- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Myozyme® (alglucosidase alfa)
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 1 Month and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2020
- U.S. locations
- 4
- States / cities
- Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1537–1560
of 1,712
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
- Interventions
- sample collection
- Other
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- 1 Month and older
- Enrollment
- 230 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida
- Conditions
- Sickle Cell Disease, Stroke, Neurologic Manifestations, Neurological Morbidity, Sickle Cell Anemia in Children, Sickle Cell Anemia
- Interventions
- This study does not entail offering any intervention; all data collected will be based on standard of care.
- Other
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 5 Years to 26 Years
- Enrollment
- 350 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Tourette's Disorder
- Interventions
- Aripiprazole, Placebo
- Drug
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 20
- States / cities
- Little Rock, Arkansas • Sacramento, California • Santa Ana, California + 16 more
- Conditions
- Urea Cycle Disorder
- Interventions
- Diagnostic Ultrasound
- Other
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 5 Years to 60 Years
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Tourette Syndrome in Children, Tourette Syndrome in Adolescence
- Interventions
- Ecopipam
- Drug
- Lead sponsor
- Emalex Biosciences Inc.
- Industry
- Eligibility
- 6 Years to 18 Years
- Enrollment
- 124 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 53
- States / cities
- Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
- Interventions
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Sjogren-Larsson Syndrome (SLS)
- Interventions
- Not listed
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 2
- States / cities
- Omaha, Nebraska • Pittsburgh, Pennsylvania
- Conditions
- Neimann-Pick Disease
- Interventions
- Vorinostat
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Pituitary Tumor, Meningioma, Rathke Cleft Cysts, Chordoma, Chondrosarcoma, Craniopharyngioma, Encephalocele, Esthesioneuroblastoma
- Interventions
- Not listed
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 400 participants
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Argininosuccinic Aciduria, Argininosuccinate Lyase Deficiency, Urea Cycle Disorders
- Interventions
- Nitric oxide supplement, Placebo
- Dietary Supplement
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 8 Years to 64 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
- Interventions
- AJ201, Placebo
- Drug
- Lead sponsor
- AnnJi Pharmaceutical Co., Ltd.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 6
- States / cities
- Orange, California • Palo Alto, California • Jacksonville, Florida + 3 more
- Conditions
- Tourette Syndrome
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 14 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2015
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2034
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Jacksonville, Florida • Atlanta, Georgia + 10 more
- Conditions
- Friedreich Ataxia
- Interventions
- Spinal tap for CSF collection, Venipuncture for blood sample
- Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Interventions
- AEB1102
- Drug
- Lead sponsor
- Immedica Pharma AB
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 4
- States / cities
- Stanford, California • Gainsville, Florida • New York, New York + 1 more
- Conditions
- Encephalopathy, Birth Defect, Intellectual Disability, Multiple Congenital Anomaly, Metabolic Disease, Epilepsy, Neuro-Degenerative Disease, Cerebral Palsy, Developmental Delay, Developmental Defect
- Interventions
- Whole Exome Sequencing
- Diagnostic Test
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 25 Years
- Enrollment
- 529 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 4
- States / cities
- Fresno, California • Oakland, California • San Francisco, California
- Conditions
- Ataxia Telangiectasia
- Interventions
- Dexamethasone sodium phosphate
- Drug
- Lead sponsor
- Quince Therapeutics S.p.A.
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 101 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Baltimore, Maryland • New Brunswick, New Jersey + 2 more
- Conditions
- Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- Westat
- Other
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008
- U.S. locations
- 5
- States / cities
- Stanford, California • St Louis, Missouri • New York, New York + 2 more
- Conditions
- Behcet's Disease, Churg-Strauss Syndrome, Vasculitis, Central Nervous System, Giant Cell Arteritis, Wegener Granulomatosis, Henoch-Schoenlein Purpura, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
- Interventions
- Not listed
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 386 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Parkinson's Disease, Tourette's Syndrome, Tic Disorders, Dystonia, Movement Disorders
- Interventions
- Transcranial Direct Current Stimulation (tDCS), Eye-tracking Device, Pulsed vibrator, Gait Trainer Treadmill (GTT), Standard psychiatric scales SCID-I, YBOCS and SCI-OBS-lifetime and cognitive scales MMSE and MOCA, iMobility, QMAT, Treadmill Test, Arm Intellistretch Device, Transcranial Sonography (TCS), Behavioral tasks, TMS single/paired pulse, MEG, EEG, MRI techniques, Peripheral Nerve Stimulation, EMG, Trancutaneous spinal direct current stimulation (tsDCS)
- Procedure · Device · Other + 1 more
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 1,273 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
- Interventions
- Growth Hormone, Whole body Protein turnover, DEXA scan
- Drug · Procedure
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 3 Years to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Huntington's Disease
- Interventions
- Lithium, Divalproex
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2005
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland