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ClinicalTrials.gov public records Last synced Oct 15, 2026, 2:37 PM EDT

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Showing 625–648 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Withdrawn No phase listed Observational

Evaluation of Phenotypic Variability in Fabry Disease

NCT03145779
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 3, 2020 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 2 Interventional Results available

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

NCT02996305
View directory record Official record
Conditions
Angelman Syndrome
Interventions
OV101 Regimen 1, OV101 regimen 2, Placebo
Drug · Other
Lead sponsor
Healx AI
Industry
Eligibility
13 Years to 49 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
12
States / cities
Phoenix, Arizona • San Diego, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2025 · Synced Oct 15, 2026, 2:37 PM EDT
Active, not recruiting Phase 2 Interventional

Safety and Modulation of ABCC9 Pathways by Nicorandil for the Treatment of Hippocampal Sclerosis of Aging

NCT04120766
View directory record Official record
Conditions
Dementia, Mild Cognitive Impairment
Interventions
Nicorandil, Placebo
Drug
Lead sponsor
Gregory Jicha, MD, PhD
Other
Eligibility
70 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 1 Interventional

Tabectedin to Treat Children and Adolescents With Cancer

NCT01453283
View directory record Official record
Conditions
Solid Tumors
Interventions
Trabectedin (ecteinascidin-743, ET-743, YONDELIS [R])
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Years to 16 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 15, 2026, 2:37 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Mechanisms of Cell Death in Spinal Muscular Atrophy

NCT01754441
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2020
U.S. locations
2
States / cities
Wilmington, Delaware • Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 21, 2020 · Synced Oct 15, 2026, 2:37 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)

NCT06719141
View directory record Official record
Conditions
Developmental and Epileptic Encephalopathy
Interventions
LP352, Placebo
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
31
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 23 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Exploration of Immunity in Gaucher Disease

NCT01358188
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 15, 2026, 2:37 PM 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 15, 2026, 2:37 PM EDT
Completed No phase listed Observational

Goal Attainment Scaling (GAS) Sub-Study

NCT06484426
View directory record Official record
Conditions
Alzheimer's Disease in Down Syndrome
Interventions
Not listed
Lead sponsor
LuMind IDSC Foundation
Other
Eligibility
35 Years and older
Enrollment
46 participants
Timeline
2021 – 2024
U.S. locations
5
States / cities
San Diego, California • Park Ridge, Illinois • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 15, 2026, 2:37 PM EDT
Recruiting No phase listed Observational

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

NCT06190626
View directory record Official record
Conditions
Zellweger Spectrum Disorder
Interventions
Not listed
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Withdrawn Phase 2 Interventional

Adjunctive Everolimus (RAD 001) Therapy for Epilepsy in Children With Sturge-Weber Syndrome (SWS)

NCT01997255
View directory record Official record
Conditions
Sturge Weber Syndrome
Interventions
Everolimus
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
2 Years to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 5, 2016 · Synced Oct 15, 2026, 2:37 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

NCT03210714
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor
Interventions
Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Erdafitinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
114
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 15, 2026, 2:37 PM 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 15, 2026, 2:37 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599
View directory record Official record
Conditions
Gaucher Disease, Gaucher Disease, Type 1
Interventions
LY3884961
Genetic
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
2
States / cities
Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Recruiting No phase listed Observational

Rapid Evacuation and Access of Cerebral Hemorrhage Registry

NCT07651631
View directory record Official record
Conditions
Intracerebral Hemorrhage
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Not applicable Interventional Results available

Minocycline in the Treatment of Angelman Syndrome

NCT01531582
View directory record Official record
Conditions
Angelman Syndrome
Interventions
minocycline
Drug
Lead sponsor
University of South Florida
Other
Eligibility
4 Years to 12 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Apr 18, 2023 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 3 Interventional Results available

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727
View directory record Official record
Conditions
Phenylketonuria
Interventions
BMN 165
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
261 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
31
States / cities
La Jolla, California • Oakland, California • Aurora, Colorado + 28 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 15, 2026, 2:37 PM EDT
Terminated No phase listed Observational

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

NCT01541722
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
Mark Batshaw
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
10
States / cities
Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2015 · Synced Oct 15, 2026, 2:37 PM EDT
Terminated Phase 3 Interventional

Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)

NCT00712426
View directory record Official record
Conditions
Huntington's Disease
Interventions
Creatine Monohydrate, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
553 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
38
States / cities
Birmingham, Alabama • Irvine, California • Sacramento, California + 35 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2016 · Synced Oct 15, 2026, 2:37 PM EDT
Recruiting No phase listed Observational

Abbott Vascular Medical Device Registry

NCT04573660
View directory record Official record
Conditions
Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism, Arterial Embolism
Interventions
Coronary and peripheral stents, Pacing catheters, Vascular plugs, Measurement and imaging (FFR and OCT), Peripheral dilatation catheters, Coronary dilatation catheters, Coronary and peripheral guidewires, Vessel closure/compression devices, Vascular access introducers
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years and older
Enrollment
3,784 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
7
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 2 Interventional Results available

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865
View directory record Official record
Conditions
Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
Interventions
afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 15, 2018 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 1 Interventional

A Pilot Study Of SD-809 (Deutetrabenazine) In Moderate To Severe Tourette Syndrome (TS)

NCT02674321
View directory record Official record
Conditions
TOURETTE SYNDROME
Interventions
SD-809
Drug
Lead sponsor
Auspex Pharmaceuticals, Inc.
Industry
Eligibility
12 Years to 18 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2021 · Synced Oct 15, 2026, 2:37 PM EDT