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ClinicalTrials.gov public records Last synced Oct 17, 2026, 7:16 AM EDT

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Showing 1009–1032 of 1,710 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 1 Interventional

Effects of Music Therapy on Huntington's Disease

NCT00178360
View directory record Official record
Conditions
Huntington's Disease
Interventions
Music Therapy
Behavioral
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Aug 22, 2011 · Synced Oct 17, 2026, 7:16 AM EDT
Completed No phase listed Observational

Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC

NCT01767779
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Day to 6 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2019 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Phase 1 Interventional

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

NCT01747135
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Industry
Eligibility
2 Years to 25 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 23, 2021 · Synced Oct 17, 2026, 7:16 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

Training Balance Among People With Neurodevelopmental Disabilities

NCT04293120
View directory record Official record
Conditions
Posture, Balance, Cerebral Palsy, Autism, Down Syndrome
Interventions
Training with weighted ball
Other
Lead sponsor
University of Hartford
Other
Eligibility
8 Years to 25 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
West Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 1, 2021 · Synced Oct 17, 2026, 7:16 AM EDT
Completed No phase listed Observational

Establishment of Biomarkers for Fabry Disease

NCT01165697
View directory record Official record
Conditions
Fabry Disease
Interventions
Fluorescein angiography
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 13, 2017 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

NCT02006472
View directory record Official record
Conditions
Huntington's Disease
Interventions
Pridopidine, Placebo
Drug · Other
Lead sponsor
Prilenia
Industry
Eligibility
21 Years and older
Enrollment
408 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
16
States / cities
La Jolla, California • Los Angeles, California • Englewood, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2021 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Not applicable Interventional

Coronary Artery Disease in Patients With Friedreich's Ataxia

NCT04649866
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
plethysmography
Diagnostic Test
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 79 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 8, 2023 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Phase 3 Interventional Results available

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

NCT03746522
View directory record Official record
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2023 · Synced Oct 17, 2026, 7:16 AM EDT
Recruiting No phase listed Observational

A Natural History Study of Angelman Syndrome

NCT07417137
View directory record Official record
Conditions
Angelman Syndrome, Neurodevelopmental Conditions
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
1 Year and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 7:16 AM EDT
Completed No phase listed Observational

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

NCT05356377
View directory record Official record
Conditions
Phenylketonurias
Interventions
Pegvaliase-Pqpz
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Boston, Massachusetts • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Dec 29, 2025 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Phase 4 Interventional Results available

Dexmedetomidine and Propofol for Pediatric MRI Sedation

NCT03513757
View directory record Official record
Conditions
Headache, Tumor, Seizure Disorder, Neurofibromatoses, Hydrocephalus, Abdominal Neoplasm, Spine Deformity
Interventions
propofol, Dexmedetomidine, Glycopyrrolate, Lidocaine 1% Injectable Solution, Nitrous Oxide, Sevoflurane
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
12 Months to 60 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 21, 2020 · Synced Oct 17, 2026, 7:16 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 17, 2026, 7:16 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

An Investigation of Pituitary Tumors and Related Hypothalmic Disorders

NCT00001595
View directory record Official record
Conditions
Panhypopituitarism, Gigantism/Acromegaly, Prolactinoma, Cushing Disease
Interventions
Tissue specimen collection, MRI
Procedure
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 70 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1997
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 17, 2026, 7:16 AM EDT
Active, not recruiting Phase 2 Interventional

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

NCT07157254
View directory record Official record
Conditions
Angelman Syndrome
Interventions
No intervention, GTX-102
Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year to 64 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
8
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 7:16 AM EDT
Not listed No phase listed Observational

Development of Assessments for Later Stage HD

NCT04370470
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
20 Years and older
Enrollment
340 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
9
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 6 more
Source: ClinicalTrials.gov public record
Updated May 1, 2020 · Synced Oct 17, 2026, 7:16 AM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
DNL952
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 17, 2026, 7:16 AM EDT
Completed No phase listed Observational

Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

NCT02311283
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Mar 7, 2018 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Not applicable Interventional Results available

Using Real-time fMRI Neurofeedback and Motor Imagery to Enhance Motor Timing and Precision in Cerebellar Ataxia

NCT05436262
View directory record Official record
Conditions
Cerebellar Ataxia, Spinocerebellar Ataxias, Cerebellar Degeneration
Interventions
Real-time fMRI with neurofeedback of motor imagery
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 8, 2025 · Synced Oct 17, 2026, 7:16 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 17, 2026, 7:16 AM EDT
Recruiting No phase listed Observational

Clinical Registry Investigating Bardet-Biedl Syndrome

NCT02329210
View directory record Official record
Conditions
Bardet-Biedl Syndrome
Interventions
Not listed
Lead sponsor
Marshfield Clinic Research Foundation
Other
Eligibility
Not listed
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 19, 2022 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Not applicable Interventional Results available

Electronic-health Application To Measure Outcomes REmotely Clinical Trial

NCT02418546
View directory record Official record
Conditions
Neurodegenerative Disease, Weight Loss, Amyotrophic Lateral Sclerosis, Parkinson's Disease, Huntington's Disease, Cachexia
Interventions
In-Person Nutritional Counseling by a Registered Dietitian, Nutritional counseling using an e-Health Application
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 20, 2020 · Synced Oct 17, 2026, 7:16 AM EDT
Enrolling by invitation Not applicable Interventional

Group Coaching Study for Life Goals

NCT07083713
View directory record Official record
Conditions
Students, Spinal Cord Injury, Ehlers Danlos Syndrome, Care Givers
Interventions
Group Coaching
Behavioral
Lead sponsor
Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 24, 2025 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Phase 1 Interventional

Influence of MMP on Brain AVM Hemorrhage

NCT00783523
View directory record Official record
Conditions
Arteriovenous Malformations, Cavernous Angiomas, Brain Aneurysms
Interventions
Doxycycline or Placebo
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
13 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 9, 2013 · Synced Oct 17, 2026, 7:16 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Copper Histidine Therapy for Menkes Diseases

NCT00001262
View directory record Official record
Conditions
Kinky Hair Syndrome
Interventions
Copper Histidine
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1990 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 30, 2015 · Synced Oct 17, 2026, 7:16 AM EDT