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ClinicalTrials.gov public records Last synced Oct 24, 2026, 2:29 AM EDT

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Showing 1489–1512 of 1,712 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, Non-Randomized, Open-Label, Single-Dose Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF 01913539] In Subjects With Severely-Impaired And Normal Renal Function

NCT00824590
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
2
States / cities
Miami, Florida • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 30, 2009 · Synced Oct 24, 2026, 2:29 AM EDT
Active, not recruiting Phase 3 Interventional

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

NCT06255782
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
Interventions
ECUR-506
Genetic
Lead sponsor
iECURE, Inc.
Industry
Eligibility
24 Hours to 7 Months · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Not yet recruiting Phase 2 Interventional

Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults

NCT07891286
View directory record Official record
Conditions
LGCMN, Large and Giant Congenital Melanocytic Nevi, NF1, Solid Tumor, Pediatric Tumors, Neurocutaneous Melanocytosis, Congenital Melanocytic Nevus, Arachnoid Cysts, Astrocytoma, Ependymoma, Melanoma
Interventions
Mirdametinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 23, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

NCT07181837
View directory record Official record
Conditions
Angelman Syndrome
Interventions
MVX-220
Genetic
Lead sponsor
MavriX Bio, LLC
Industry
Eligibility
4 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

NCT00964236
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
6 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 4, 2018 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Phase 2 Interventional Results available

Randomized Trial of Hydrocortisone in Very Preterm High-Risk Infants

NCT00167544
View directory record Official record
Conditions
Bronchopulmonary Dysplasia, Encephalomalacia, Premature Birth
Interventions
Hydrocortisone, Placebo
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
1 Week to 3 Weeks
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 30, 2013 · Synced Oct 24, 2026, 2:29 AM EDT
Recruiting No phase listed Observational

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

NCT06805695
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
Interventions
No Intervention
Other
Lead sponsor
iECURE, Inc.
Industry
Eligibility
7 Months to 15 Months
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (agalsidase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 24, 2026, 2:29 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients with HPDL Mutations

NCT05848271
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Phase 4 Interventional Results available

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

NCT01288027
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Alglucosidase Alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
9
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2014 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Phase 3 Interventional Accepts healthy volunteers Results available

Evaluating the Efficacy of Dextromethorphan/Quinidine in Treating Irritability in Huntington's Disease

NCT03854019
View directory record Official record
Conditions
Huntington Disease, Irritability
Interventions
Dextromethorphan/quinidine 20mg/10mg (DM/Q 20mg/10mg), Placebo
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 24, 2026, 2:29 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Comprehensive HHT Outcomes Registry of the United States (CHORUS)

NCT06259292
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia, Arteriovenous Malformations, Telangiectasia, Epistaxis, GastroIntestinal Bleeding, Cerebral Arteriovenous Malformations, Vascular Malformation
Interventions
Not listed
Lead sponsor
Cure HHT
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2033
U.S. locations
16
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Enrolling by invitation Not applicable Interventional

Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS

NCT07225231
View directory record Official record
Conditions
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Interventions
Byteflies Remote patient monitoring solution (with reduced EEG)
Device
Lead sponsor
Byteflies
Industry
Eligibility
3 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Louisville, Kentucky • Austin, Texas • Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Completed No phase listed Observational

Evaluation and Treatment of Patients Spinal Blood Vessel Abnormalities

NCT00001364
View directory record Official record
Conditions
Arteriovenous Fistula, Arteriovenous Malformation, Spinal Cord Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
4 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Not applicable Interventional Results available

Median Nerve Stimulation Pilot

NCT04731714
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders, Tic Disorder, Chronic Motor or Vocal
Interventions
Rhythmic median nerve stimulation, Arrhythmic median nerve stimulation
Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 64 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 5, 2023 · Synced Oct 24, 2026, 2:29 AM EDT
Recruiting Not applicable Interventional

Cardiac Output and Fatigue in Friedreich's Ataxia

NCT07444333
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Aerobic Exercise
Behavioral
Lead sponsor
Scott Barbuto
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT05127226
View directory record Official record
Conditions
Angelman Syndrome
Interventions
ION582
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
San Diego, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 24, 2026, 2:29 AM EDT
Completed No phase listed Observational

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

NCT00778206
View directory record Official record
Conditions
Phenylketonuria, Hyperphenylalaninaemia
Interventions
Kuvan
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
1,887 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
45
States / cities
Los Angeles, California • Orange, California • San Diego, California + 38 more
Source: ClinicalTrials.gov public record
Updated Oct 12, 2022 · Synced Oct 24, 2026, 2:29 AM EDT
Terminated Phase 1 Interventional

Hepatocyte Transplantation for Liver Based Metabolic Disorders

NCT01345578
View directory record Official record
Conditions
Metabolic Diseases
Interventions
human hepatocyte transplantation, Preparative Radiation Therapy
Drug · Radiation
Lead sponsor
Ira Fox
Other
Eligibility
Up to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Not applicable Interventional Results available

Transcranial Magnetic Stimulation for Facial Pain

NCT02633306
View directory record Official record
Conditions
Trigeminal Nerve Injuries, Post-herpetic Neuralgia, Facial Pain, Nervus Intermedius Neuralgia
Interventions
transcranial magnetic stimulation
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 110 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 8, 2021 · Synced Oct 24, 2026, 2:29 AM EDT
Withdrawn Not applicable Interventional

Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery

NCT04344626
View directory record Official record
Conditions
Epilepsy, Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly, Polymicrogyria, Rasmussen Encephalitis, Sturge-Weber Syndrome, Gliosis, Stroke, Tumor, Brain
Interventions
Intra-operative brain tonometry
Device
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 10, 2023 · Synced Oct 24, 2026, 2:29 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539
View directory record Official record
Conditions
Fabry Disease
Interventions
Lucerastat, Placebo
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2024 · Synced Oct 24, 2026, 2:29 AM EDT
Terminated No phase listed Observational

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

NCT04909346
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 12, 2023 · Synced Oct 24, 2026, 2:29 AM EDT
Not yet recruiting Phase 4 Interventional

A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS

NCT07728097
View directory record Official record
Conditions
Lennox-Gastaut Syndrome
Interventions
Cannabidiol Oral Solution
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Fresno, California • New Orleans, Louisiana • Raleigh, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 22, 2026 · Synced Oct 24, 2026, 2:29 AM EDT