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ClinicalTrials.gov public records Last synced Oct 11, 2026, 10:02 AM EDT

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Showing 169–192 of 1,707 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 3 Interventional Results available

Safety and Tolerability of Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder

NCT01727713
View directory record Official record
Conditions
Tourette's Disorder, Tic Disorder
Interventions
Aripiprazole
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 18 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
26
States / cities
Dothan, Alabama • Goodyear, Arizona • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2015 · Synced Oct 11, 2026, 10:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Electrical Impedance Myography (EIM) in ALS

NCT02011204
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
Interventions
Electrical impedance myography (EIM)
Device
Lead sponsor
Skulpt, Inc.
Industry
Eligibility
35 Years to 80 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 11, 2016 · Synced Oct 11, 2026, 10:02 AM EDT
Recruiting Phase 1Phase 2 Interventional

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

NCT07429240
View directory record Official record
Conditions
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Interventions
PBGENE-DMD (IV)
Biological
Lead sponsor
Precision BioSciences, Inc.
Industry
Eligibility
2 Years to 7 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Little Rock, Arkansas • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 11, 2026, 10:02 AM EDT
Completed No phase listed Observational

Research Study on Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL)

NCT01114815
View directory record Official record
Conditions
CADASIL
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2013 · Synced Oct 11, 2026, 10:02 AM EDT
Withdrawn Phase 1 Interventional

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

NCT01787578
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
Interventions
Sobetirome
Drug
Lead sponsor
Thomas S. Scanlan
Other
Eligibility
18 Years to 64 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 14, 2014 · Synced Oct 11, 2026, 10:02 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Investigation of Neurofeedback With Real-Time fMRI in Healthy Volunteers and Patients With Hyperkinetic Movement Disorders

NCT00885040
View directory record Official record
Conditions
Movement Disorder, Tourette Syndrome, Healthy Volunteer
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 60 Years
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2019 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 15, 2023 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

NCT01678898
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102
Drug
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
9
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 11, 2026, 10:02 AM EDT
Completed No phase listed Observational

Pregnancy and Birth Outcome in Women With Pompe Disease

NCT01556516
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
18 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2016 · Synced Oct 11, 2026, 10:02 AM EDT
Active, not recruiting Phase 2 Interventional

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

NCT02171104
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Not applicable Interventional Results available

Transcranial Magnetic Stimulation in Spino-Cerebellar Ataxia

NCT01975909
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Transcranial Magnetic Stimulation
Device
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 11, 2026, 10:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of Data Collected From Individuals Administered Neurobehavioral Assessments

NCT01445314
View directory record Official record
Conditions
HIV, Brain Tumors, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
598 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 16, 2024 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 11, 2026, 10:02 AM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Brain Structure and Function in Juvenile-onset Huntington's Disease

NCT05707663
View directory record Official record
Conditions
Juvenile Huntington Disease, Juvenile-Onset Huntington Disease
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
4 Years to 30 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Sacramento, California • Iowa City, Iowa • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 2 Interventional Results available

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

NCT01419639
View directory record Official record
Conditions
Neurofibromatosis Type II
Interventions
Everolimus (RAD001) , Afinitor®
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
3 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 18, 2017 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

NCT02111200
View directory record Official record
Conditions
Urea Cycle Disorders, Inborn
Interventions
Sodium Benzoate, Sodium Phenylbutyrate
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 64 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 9, 2018 · Synced Oct 11, 2026, 10:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD)

NCT04828863
View directory record Official record
Conditions
Maple Syrup Urine Disease
Interventions
Neurocognitive testing
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
21 Years to 80 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 2 Interventional Results available

HSCT for High Risk Inherited Inborn Errors

NCT00383448
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
Interventions
Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
Drug · Procedure · Biological + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 70 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Oct 11, 2026, 10:02 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 2 Interventional Results available

Impact of Probiotics on Urinary Symptoms in Spinal Cord Injury SCI and SB

NCT02748317
View directory record Official record
Conditions
Lower Urinary Tract Symptoms
Interventions
Lactobacillus rhamnosus GG
Drug
Lead sponsor
Medstar Health Research Institute
Other
Eligibility
18 Years and older
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 12, 2020 · Synced Oct 11, 2026, 10:02 AM EDT
Enrolling by invitation Not applicable Interventional

Ready, Set, Go! A Physical Fitness Intervention for Children With Mobility Challenges

NCT04789746
View directory record Official record
Conditions
Cerebral Palsy, Myelomeningocele, Arthrogryposis, Motor Skills Disorders, Gross Motor Development Delay
Interventions
adapted Chicago Run running program
Behavioral
Lead sponsor
Northwestern University
Other
Eligibility
6 Years to 18 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
1
States / cities
Chicago Heights, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

NCT04526210
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Bupropion Hydrochloride
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Diagnosis of Central Adrenal Insufficiency in Patients With Prader-Willi Syndrome

NCT02368379
View directory record Official record
Conditions
Prader Willi Syndrome, Adrenal Insufficiency
Interventions
Low dose (1 mcg) ACTH stimulation test, Overnight metyrapone test
Other
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
2 Years and older
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 29, 2018 · Synced Oct 11, 2026, 10:02 AM EDT
Completed Not applicable Interventional

Hippotherapy to Improve the Balance of Children With Movement Disorders

NCT01313325
View directory record Official record
Conditions
Neuromuscular Diseases, Cerebral Palsy, Down Syndrome, Autism, Developmental Coordination Disorder
Interventions
Hippotherapy
Other
Lead sponsor
Central Michigan University
Other
Eligibility
5 Years to 17 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Dansville, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 11, 2011 · Synced Oct 11, 2026, 10:02 AM EDT