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ClinicalTrials.gov public records Last synced Oct 16, 2026, 3:52 PM EDT

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Showing 865–888 of 1,710 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 2 Interventional Results available

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

NCT01599286
View directory record Official record
Conditions
Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
Interventions
Carbaglu, Placebo
Drug
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Week to 99 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
9
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Oct 16, 2026, 3:52 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

NCT03652259
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy, Type 2E
Interventions
SRP-9003
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 15 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Oct 16, 2026, 3:52 PM EDT
Completed No phase listed Observational

VCRC Patient Contact Registry Patient-Reported Data Validation Study

NCT02190942
View directory record Official record
Conditions
Behcet's Disease, Churg-Strauss Syndrome, Giant Cell Arteritis, Wegener Granulomatosis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
198 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced Oct 16, 2026, 3:52 PM EDT
Recruiting No phase listed Observational

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

NCT06663358
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase-alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 16, 2026, 3:52 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Central Mechanisms in Speech Motor Control Studied With H215O PET

NCT00001308
View directory record Official record
Conditions
Communication Disorder, Healthy, Stuttering, Tourette Syndrome, Voice Disorder
Interventions
Not listed
Lead sponsor
National Institute on Deafness and Other Communication Disorders (NIDCD)
NIH
Eligibility
18 Years to 85 Years
Enrollment
1,163 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1992 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 1 Interventional

Temozolomide and O6-benzylguanine in Treating Children With Solid Tumors

NCT00020150
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
O6-benzylguanine, temozolomide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 29, 2015 · Synced Oct 16, 2026, 3:52 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 16, 2026, 3:52 PM EDT
Recruiting Phase 2 Interventional

A Study of MZE782 in Adults With PKU

NCT07694440
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
MZE782, Placebo
Drug · Other
Lead sponsor
Maze Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
7
States / cities
Oakland, California • Palo Alto, California • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 16, 2026, 3:52 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Timeline
2018 – 2029
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Oct 16, 2026, 3:52 PM EDT
Completed No phase listed Observational

The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study

NCT00071903
View directory record Official record
Conditions
Pseudotumor Cerebri, Cystinosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 50 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 2 Interventional Results available

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

NCT01125046
View directory record Official record
Conditions
Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions
bevacizumab
Biological
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
5
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2021 · Synced Oct 16, 2026, 3:52 PM EDT
Completed No phase listed Observational

Beacon Tip Sizing Catheter and Slip-Cath Beacon Tip Catheter Study

NCT06986954
View directory record Official record
Conditions
Stenosis, Bleed Intracranial, Stroke, TIA, Vascular Malformation, Pseudoaneurysm
Interventions
Beacon Tip Catheters
Device
Lead sponsor
Cook Research Incorporated
Industry
Eligibility
Not listed
Enrollment
816 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Darien, Connecticut
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 16, 2026, 3:52 PM EDT
Completed No phase listed Observational

Longitudinal Studies of the Glycoproteinoses

NCT01891422
View directory record Official record
Conditions
Aspartylglucosaminuria, Fucosidosis, Galactosialidosis, Alpha Mannosidosis, Beta Mannosidosis, Mucolipidosis II, Mucolipidosis III, Schindler Disease, Sialidosis
Interventions
Non-interventional.
Other
Lead sponsor
Greenwood Genetic Center
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
North Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 16, 2026, 3:52 PM EDT
Completed No phase listed Observational Results available

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

NCT02354651
View directory record Official record
Conditions
Pompe Disease
Interventions
Diaphragm conditioning via phrenic nerve stimulation
Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 16, 2026, 3:52 PM EDT
Recruiting No phase listed Observational

A Multicenter Pediatric Deep Brain Stimulation Registry

NCT06585618
View directory record Official record
Conditions
Dystonia, Epilepsy in Children, Cerebral Palsy, Tourette Syndrome, Obsessive-Compulsive Disorder, Neurologic Disorder, Movement Disorders in Children, Movement Disorders, Deep Brain Stimulation
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
0 Years to 18 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 16, 2026, 3:52 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

MRI Study of Tic Remission in Tourette Syndrome

NCT01405859
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 35 Years · Male only
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 12, 2016 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 3 Interventional Results available

Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease

NCT04102579
View directory record Official record
Conditions
Chorea, Huntington
Interventions
Valbenazine, Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
42
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2023 · Synced Oct 16, 2026, 3:52 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Drug Discovery for Parkinson's With Mutations in the GBA Gene

NCT05536388
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease, Healthy, GBA Gene Mutation
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

NCT03500627
View directory record Official record
Conditions
Healthy
Interventions
OP-101
Drug
Lead sponsor
Orpheris, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 17, 2018 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

NCT02797080
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
interferon γ-1b
Drug
Lead sponsor
Amgen
Industry
Eligibility
11 Years to 27 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 4 Interventional Results available

A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

NCT04718779
View directory record Official record
Conditions
Gaucher Disease
Interventions
Digital Engagement Application (GD App), No Intervention
Other
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 21, 2023 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 2 Interventional Results available

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
14
States / cities
Aurora, Colorado • Gainesville, Florida • Chicago, Illinois + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 16, 2026, 3:52 PM EDT
Not listed Not applicable Interventional

Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)

NCT05174559
View directory record Official record
Conditions
Phenylketonurias
Interventions
PreKUnil® LNAA Medical Food for PKU
Other
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 4, 2023 · Synced Oct 16, 2026, 3:52 PM EDT
Completed Phase 3 Interventional Results available

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

NCT01218659
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, agalsidase
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2018 · Synced Oct 16, 2026, 3:52 PM EDT