Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 22, 2026, 11:58 AM EDT

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Showing 1–24 of 191 matching trials from the live ClinicalTrials.gov search.
Conditions
Thalassemia, Sickle Cell Disease, Healthy Controls
Interventions
Hydroxyurea, Acetazolamide
Drug
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
7 Years and older
Enrollment
165 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Dementia, Vascular
Interventions
Donepezil Hydrochloride
Drug
Lead sponsor
Eisai Inc.
Industry
Eligibility
41 Years and older
Enrollment
974 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Hot Springs, Arkansas • St. Petersburg, Florida • Peoria, Illinois + 2 more
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
Natural History Observation
Other
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 1 more
No Longer Available No phase listed Expanded access

Single Patient Expanded Access Protocol: Metabolic Boost

NCT02021266
Conditions
Metachromatic Leukodystrophy
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Female only
U.S. locations
1
States / cities
Durham, North Carolina
Completed Not applicable Interventional Accepts healthy volunteers Results available

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

NCT01594853
Conditions
X-linked Adrenoleukodystrophy
Interventions
exercise training
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
21 Years to 70 Years · Female only
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Not listed Phase 2 Interventional Accepts healthy volunteers

n-3 PUFA for Vascular Cognitive Aging

NCT01953705
Conditions
Age Related Cognitive Decline, Alzheimer's Disease, Vascular Dementia, Endothelial Dysfunction, Executive Dysfunction
Interventions
Omega 3 PUFA, Placebo
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
75 Years and older
Enrollment
102 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2021
U.S. locations
1
States / cities
Portland, Oregon
Terminated No phase listed Observational Accepts healthy volunteers

Alpers Huttenlocher Natural History Study

NCT03034512
Conditions
Alpers Huttenlocher Syndrome
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2022
U.S. locations
2
States / cities
New York, New York • Seattle, Washington
Conditions
Metachromatic Leukodystrophy
Interventions
Warfarin
Drug
Lead sponsor
The Cooper Health System
Other
Eligibility
1 Year to 10 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Camden, New Jersey
Recruiting No phase listed Observational Accepts healthy volunteers

Longitudinal Cognitive Assessment by BoCA

NCT04114994
Conditions
Alzheimer Disease, Mild Cognitive Impairment, Vascular Dementia, Frontotemporal Dementia, Parkinson Disease, Multiple Sclerosis, TBI
Interventions
Boston Cognitive Assessment (BoCA)
Diagnostic Test
Lead sponsor
Alzheimer's Light LLC
Industry
Eligibility
50 Years and older
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2029
U.S. locations
1
States / cities
Miami, Florida
Conditions
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
Interventions
Patient Registry, Dry blood spots sampling
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Diego, California
Conditions
Cerebrovascular Disease, Ischemic Stroke, Transient Ischemic Attack, Intracerebral Hemorrhage, Aneurysmal Subarachnoid Hemorrhage, Vascular Dementia, Anoxic Brain Injury, Unruptured Intracranial Aneurysm, Carotid Artery Stenosis Symptomatic, Asymptomatic Carotid Artery Stenosis, Non-Aneurysmal Perimesencephalic Subarachnoid Haemorrhage, Cerebral Venous Thrombosis, Moyamoya Disease, Fibromuscular Dysplasia, Subarachnoid Hemorrhage, Leukoaraiosis, Arteriovenous Fistula, Reversible Cerebral Vasoconstriction Syndrome, CADASIL
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Jacksonville, Florida
Conditions
Canavan Disease
Interventions
AAV9 BBP-812
Biological
Lead sponsor
Aspa Therapeutics
Industry
Eligibility
Up to 30 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
ALSP
Interventions
VGL101
Drug
Lead sponsor
Vigil Neuroscience, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
5
States / cities
San Francisco, California • Englewood, Colorado • Jacksonville, Florida + 2 more
Conditions
Leukoencephalopathies, LBSL, Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, White Matter Disease, Ataxia, Cerebellar, Genetic Disease
Interventions
Not listed
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
New York, New York
Completed Phase 3 Interventional Accepts healthy volunteers Results available

The Healthy Patterns Sleep Study

NCT03682185
Conditions
Dementia, Alzheimer Disease, Circadian Rhythm Disorders, Circadian Rhythm Sleep Disorder, Insomnia, Hypersomnia, Cognitive Impairment, Cognitive Decline, Mild Cognitive Impairment, Frontotemporal Dementia, Neurocognitive Disorders, Vascular Dementia, Sleep Disorder, Memory Impairment
Interventions
Attention-Control Condition, Timed Activity Intervention
Behavioral
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 110 Years
Enrollment
421 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Pelizaeus-Merzbacher Disease
Interventions
HuCNS-SC cells
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 5 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Memory Disorders, Alzheimer Disease, Dementia, Cognitive Impairment, Frontotemporal Dementia
Interventions
COGNISION™ System
Device
Lead sponsor
Neuronetrix, Inc.
Industry
Eligibility
60 Years to 90 Years
Enrollment
204 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2014
U.S. locations
7
States / cities
West Palm Beach, Florida • Lexington, Kentucky • Louisville, Kentucky + 4 more
Conditions
Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD), Nervous System Diseases, Brain Diseases, Central Nervous System Diseases, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Sphingolipidoses, Hereditary Central Nervous System Demyelinating Diseases, Metabolic Inborn Brain Diseases, Lysosomal Storage Diseases, Metabolic Diseases, Sulfatidosis
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Up to 12 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
Torrance, California • Washington D.C., District of Columbia • Chicago, Illinois + 1 more
Conditions
Stroke, Vascular Dementia, Memory Deficits
Interventions
Donepezil and self-generated memory training, Donepezil and experimenter-administered memory training
Drug
Lead sponsor
Kessler Foundation
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2021
U.S. locations
1
States / cities
West Orange, New Jersey
Conditions
Dementia, Vascular, Stroke Sequelae
Interventions
Home telerehabilitation therapy
Other
Lead sponsor
The Methodist Hospital Research Institute
Other
Eligibility
18 Years to 110 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Houston, Texas