosylate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2022
U.S. locations
3
States / cities
St Louis, Missouri • Durham, North Carolina • Columbus, Ohio
Conditions
Cancer
Interventions
therapeutic allogeneic lymphocytes
Biological
Lead sponsor
University of Medicine and Dentistry of New Jersey
Other
Eligibility
Not listed
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
New Brunswick, New Jersey