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ClinicalTrials.gov public records Last synced Oct 14, 2026, 9:56 PM EDT

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Showing 481–504 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Not listed Phase 3 Interventional

Vitamin E in Aging Persons With Down Syndrome

NCT00056329
View directory record Official record
Conditions
Down Syndrome, Alzheimer Disease
Interventions
Vitamin E, multivitamin, Placebo
Drug
Lead sponsor
New York State Institute for Basic Research
Government
Eligibility
50 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2012
U.S. locations
14
States / cities
Farmington, Connecticut • Tampa, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated May 4, 2012 · Synced Oct 14, 2026, 9:56 PM EDT
Completed No phase listed Observational

Natural History and Functional Status Study of Patients With Lafora Disease

NCT03876522
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 14, 2026, 9:56 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 14, 2026, 9:56 PM EDT
Completed Not applicable Interventional

Music in Reducing Anxiety and Pain in Adult Patients Undergoing Bone Marrow Biopsy for Hematologic Cancers or Other Diseases

NCT00376922
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Psychosocial Effects of Cancer and Its Treatment
Interventions
music therapy
Procedure
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 120 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 14, 2026, 9:56 PM EDT
Terminated Phase 3 Interventional Results available

Open-Label Extension of EryDex Study IEDAT-04-2022

NCT06664853
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Dexamethasone sodium phosphate
Drug
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
5
States / cities
Los Angeles, California • Baltimore, Maryland • New Brunswick, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 14, 2026, 9:56 PM EDT
Completed Phase 1 Interventional

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

NCT02595489
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
vitamin D3
Dietary Supplement
Lead sponsor
Stanford University
Other
Eligibility
18 Months to 25 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Oct 14, 2026, 9:56 PM EDT
Active, not recruiting Phase 2 Interventional

Trial of Atezolizumab, Bevacizumab, and Tiragolumab in Patients With Microsatellite Stable, Metastatic Colorectal Cancer

NCT06784947
View directory record Official record
Conditions
Microsatellite Stable (MSS) Colorectal Cancer (CRC), Metastatic Colorectal Cancer (CRC), Colorectal Cancer Stage IV
Interventions
Pre-Treatment Biopsy, On-Treatment Biopsy, Tiragolumab, Atezolizumab, Bevacizumab
Procedure · Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Oct 2, 2025 · Synced Oct 14, 2026, 9:56 PM EDT
Completed Phase 4 Interventional Results available

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

NCT01257737
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
15
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 22, 2024 · Synced Oct 14, 2026, 9:56 PM EDT
Completed No phase listed Observational

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

NCT04469894
View directory record Official record
Conditions
Niemann-Pick Diseases
Interventions
Not listed
Lead sponsor
National Niemann-Pick Disease Foundation
Other
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 6, 2020 · Synced Oct 14, 2026, 9:56 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase, Diet Only
Drug · Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
12 Years to 17 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 14, 2026, 9:56 PM EDT
Not listed Not applicable Interventional

Evaluate the Efficacy, Safety and Tolerability of TSUPPORT for Adults With Tourette Syndrome

NCT05183594
View directory record Official record
Conditions
Tourette Syndrome
Interventions
TSupport (a Traditional Chinese Medicine)
Dietary Supplement
Lead sponsor
Tasly Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 11, 2022 · Synced Oct 14, 2026, 9:56 PM EDT
Completed No phase listed Observational

Transitional Life Events in Patients With Friedreich's Ataxia: Implications for Genetic Counseling

NCT00056186
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 14, 2026, 9:56 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

NCT02292654
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2022 · Synced Oct 14, 2026, 9:56 PM EDT
Completed Not applicable Interventional

The Effect of Supplemental Docosahexaenoic Acid (DHA) on Neurocognitive Outcomes in Teen and Adult Women With Phenylketonuria(PKU)

NCT00892554
View directory record Official record
Conditions
Phenylketonuria
Interventions
Docosahexaenoic Acid, Corn/soy oil
Dietary Supplement
Lead sponsor
Emory University
Other
Eligibility
12 Years to 50 Years · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 11, 2015 · Synced Oct 14, 2026, 9:56 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

NCT02536911
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 10, 2017 · Synced Oct 14, 2026, 9:56 PM EDT
Completed No phase listed Observational

Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers

NCT06667414
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 60 Years
Enrollment
574 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
13
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 14, 2026, 9:56 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria

NCT02297347
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 5, 2018 · Synced Oct 14, 2026, 9:56 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 14, 2026, 9:56 PM EDT
Completed Phase 1 Interventional Results available

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952
View directory record Official record
Conditions
Spinal Muscular Atrophy 1
Interventions
AVXS-101
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 15, 2022 · Synced Oct 14, 2026, 9:56 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

NCT05854433
View directory record Official record
Conditions
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Interventions
Non-interventional study
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
30 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 14, 2026, 9:56 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of Data Collected From Individuals Administered Neurobehavioral Assessments

NCT01445314
View directory record Official record
Conditions
HIV, Brain Tumors, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
598 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 16, 2024 · Synced Oct 14, 2026, 9:56 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 14, 2026, 9:56 PM EDT
Completed Phase 2 Interventional Results available

A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod

NCT02215616
View directory record Official record
Conditions
Huntington's Disease
Interventions
Laquinimod, Placebo
Drug
Lead sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Industry
Eligibility
21 Years to 55 Years
Enrollment
352 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
16
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 13 more
Source: ClinicalTrials.gov public record
Updated May 4, 2020 · Synced Oct 14, 2026, 9:56 PM EDT
Recruiting No phase listed Observational

Evaluation of Outcome Metrics in Alexander Disease

NCT02714764
View directory record Official record
Conditions
Alexander Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 14, 2026, 9:56 PM EDT