- Conditions
- Healthy, Strabismus
- Interventions
- Not listed
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1999 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
- Interventions
- INZ-701
- Drug
- Lead sponsor
- Inozyme Pharma
- Industry
- Eligibility
- Up to 1 Year
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 3
- States / cities
- San Diego, California • Columbus, Ohio • Philadelphia, Pennsylvania
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
- Interventions
- mipomersen sodium
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 5
- States / cities
- Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more
- Conditions
- Families With Infants
- Interventions
- HealthySteps (HS), PlayReadVIP
- Behavioral
- Lead sponsor
- NYU Langone Health
- Other
- Eligibility
- Up to 6 Months
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
- Interventions
- Screening for FH
- Diagnostic Test
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
- Interventions
- anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
- Drug · Procedure
- Lead sponsor
- Fairview University Medical Center
- Other
- Eligibility
- Up to 35 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2002
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Premature Birth, NICU, Premature, Infant
- Interventions
- Group 1: Standard of Care, Group 2: NICU2HOME+ app
- Other
- Lead sponsor
- Northwestern University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 413 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2025
- U.S. locations
- 3
- States / cities
- Arlington Heights, Illinois • Chicago, Illinois • Winfield, Illinois
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
- Interventions
- Not listed
- Lead sponsor
- xCures
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Los Altos, California
- Conditions
- Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome, Duane Radial Ray Syndrome, Mobius Syndrome, Brown Syndrome, Marcus Gunn Syndrome, Strabismus Congenital, Horizontal Gaze Palsy, Horizontal Gaze Palsy With Progressive Scoliosis, Facial Palsy, Facial Paresis, Hereditary, Congenital, Third Nerve Palsy, Fourth Nerve Palsy, Sixth Nerve Palsy, Synkinesis, Ocular Motility Disorders, Levator-Medial Rectus Synkinesis, Athabaskan Brainstem Dysgenesis, Tongue Paralysis, Ninth Nerve Disorder, Fifth Nerve Palsy, Seventh Nerve Palsy, Eleventh Nerve Disorder, Twelfth Nerve Disorder, Vagus Nerve Paralysis, Moebius Sequence
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Pseudomonas Aeruginosa, Cystic Fibrosis
- Interventions
- Not listed
- Lead sponsor
- Zafer Soultan
- Other
- Eligibility
- 5 Years to 19 Years
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Syracuse, New York
- 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
- Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
- Interventions
- imatinib, TruSight Oncology
- Drug · Device
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Child Development, Child Behavior, Social Skills, Infant Development
- Interventions
- I-T CHILD
- Other
- Lead sponsor
- Yale University
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- 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
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
- Interventions
- Mipomersen Sodium
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 144 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 20
- States / cities
- Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more
- Conditions
- Obesity, Childhood
- Interventions
- Pilot Intervention to reduce SSB consumption in children and families enrolled in WIC
- Behavioral
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 6 Months to 4 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Winston-Salem, North Carolina
- Conditions
- Anxiety, Stress
- Interventions
- Participant guided peer support gathering
- Behavioral
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Pulmonary Hypertension, Familial Persistent, of the Newborn
- Interventions
- placebo, iv sildenafil
- Drug
- Lead sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
- Industry
- Eligibility
- 0 Days to 4 Days
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2020
- U.S. locations
- 20
- States / cities
- Little Rock, Arkansas • Sacramento, California • Washington D.C., District of Columbia + 8 more
- Conditions
- Prenatal Alcohol Exposure, Fetal Alcohol Spectrum Disorders
- Interventions
- Families Moving Forward Bridges
- Behavioral
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 6 Months and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 1
- States / cities
- Shoreline, Washington
- Conditions
- Time to Shelter Exit, Family Well-being, Child Well-being
- Interventions
- Large unconditional cash transfers plus voluntary peer support., Nominal cash transfers, Passive comparison group
- Behavioral
- Lead sponsor
- Vanderbilt University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Intrauterine Growth Retardation, Prematurity
- Interventions
- Newborn Individualized Developmental Care
- Behavioral
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Up to 36 Weeks
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2002
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts
- Conditions
- Premature Birth of Newborn
- Interventions
- Family Integrated Care
- Behavioral
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 33 Weeks
- Enrollment
- 347 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 6
- States / cities
- Fresno, California • La Jolla, California • Oakland, California + 3 more
- Conditions
- Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
- Interventions
- Maralixibat
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- 0 Days to 364 Days
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 7
- States / cities
- Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 4 more
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Respiratory Distress Syndrome (RDS), Sepsis, Pneumonia, Hypertension, Pulmonary
- Interventions
- Inhaled nitric oxide, Placebo
- Drug
- Lead sponsor
- NICHD Neonatal Research Network
- Network
- Eligibility
- Up to 120 Hours
- Enrollment
- 420 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2006
- U.S. locations
- 17
- States / cities
- Birmingham, Alabama • Palo Alto, California • San Diego, California + 14 more