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ClinicalTrials.gov public records Last synced Oct 20, 2026, 8:04 PM EDT

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Showing 1321–1344 of 1,710 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated Phase 3 Interventional Results available

Tanezumab In Osteoarthritis Of The Hip Or Knee

NCT00985621
View directory record Official record
Conditions
Osteoarthritis
Interventions
tanezumab 10 mg, tanezumab 5 mg, oxycodone, placebo
Biological · Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
614 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
66
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 54 more
Source: ClinicalTrials.gov public record
Updated May 14, 2021 · Synced Oct 20, 2026, 8:04 PM EDT
Recruiting No phase listed Observational

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

NCT05843552
View directory record Official record
Conditions
Gaucher Disease
Interventions
no intervention
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 20, 2026, 8:04 PM EDT
Terminated Phase 2 Interventional Results available

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 18, 2019 · Synced Oct 20, 2026, 8:04 PM EDT
No Longer Available No phase listed Expanded access

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

NCT03639844
View directory record Official record
Conditions
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
Interventions
rivogenlecleucel, rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
3 Months to 21 Years
U.S. locations
2
States / cities
Los Angeles, California • Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 5, 2020 · Synced Oct 20, 2026, 8:04 PM EDT
Recruiting No phase listed Observational

A Natural History Study of the Gangliosidoses

NCT00668187
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
Not listed
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Oct 20, 2026, 8:04 PM EDT
Withdrawn No phase listed Observational

B7 Coreceptor Molecules in Hyper IgD Syndrome Form of Mevalonate Kinase Deficiency

NCT01568736
View directory record Official record
Conditions
Hyper IgD Syndrome, Mevalonate Kinase Deficiency
Interventions
Not listed
Lead sponsor
Michigan Technological University
Other
Eligibility
18 Years to 89 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Houghton, Michigan • Omaha, Nebraska • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 18, 2018 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Remote Neurocognitive and Psychological Assessment in PKU

NCT04943393
View directory record Official record
Conditions
Phenylketonurias
Interventions
nointervention
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 40 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 22, 2022 · Synced Oct 20, 2026, 8:04 PM EDT
Completed Phase 1 Interventional

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

NCT01005004
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
HuCNS-SC cells
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 5 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 20, 2026, 8:04 PM EDT
Terminated Phase 1 Interventional Results available

Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer

NCT03478904
View directory record Official record
Conditions
Prostate Cancer
Interventions
Enzalutamide Liquid, Enzalutamide Capsule
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 10, 2021 · Synced Oct 20, 2026, 8:04 PM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational

Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia

NCT01158807
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia
Interventions
Not listed
Lead sponsor
Unity Health Toronto
Other
Eligibility
Not listed
Enrollment
2,272 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2025
U.S. locations
14
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 20, 2026, 8:04 PM EDT
Completed Phase 2 Interventional

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

NCT00763932
View directory record Official record
Conditions
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Oakland, California • Orange, California • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

I-123 Brain Studies of Serotonin Metabolism in Psychiatric Patients and Normal Volunteers

NCT00001771
View directory record Official record
Conditions
Healthy, Mental Disorder, Obsessive Compulsive Disorder, Schizophrenia, Tourette Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
112 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 8:04 PM EDT
Terminated Phase 3 Interventional Results available

Open-label Extension Study of Pramipexole in the Treatment of Children and Adolescents With Tourette Syndrome

NCT00681863
View directory record Official record
Conditions
Tourette Syndrome
Interventions
pramipexole 0.125 mg BID, pramipexole 0.0625 mg QD, pramipexole 0.125 mg TID, pramipexole 0.25 mg BID, pramipexole 0.0625 mg BID
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
6 Years to 17 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
13
States / cities
Bradenton, Florida • Tampa, Florida • Columbus, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated May 23, 2014 · Synced Oct 20, 2026, 8:04 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 4, 2025 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
View directory record Official record
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 8:04 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

NCT02991144
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 20, 2026, 8:04 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas

NCT02831257
View directory record Official record
Conditions
Neurofibromatosis 2, Meningioma
Interventions
AZD2014
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 13, 2022 · Synced Oct 20, 2026, 8:04 PM EDT
Recruiting Phase 1 Interventional

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

NCT05302271
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Biological · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 20, 2026, 8:04 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

A Pilot Study on Diurnal Variation

NCT01806051
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Kuvan
Drug
Lead sponsor
Dr. Linda Randolph
Other
Eligibility
4 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 20, 2016 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational

Biomarkers for Detection of Brain Ischemia

NCT00490295
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
1 Day to 30 Days
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 27, 2013 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

NCT01701154
View directory record Official record
Conditions
Pompe Disease
Interventions
respiratory muscle strength training
Other
Lead sponsor
Duke University
Other
Eligibility
3 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 30, 2023 · Synced Oct 20, 2026, 8:04 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Complex Eye Movements in Parkinson's Disease and Related Movement Disorders

NCT04925622
View directory record Official record
Conditions
Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, Corticobasal Degeneration, Parkinsonian Disorders, Essential Tremor, Vascular Parkinsonism, Multiple System Atrophy, Parkinson Variant, Parkinsonian Syndrome, Huntington Disease
Interventions
Complex eye movement exam
Diagnostic Test
Lead sponsor
Saccadous, Inc.
Other
Eligibility
30 Years and older
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated May 4, 2022 · Synced Oct 20, 2026, 8:04 PM EDT
Not yet recruiting Phase 2 Interventional

Neoadjuvant Pembrolizumab and Porustobart in Resectable pMMR/MSS Colon Cancer

NCT07808151
View directory record Official record
Conditions
Colon Cancer, Microsatellite Stable (MSS) Colon Cancer, Proficient Mismatch Repair (pMMR) Colon Cancer
Interventions
Pembrolizumab, Porustobart
Drug
Lead sponsor
Solstice Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
11
States / cities
Los Angeles, California • Tampa, Florida • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 20, 2026, 8:04 PM EDT