- Conditions
- Fabry Disease
- Interventions
- Enzyme Replacement Therapy (ERT)
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 82 participants
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- 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
- Ischemic Stroke, Patent Foramen Ovale
- Interventions
- Warfarin, Aspirin
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 30 Years to 85 Years
- Enrollment
- 630 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2000
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Major Depression, Mania, Anxiety Disorders, Psychotic Disorder, Alcohol Dependence, Drug Dependence, Eating Disorders, Suicidality, Dysthymia, ADHD, Tourettes Disorder, Conduct Disorder, Oppositional Defiant Disorder, Pervasive Developmental Disorder
- Interventions
- MINI-KID
- Other
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 230 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2008
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Alzheimer Disease, Alzheimer Dementia, Vascular Dementia, Lewy Body Disease, Lewy Body Dementia With Behavioral Disturbance (Disorder), Dementia, Mixed, Parkinson-Dementia Syndrome, Chronic Traumatic Encephalopathy, Huntington's Dementia, Wernicke Korsakoff Syndrome, Traumatic Brain Injury, Dementia, Multi-Infarct, Autism, Autism Spectrum Disorder, Autistic Behavior, Autistic Disorder, Current or Active State, Cadasil, LATE Limbic-predominant Age-related TDP-43 Encephalopathy
- Interventions
- Intravenous Bone Marrow Stem Cell (BMSC) Fraction, Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction, Near Infrared Light
- Procedure
- Lead sponsor
- MD Stem Cells
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 2
- States / cities
- Westport, Connecticut • Coral Springs, Florida
- Conditions
- Tourette Syndrome
- Interventions
- Thalamic deep brain stimulation
- Device
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Ornithine Transcarbamylase (OTC) Deficiency
- Interventions
- scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
- Genetic · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Friedreich Ataxia
- Interventions
- Idebenone
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Solid Tumors
- Interventions
- thioureidobutyronitrile
- Drug
- Lead sponsor
- Cellceutix Corporation
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
- Interventions
- Not listed
- Lead sponsor
- University of Miami
- Other
- Eligibility
- Not listed
- Enrollment
- 708 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2031
- U.S. locations
- 13
- States / cities
- Palo Alto, California • San Diego, California • San Francisco, California + 10 more
- Conditions
- CCM, Cavernoma, Cerebral Cavernous Malformation, Cerebral Cavernous Malformations 1, Cerebral Cavernous Malformations 2, Cerebral Cavernous Malformations 3, Cavernous Angioma
- Interventions
- Not listed
- Lead sponsor
- University of Chicago
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 123 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- Tafamidis
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1,733 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2023
- U.S. locations
- 41
- States / cities
- Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more
- Conditions
- Phenylketonuria, Maternal
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Female only
- Enrollment
- 50 participants
- Timeline
- 2022 – 2032
- U.S. locations
- 1
- States / cities
- Wilmington, North Carolina
- Conditions
- Parkinson Disease, Huntington Disease, Autistic Spectrum Disorders, Fragile X Syndrome, Alzheimer Disease, Mild Cognitive Impairment
- Interventions
- [18F]FPEB
- Drug
- Lead sponsor
- Institute for Neurodegenerative Disorders
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2016
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
- Interventions
- acoramidis, Placebo Oral Tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 632 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 40
- States / cities
- Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
- Conditions
- Parkinson's Disease, Gaucher's Disease
- Interventions
- N-acetylcysteine
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Tourette Syndrome, Tic Disorders
- Interventions
- Habit reversal therapy, Supportive therapy
- Behavioral
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 16 Years and older
- Enrollment
- 122 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2010
- U.S. locations
- 3
- States / cities
- New Haven, Connecticut • Boston, Massachusetts • San Antonio, Texas
- Conditions
- Generalized Epilepsy, Infantile Spasms, Metabolic Disease, Partial Epilepsy, Seizures
- Interventions
- 18 FDG
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Adrenoleukodystrophy
- Interventions
- MIN-102, Placebos
- Drug
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 105 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2025
- U.S. locations
- 3
- States / cities
- Stanford, California • Baltimore, Maryland • Boston, Massachusetts
- Conditions
- Fragile X Syndrome, Healthy Volunteers
- Interventions
- [15-O]water
- Drug
- Lead sponsor
- National Institute of Mental Health (NIMH)
- NIH
- Eligibility
- 18 Years to 24 Years · Male only
- Enrollment
- 147 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2020
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Brain Arteriovenous Malformation, Brain Vascular Malformations, Vein of Galen Malformation
- Interventions
- Apollo Micro Catheter device
- Device
- Lead sponsor
- St. Luke's-Roosevelt Hospital Center
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- Alglucosidase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003
- U.S. locations
- 1
- States / cities
- New Brunswick, New Jersey
- Conditions
- Lysosomal Acid Lipase Deficiency, Wolman Disease
- Interventions
- Sebelipase alfa (SBC-102)
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2018
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2021
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • Decatur, Georgia • Detroit, Michigan