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ClinicalTrials.gov public records Last synced Oct 14, 2026, 8:35 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 433–456 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Not listed Phase 1 Interventional

AN0025 and Pembrolizumab Combination in Advanced Solid Tumors

NCT04432857
View directory record Official record
Conditions
Triple-negative Breast Cancer, NSCLC, Squamous or Non-Squamous, Urothelial Carcinoma of the Bladder, Microsatellite Stable (MSS) Colorectal Cancer (CRC), Cervical Cancer
Interventions
AN0025, Pembrolizumab
Drug
Lead sponsor
Adlai Nortye Biopharma Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
4
States / cities
St Louis, Missouri • Houston, Texas • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Oct 14, 2026, 8:35 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

NCT04849741
View directory record Official record
Conditions
Alexander Disease
Interventions
zilganersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
4
States / cities
Palo Alto, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 14, 2026, 8:35 PM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers

High-Dose Erythropoietin in Extremely Premature Infants to Prevent/Attenuate Brain Injury: A Phase II Study

NCT00589953
View directory record Official record
Conditions
Infant, Premature, Erythropoietin, Brain Injury, Intraventricular Hemorrhage, Periventricular Leukomalacia, Neurodevelopmental Outcomes, Randomized Clinical Trial
Interventions
Erythropoietin, Saline placebo
Drug
Lead sponsor
Atlantic Health System
Other
Eligibility
Up to 24 Hours
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
1
States / cities
Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Jul 30, 2013 · Synced Oct 14, 2026, 8:35 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NCT00439569
View directory record Official record
Conditions
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
5
States / cities
Stanford, California • St Louis, Missouri • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2010 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 4 Interventional Results available

Developing New Treatments for Tourette Syndrome: Therapeutic Trials With Modulators of Glutamatergic Neurotransmission

NCT01018056
View directory record Official record
Conditions
Tourette Syndrome
Interventions
D-serine, Riluzole, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
8 Years to 17 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Blood Flow Regulation in Individuals With Down Syndrome - Training Study

NCT04854122
View directory record Official record
Conditions
Down Syndrome
Interventions
Combined exercise intervention, Usual care / Sham intervention
Behavioral
Lead sponsor
University of Nevada, Las Vegas
Other
Eligibility
18 Years to 35 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 2 Interventional

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

NCT05630066
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Alogabat
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
5 Years to 17 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 14, 2026, 8:35 PM 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 14, 2026, 8:35 PM EDT
Not listed Phase 3 Interventional

AT-007 in Adult Subjects With Classic Galactosemia (CG)

NCT05418829
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007
Drug
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Berlin, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 14, 2022 · Synced Oct 14, 2026, 8:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scan of Brain Metabolism in Relation to Age and Disease

NCT00001972
View directory record Official record
Conditions
Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
Interventions
15 O Water
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
123 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Not applicable Interventional

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

NCT03893240
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions
Neutralizing Antibody to SPK-3006 capsid
Diagnostic Test
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
6
States / cities
Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2023 · Synced Oct 14, 2026, 8:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism

NCT01358474
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 85 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2018
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 18, 2019 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 3 Interventional Results available

Clobazam in Patients With Lennox-Gastaut Syndrome

NCT00518713
View directory record Official record
Conditions
Epilepsy, Epilepsy, Generalized, Seizures
Interventions
Clobazam Low Dose, Clobazam Medium Dose, Clobazam High Dose, Placebo
Drug
Lead sponsor
Lundbeck LLC
Industry
Eligibility
2 Years to 60 Years
Enrollment
238 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
34
States / cities
Huntsville, Alabama • Phoenix, Arizona • Los Angeles, California + 26 more
Source: ClinicalTrials.gov public record
Updated Feb 9, 2012 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

NCT06188325
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 4 Interventional Results available

Growth and Development Study of Alglucosidase Alfa

NCT00486889
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 26, 2022 · Synced Oct 14, 2026, 8:35 PM EDT
Terminated No phase listed Observational

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

NCT05008874
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 14, 2026, 8:35 PM EDT
Completed No phase listed Observational

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

NCT02998710
View directory record Official record
Conditions
Homocystinuria Due to CBS Deficiency
Interventions
Not listed
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
1 Year to 65 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 14, 2026, 8:35 PM EDT
Recruiting Not applicable Interventional

Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders

NCT05726591
View directory record Official record
Conditions
Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Incomplete Spinal Cord Injury
Interventions
EA-KAFO
Device
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
3 Years to 17 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2025 · Synced Oct 14, 2026, 8:35 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Quantitative Assessment of Sucking for Early Diagnosis of Brain Injury in Infants at High Risk

NCT03246243
View directory record Official record
Conditions
Hypoxic-Ischemic Encephalopathy, Hypoglycemia, Neonatal Abstinence Syndrome
Interventions
nfant feeding solution
Device
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 6 Months
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 21, 2021 · Synced Oct 14, 2026, 8:35 PM EDT
Completed No phase listed Observational

Regulation of Cerebral Blood Flow in Very Low Birth Weight Infants

NCT00579943
View directory record Official record
Conditions
Infant, Very Low Birth Weight, Infant, Premature, Diseases, Cerebral Hemorrhage
Interventions
Not listed
Lead sponsor
University of Arkansas
Other
Eligibility
1 Hour to 7 Days
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Jun 4, 2008 · Synced Oct 14, 2026, 8:35 PM EDT
Completed No phase listed Observational

Mitochondrial Video Assessment Source Material Study

NCT06180421
View directory record Official record
Conditions
PolG, Primary Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
16 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 1 Interventional

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

NCT01898364
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
Interventions
GZ402666
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
7
States / cities
Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2023 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Phase 4 Interventional

Study of Zinc for Wilson Disease

NCT00004338
View directory record Official record
Conditions
Wilson Disease
Interventions
zinc acetate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1993
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 8:35 PM EDT
Completed Not applicable Interventional Results available

Molecular-Guided Therapy for Childhood Cancer

NCT02162732
View directory record Official record
Conditions
Neuroblastoma, Medulloblastoma, Glioma, Ependymoma, Choroid Plexus Neoplasms, Craniopharyngioma, Dysembryoplastic Neuroepithelial Tumor, Meningioma, Primitive Neuroectodermal Tumors (PNETs), Germ Cell Tumors, Rhabdomyosarcoma, Non-rhabdomyosarcoma, Ewings Sarcoma, Osteosarcoma, Wilms Tumor, Renal Cell Carcinoma, Malignant Rhabdoid Tumor, Clear Cell Sarcoma, Liver Tumors
Interventions
Guided Therapy
Device
Lead sponsor
Giselle Sholler
Other
Eligibility
13 Months to 21 Years
Enrollment
186 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
18
States / cities
Little Rock, Arkansas • San Diego, California • Hartford, Connecticut + 15 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Oct 14, 2026, 8:35 PM EDT