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Showing 25–48 of 59 matching trials from the live ClinicalTrials.gov search.
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
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Phase 1 Safety and Immunogenicity of Meningococcal Vaccine

NCT00678652
Conditions
Meningococcal Infection, Group B
Interventions
10 μg Group B Meningococcal 8570 HOPS-G NOMV Vaccine, 25 μg Group B Meningococcal 8570 HOPS-G NOMV Vaccine, 50 μg Group B Meningococcal 8570 HOPS-G NOMV Vaccine, 75 μg Group B Meningococcal 8570 HOPS-G NOMV Vaccine
Biological
Lead sponsor
U.S. Army Medical Research and Development Command
Federal
Eligibility
18 Years to 45 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Silver Spring, Maryland
Conditions
Rare Kidney Stone Diseases
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
6,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Terminated Phase 1 Interventional Accepts healthy volunteers

Ph1 Marinol Interaction Study - Part 1 - 1

NCT00438139
Conditions
Marijuana Dependence
Interventions
Dronabinol
Drug
Lead sponsor
National Institute on Drug Abuse (NIDA)
NIH
Eligibility
18 Years to 45 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
HER2 Negative Metastatic Breast Cancer
Interventions
ADI-PEG 20
Drug
Lead sponsor
Polaris Group
Industry
Eligibility
18 Years and older · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Houston, Texas
Completed Phase 1 Interventional Accepts healthy volunteers

Surufatinib DDI With a PPI and a CYP3A Inducer

NCT04510649
Conditions
Healthy
Interventions
Part A, Part B
Drug
Lead sponsor
Hutchison Medipharma Limited
Industry
Eligibility
18 Years to 55 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Cypress, California
Recruiting No phase listed Observational

Rare Kidney Stone Consortium Biobank

NCT02026388
Conditions
Primary Hyperoxaluria, Dent Disease, APRT Deficiency, Cystinuria
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota
Not listed Phase 1 Interventional Accepts healthy volunteers

Ph1 Lobeline Interaction Study - 1

NCT00439504
Conditions
Methamphetamine Dependence
Interventions
Lobeline
Drug
Lead sponsor
National Institute on Drug Abuse (NIDA)
NIH
Eligibility
18 Years to 45 Years · Female only
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
U.S. locations
1
States / cities
San Francisco, California
Conditions
Metastatic Breast Cancer
Interventions
PMD-026, fulvestrant
Drug
Lead sponsor
Phoenix Molecular Designs
Industry
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
10
States / cities
Gilbert, Arizona • Duarte, California • Irvine, California + 7 more
Conditions
Influenza, Pandemic
Interventions
BW-1014: 25 µg rH5 in 20% NE - pipette - IN, BW-1014: 50 µg rH5 in 20% NE - pipette - IN, BW-1014: 100 µg rH5 in 20% NE - pipette - IN, rH5 (100 µg) control - pipette - IN, Saline (Placebo) - pipette - IN, H5N1 IIV - IM
Biological
Lead sponsor
BlueWillow Biologics
Industry
Eligibility
18 Years to 45 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Anemia in Myelodysplastic Syndromes
Interventions
TP-0184
Drug
Lead sponsor
Sumitomo Pharma America, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
3
States / cities
Plantation, Florida • The Bronx, New York • Austin, Texas
Conditions
Solid Tumor
Interventions
STP707
Drug
Lead sponsor
Sirnaomics
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
9
States / cities
Phoenix, Arizona • Los Angeles, California • Denver, Colorado + 6 more
Conditions
Pancreatic Cancer
Interventions
Part B, Cohort 1- VS-4718, nab-paclitaxel, gemcitabine, Part B, Cohort 2- VS4718, nab-paclitaxel, gemcitabine, Part A- VS-4718, nab-paclitaxel, gemcitabine
Drug
Lead sponsor
Verastem, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
St Louis, Missouri • Gettysburg, Pennsylvania
Conditions
Pre-exposure Prophylaxis Against Pneumonic Plague in Healthy Adults
Interventions
Monoclonal antibody JST-010, Placebo
Biological · Drug
Lead sponsor
Just-Evotec Biologics
Industry
Eligibility
18 Years to 55 Years
Enrollment
104 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Las Vegas, Nevada
Conditions
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease, Primary Hyperoxaluria Type 3 (PH3)
Interventions
DCR-PHXC
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
4
States / cities
San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 1 more
Conditions
Malignant Glioma (WHO Grade III or IV)
Interventions
CTO, Lomustine
Drug
Lead sponsor
Annick Desjardins
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Acute Myelogenous Leukemia, B-cell Acute Lymphoblastic Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm, Chronic Myeloid Leukemia, Blast Crisis
Interventions
XmAb14045
Biological
Lead sponsor
Xencor, Inc.
Industry
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
9
States / cities
Jacksonville, Florida • Atlanta, Georgia • Chicago, Illinois + 3 more
Conditions
Non Small Cell Lung Cancer, Triple Negative Breast Cancer, Melanoma, Head and Neck Cancer
Interventions
CAB-ROR2-ADC, PD-1 inhibitor
Biological
Lead sponsor
BioAtla, Inc.
Industry
Eligibility
18 Years and older
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
38
States / cities
Tucson, Arizona • Duarte, California • La Jolla, California + 30 more
Conditions
Cystic Fibrosis, Pseudomonas Aeruginosa, Pseudomonas, Lung Infection, Lung Infection Pseudomonal
Interventions
AP-PA02, Placebo
Biological · Other
Lead sponsor
Armata Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
20
States / cities
Los Angeles, California • Tampa, Florida • Boise, Idaho + 16 more
Conditions
AML/MDS
Interventions
Mana 312
Biological
Lead sponsor
Mana Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
8
States / cities
Denver, Colorado • Atlanta, Georgia • Kansas City, Kansas + 4 more
Conditions
Primary Hyperoxaluria, Dent Disease, Cystinuria, APRT Deficiency
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
0 Years to 100 Years
Enrollment
730 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2028
U.S. locations
3
States / cities
Rochester, Minnesota • New York, New York
Conditions
Primary Hyperoxaluria Type 1, End Stage Renal Disease
Interventions
DCR-PHXC
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 1 more
Conditions
Ovarian Tumors
Interventions
GRN-300, Paclitaxel
Drug
Lead sponsor
Green3Bio, Inc.
Industry
Eligibility
18 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Houston, Texas
Suspended Phase 1 Interventional Accepts healthy volunteers

Ph1 Study of Valortim and Ciprofloxacin in Humans

NCT00964561
Conditions
Anthrax
Interventions
Ciprofloxacin and Valortim, Placebo Antibiotic and Valortim, Placebo Antibiotic and Placebo Valortim
Drug · Other
Lead sponsor
PharmAthene, Inc.
Industry
Eligibility
18 Years to 59 Years
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
1
States / cities
Overland Park, Kansas