- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
- Interventions
- Whole-body Electrical Muscle Stimulation Exercise
- Device
- Lead sponsor
- University of Missouri-Columbia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 1
- States / cities
- Columbia, Missouri
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Showing 25–45
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- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2019
- U.S. locations
- 3
- States / cities
- Oakland, California • Minneapolis, Minnesota • New York, New York
- Conditions
- Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
- Interventions
- LSFG-NAVI
- Device
- Lead sponsor
- Randy Kardon
- Other
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Orange County
- Other
- Eligibility
- 0 Years to 99 Years
- Enrollment
- 240 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2030
- U.S. locations
- 3
- States / cities
- Orange, California • Minneapolis, Minnesota • St Louis, Missouri
- Conditions
- Disorders of Sex Development (DSD), Pediatric and Adolescent Cancers of the Genital Tract, Reproductive Endocrine Conditions in Puberty, Structural Gynecologic Conditions Including Vulvar and Vaginal Conditions
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 11,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2039
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Skin Infections, Bacterial
- Interventions
- Retapamulin Ointment, 1%
- Drug
- Lead sponsor
- GlaxoSmithKline
- Industry
- Eligibility
- 2 Months to 24 Months
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 6
- States / cities
- Tuscaloosa, Alabama • Bentonville, Arkansas • Orange, California + 3 more
- Conditions
- Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
- Interventions
- The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
- Drug
- Lead sponsor
- Daniel Benjamin
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 3,520 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2019
- U.S. locations
- 43
- States / cities
- Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more
- Conditions
- Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Andrea Gropman
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2007 – 2010
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Spinocerebellar Ataxia
- Interventions
- Error-reduction, International Cooperative Ataxia Rating Scale, Scale for the Assessment and Rating of Ataxia, Beck Depression Inventory, 2nd Ed, Stroop, Purdue Pegboard, Brief Test of Attention, 6-minute Walk, Hand Grip Dynamometer, Montreal Cognitive Assessment, Physical Performance Function, Biomechanical Assessments of Dysmetria, Neurophysiological assessment of brain activity, Biomechanical gait analysis
- Behavioral
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 21 Years to 85 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Pulmonary Artery Hypertension, Congenital Heart Disease, Structural Heart Disease
- Interventions
- MRI Heart guidewire catheterization
- Diagnostic Test
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Nemaline Myopathy
- Interventions
- Not listed
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 0 Years to 18 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 5
- States / cities
- Palo Alto, California • Bethesda, Maryland • Boston, Massachusetts + 2 more
- Conditions
- Atrial Fibrillation, Stroke, Valve Disease, Heart, Atrial Septal Defect, Patent Foramen Ovale, Mitral Valve Disease, Tricuspid Valve Disease, Aortic Valve Disease
- Interventions
- ICE Image guidance
- Device
- Lead sponsor
- Philips Clinical & Medical Affairs Global
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 155 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 5
- States / cities
- Aurora, Colorado • Chicago, Illinois • Boston, Massachusetts + 2 more
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Interventions
- Acoramidis (AG10) oral tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Autism Spectrum Disorders
- Interventions
- Not listed
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- 2 Years to 50 Years
- Enrollment
- 138 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2014
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
- Interventions
- NT-501, Sham
- Drug · Procedure
- Lead sponsor
- Neurotech Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Mucopolysaccharidoses, Carotid Disease, Cardiac Disease, Inflammation
- Interventions
- Carotid ultrasonography, Echocardiography, transthoracic, Venipuncture
- Diagnostic Test · Procedure
- Lead sponsor
- Children's Hospital of Orange County
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 1
- States / cities
- Orange, California
- Conditions
- Schizophrenia, Schizoaffective Disorder, Hallucinations
- Interventions
- functional magnetic resonance imaging
- Device
- Lead sponsor
- Vanderbilt University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2006
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Congenital Heart Disease
- Interventions
- Not listed
- Lead sponsor
- Emory University
- Other
- Eligibility
- Not listed
- Enrollment
- 224 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2010
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Infant, Premature, Brain Injuries
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 24 Months to 27 Months
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2006
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Kansas City, Missouri • Philadelphia, Pennsylvania
- Conditions
- Cystic Fibrosis, Healthy
- Interventions
- Not listed
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 3 Years to 21 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Cystic Fibrosis
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 273 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts