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ClinicalTrials.gov public records Last synced Oct 14, 2026, 2:54 AM EDT

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Showing 217–240 of 1,708 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 4 Interventional Results available

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

NCT01350440
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
IVIG
Biological
Lead sponsor
University of South Florida
Other
Eligibility
10 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jul 23, 2013 · Synced Oct 14, 2026, 2:54 AM EDT
Completed No phase listed Observational

International Ataxia Rating Scale in Younger Patients

NCT01942850
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Not listed
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years to 18 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Dec 31, 2014 · Synced Oct 14, 2026, 2:54 AM EDT
Terminated No phase listed Observational

Natural History Clinical Study in Adult PKU

NCT04768348
View directory record Official record
Conditions
Phenylketonurias, PKU
Interventions
Not listed
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
8
States / cities
Los Angeles, California • Orange, California • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2023 · Synced Oct 14, 2026, 2:54 AM EDT
Not yet recruiting No phase listed Observational Accepts healthy volunteers

Multi-Modal Digital Monitoring of Disease Symptoms Huntington's Disease

NCT07315984
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
BioSensics
Industry
Eligibility
25 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2027 – 2028
U.S. locations
2
States / cities
Rochester, New York • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 14, 2026, 2:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

University of Central Florida CereBank

NCT06876857
View directory record Official record
Conditions
Biospecimen Donation, Brain Injury, Brain Diseases
Interventions
Not listed
Lead sponsor
University of Central Florida
Other
Eligibility
18 Years and older · Female only
Enrollment
50,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2075
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 14, 2026, 2:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Timing of Voluntary Movement in Patients With Tourette Syndrome and Chronic Tic Disorder

NCT00081419
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 65 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 14, 2026, 2:54 AM EDT
Not listed No phase listed Observational

Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)

NCT06194539
View directory record Official record
Conditions
Fertility Issues
Interventions
Not listed
Lead sponsor
Bar-Chama, Natan, M.D.
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
75 participants
Timeline
2024 – 2026
U.S. locations
1
States / cities
Cresskill, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 8, 2024 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Not applicable Interventional

Study for Treatment of Cancer in Children With Ataxia-telangiectasia

NCT00187057
View directory record Official record
Conditions
Ataxia-Telangiectasia
Interventions
vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine, dexamethasone, procarbazine, chemotherapy, intrathecal chemotherapy, steroid therapy
Drug · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 10 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2013
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 27, 2015 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Not applicable Interventional

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

NCT02801539
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
RMT therapy using modified RMT device, Sham-RMT therapy using modified RMT device
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 30, 2019 · Synced Oct 14, 2026, 2:54 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

NCT02867592
View directory record Official record
Conditions
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue, Clear Cell Sarcoma of Soft Tissue, Ewing Sarcoma, Hepatoblastoma, Hepatocellular Carcinoma, Osteosarcoma, Recurrent Adrenal Cortical Carcinoma, Recurrent Alveolar Soft Part Sarcoma, Recurrent Clear Cell Sarcoma of Soft Tissue, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Kidney Wilms Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Osteosarcoma, Recurrent Primary Malignant Central Nervous System Neoplasm, Recurrent Renal Cell Carcinoma, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Medullary Carcinoma, Refractory Adrenal Cortical Carcinoma, Refractory Alveolar Soft Part Sarcoma, Refractory Clear Cell Sarcoma of Soft Tissue, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Primary Malignant Central Nervous System Neoplasm, Refractory Renal Cell Carcinoma, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Medullary Carcinoma, Refractory Wilms Tumor, Renal Cell Carcinoma, Rhabdomyosarcoma, Soft Tissue Sarcoma, Solid Neoplasm, Thyroid Gland Medullary Carcinoma, Wilms Tumor
Interventions
Cabozantinib, Cabozantinib S-malate, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 30 Years
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
142
States / cities
Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 104 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 1 Interventional

Neonatal Erythropoietin in Asphyxiated Term Newborns

NCT00719407
View directory record Official record
Conditions
Hypoxic-ischemic Encephalopathy
Interventions
erythropoietin
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 24 Hours
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 9, 2012 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine

NCT01192828
View directory record Official record
Conditions
Homocystinuria
Interventions
taurine
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
8 Years to 49 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
4
States / cities
Aurora, Colorado • Durham, North Carolina • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 9, 2018 · Synced Oct 14, 2026, 2:54 AM EDT
Terminated Phase 3 Interventional Results available

Coenzyme Q10 in Huntington's Disease (HD)

NCT00608881
View directory record Official record
Conditions
Huntington's Disease
Interventions
coenzyme Q10, placebo
Drug · Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
16 Years and older
Enrollment
609 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
41
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Fayetteville, Arkansas + 37 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2016 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

NCT03826628
View directory record Official record
Conditions
Facial Angiofibroma, Tuberous Sclerosis
Interventions
rapamycin, placebo
Drug
Lead sponsor
Dermatology Specialties Limited Partnership
Industry
Eligibility
6 Years to 65 Years
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
6
States / cities
Phoenix, Arizona • La Jolla, California • St. Petersburg, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2023 · Synced Oct 14, 2026, 2:54 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

NCT04833907
View directory record Official record
Conditions
Canavan Disease
Interventions
rAAV-Olig001-ASPA, Levetiracetam, Prednisone
Drug
Lead sponsor
Myrtelle Inc.
Industry
Eligibility
3 Months to 60 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Sexual Dysfunction in Hypogonadal Men With Epilepsy With Testosterone and Either Anastrozole or Placebo

NCT00179517
View directory record Official record
Conditions
Seizure Disorder, Hypogonadism, Erectile Dysfunction
Interventions
Anastrozole 1mg, Placebo Oral Tablet
Drug
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Alternatives for Reducing Tics in Tourette Syndrome (TS): A Study of TEV-50717 (Deutetrabenazine) for the Treatment of Tourette Syndrome in Children and Adolescents

NCT03452943
View directory record Official record
Conditions
Tourette Syndrome
Interventions
TEV-50717, Placebo
Drug
Lead sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Industry
Eligibility
6 Years to 16 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
27
States / cities
Dothan, Alabama • Sun City, Arizona • Rogers, Arkansas + 22 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2021 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 1 Interventional

BCX-1777 in Treating Patients With Refractory Cancer

NCT00073944
View directory record Official record
Conditions
Cancer
Interventions
forodesine hydrochloride
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated May 30, 2013 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 2 Interventional

Premanifest Huntington's Disease Extension Study II: Creatine Safety & Tolerability

NCT01411163
View directory record Official record
Conditions
Huntington's Disease
Interventions
Creatine monohydrate
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
26 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 10, 2014 · Synced Oct 14, 2026, 2:54 AM EDT
Completed No phase listed Observational

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

NCT05642221
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 110 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

NCT03678324
View directory record Official record
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Oct 14, 2026, 2:54 AM EDT
Completed No phase listed Observational

The Natural History and Pathogenesis of Mucolipidosis Type IV

NCT00015782
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 14, 2026, 2:54 AM EDT
Completed Phase 1Phase 2 Interventional

Safety Study of the Novel Drug Dimebon to Treat Patients With Huntington's Disease

NCT00387270
View directory record Official record
Conditions
Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Medivation, Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 4, 2008 · Synced Oct 14, 2026, 2:54 AM EDT