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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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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
Aortic Stenosis Symptomatic
Interventions
Siegel Transcatheter Aortic Valve (TAVR)
Device
Lead sponsor
MiRus
Industry
Eligibility
50 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
8
States / cities
Los Angeles, California • Palo Alto, California • Naples, Florida + 5 more
Conditions
Hypertrophic Cardiomyopathy, Obstructive Hypertrophic Cardiomyopathy, Non-obstructive Hypertrophic Cardiomyopathy
Interventions
mavacamten
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
314 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
31
States / cities
Scottsdale, Arizona • Los Angeles, California • San Francisco, California + 26 more
Conditions
Non-obstructive Hypertrophic Cardiomyopathy
Interventions
Ninerafaxstat 200mg MR, Placebo
Drug
Lead sponsor
Imbria Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
22
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 18 more
Conditions
Cardiomyopathy, Hypertrophic
Interventions
MYK-224
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 80 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
12
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 9 more
Conditions
Severe Aortic Stenosis
Interventions
Medtronic CoreValve® System Transcatheter Aortic Valve Implantation (TAVI)
Device
Lead sponsor
Medtronic Cardiovascular
Industry
Eligibility
Not listed
Enrollment
2,777 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
45
States / cities
Phoenix, Arizona • Los Angeles, California • Mountain View, California + 35 more
Not listed Not applicable Interventional Results available

JENAVALVE AS EFS TRIAL: Pericardial TAVR Aortic Stenosis Study

NCT02732691
Conditions
Aortic Valve Stenosis, Heart Valve Diseases, Heart Diseases, Cardiovascular Diseases, Ventricular Outflow Obstruction
Interventions
JenaValve Pericardial TAVR System
Device
Lead sponsor
JenaValve Technology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
2
States / cities
Washington D.C., District of Columbia • New York, New York
Conditions
Aortic Stenosis
Interventions
Transfemoral TAVR, SAVR
Device
Lead sponsor
Medstar Health Research Institute
Other
Eligibility
Not listed
Enrollment
277 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
11
States / cities
Sacramento, California • San Diego, California • Washington D.C., District of Columbia + 7 more
Completed Not applicable Interventional Results available

THE PARTNER TRIAL: Placement of AoRTic TraNscathetER Valve Trial

NCT00530894
Conditions
Critical Aortic Stenosis
Interventions
Edwards SAPIEN Transcatheter Heart Valve, Surgical Valve Replacement, medical management and/or balloon aortic valvuloplasty
Device · Other
Lead sponsor
Edwards Lifesciences
Industry
Eligibility
Not listed
Enrollment
1,057 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2017
U.S. locations
22
States / cities
La Jolla, California • Los Angeles, California • Stanford, California + 17 more
Conditions
Obstructive Cardiomyopathy, Hypertrophic, Non-obstructive Hypertrophic Cardiomyopathy
Interventions
Sotagliflozin, Placebo
Drug
Lead sponsor
Lexicon Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
33
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Los Angeles, California + 28 more
Conditions
Severe, Symptomatic, Calcific Aortic Stenosis
Interventions
TAVR Implantation with SAPIEN XT, TAVR Implantation with SAPIEN
Device
Lead sponsor
Edwards Lifesciences
Industry
Eligibility
19 Years and older
Enrollment
560 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
53
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 45 more
Conditions
Cardiomyopathy, Hypertrophic
Interventions
Mavacamten, Placebo
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
12 Years to 17 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
31
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 26 more
Conditions
Obstructive Hypertrophic Cardiomyopathy (oHCM)
Interventions
Aficamten (CK-3773274) (5 mg, 10 mg, 15 mg and 20 mg), Placebo to match aficamten, Metoprolol succinate (50 mg, 100 mg, 150 mg and 200 mg), Placebo to match metoprolol succinate
Drug
Lead sponsor
Cytokinetics
Industry
Eligibility
18 Years to 85 Years
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
28
States / cities
Anchorage, Alaska • Phoenix, Arizona • La Jolla, California + 21 more
Conditions
Cardiomyopathy, Hypertrophic
Interventions
Mavacamten
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
7
States / cities
West Hollywood, California • Atlanta, Georgia • Boston, Massachusetts + 4 more
Conditions
Hypertrophic Cardiomyopathy
Interventions
Pirfenidone
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Aortic Valve Stenosis, Heart Valve Diseases, Aortic Valve Disease, Heart Disease Structural Disorder
Interventions
Navitor Transcatheter Aortic Valve Implantation (TAVI) System, Any Commercially Available Transcatheter Aortic Valve (CAV) System
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years and older
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2036
U.S. locations
67
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 58 more
Conditions
Severe Aortic Stenosis
Interventions
Medtronic CoreValve® System Transcatheter Aortic Valve Implantation (TAVI), Surgical Aortic Valve Replacement (SAVR)
Device
Lead sponsor
Medtronic Cardiovascular
Industry
Eligibility
Not listed
Enrollment
1,453 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
45
States / cities
Phoenix, Arizona • Los Angeles, California • Mountain View, California + 35 more
Conditions
Symptomatic Severe Aortic Stenosis
Interventions
TAVR Implantation with SAPIEN XT, SAVR Implantation
Device
Lead sponsor
Edwards Lifesciences
Industry
Eligibility
Not listed
Enrollment
2,032 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2024
U.S. locations
55
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 46 more
Conditions
Obstructive Hypertrophic Cardiomyopathy (oHCM)
Interventions
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg), Placebo to match aficamten
Drug
Lead sponsor
Cytokinetics
Industry
Eligibility
18 Years to 85 Years
Enrollment
282 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
48
States / cities
Anchorage, Alaska • La Jolla, California • Los Angeles, California + 39 more
Conditions
Severe Aortic Stenosis
Interventions
Medtronic CoreValve® System Transcatheter Aortic Valve Implantation (TAVI), Surgical Aortic Valve Replacement (SAVR), Medtronic CoreValve® Evolut R System Transcatheter Aortic Valve Implantation (TAVI)
Device · Procedure
Lead sponsor
Medtronic Cardiovascular
Industry
Eligibility
Not listed
Enrollment
1,746 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
65
States / cities
Phoenix, Arizona • Hollywood, California • La Jolla, California + 55 more
Conditions
Severe Aortic Stenosis
Interventions
Medtronic CoreValve® System Transcatheter Aortic Valve Implantation (TAVI)
Device
Lead sponsor
Medtronic Cardiovascular
Industry
Eligibility
Not listed
Enrollment
782 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2025
U.S. locations
43
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 33 more
Conditions
Obstructive Hypertrophic Cardiomyopathy
Interventions
mavacamten, Placebo
Drug
Lead sponsor
MyoKardia, Inc.
Industry
Eligibility
18 Years and older
Enrollment
251 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
29
States / cities
Scottsdale, Arizona • Los Angeles, California • San Francisco, California + 23 more
Conditions
Cardiomyopathy, Hypertrophic Obstructive, Left Ventricular Outflow Tract Obstruction
Interventions
MYK-461
Drug
Lead sponsor
MyoKardia, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
7
States / cities
Scottsdale, Arizona • New Haven, Connecticut • Boston, Massachusetts + 4 more
Conditions
Symptomatic Severe Aortic Stenosis
Interventions
TAVR Implantation of the THV Prosthesis
Device
Lead sponsor
Edwards Lifesciences
Industry
Eligibility
19 Years and older
Enrollment
583 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
39
States / cities
Los Angeles, California • Sacramento, California • Stanford, California + 34 more