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ClinicalTrials.gov public records Last synced Oct 14, 2026, 9:11 PM EDT

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Showing 457–480 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 1 Interventional

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B

NCT03787758
View directory record Official record
Conditions
Huntington Disease
Interventions
SAGE-718
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
2
States / cities
Long Beach, California • Marlton, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 17, 2025 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A More Engaging Visual Field Test to Increase Use and Reliability in Pediatrics

NCT02157025
View directory record Official record
Conditions
Glaucoma, Suspect, Optic Nerve Hypoplasia and Abnormalities of the Central Nervous System, Congenital Coloboma of the Optic Nerve, Optic Nerve Head Pits, Bilateral Congenital
Interventions
Cartoon video fixation target and cartoon character voice audio instructions during Humphrey perimetry, Usual Care procedures during Humphrey perimetry for children
Device · Other
Lead sponsor
Nova Southeastern University
Other
Eligibility
5 Years to 8 Years
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 14, 2026, 9:11 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

NCT01694940
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 14, 2026, 9:11 PM EDT
Recruiting No phase listed Observational

Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)

NCT05299710
View directory record Official record
Conditions
Cancers
Interventions
Laparoscopic surgery
Procedure
Lead sponsor
Erin Rowell
Other
Eligibility
Up to 11 Years · Female only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2035
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00947297
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
21
States / cities
Long Beach, California • Los Angeles, California • Stanford, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Oct 14, 2026, 9:11 PM EDT
Terminated Phase 2 Interventional

Safety and Efficacy of OMS643762 in Subjects With Huntington's Disease

NCT02074410
View directory record Official record
Conditions
Huntington's Disease
Interventions
OMS643762, Placebo
Drug
Lead sponsor
Omeros Corporation
Industry
Eligibility
18 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
9
States / cities
San Diego, California • Englewood, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2018 · Synced Oct 14, 2026, 9:11 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

NCT02895789
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 8, 2022 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 2 Interventional Results available

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
GZ402665
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 14, 2026, 9:11 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Rocky Mountain Alzheimer's Disease Center Longitudinal Biomarker and Clinical Phenotyping Study

NCT02612376
View directory record Official record
Conditions
Alzheimer Disease, Down Syndrome, Mild Cognitive Impairment
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 3, 2025 · Synced Oct 14, 2026, 9:11 PM EDT
Recruiting No phase listed Observational

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Gainesville, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 14, 2026, 9:11 PM EDT
Recruiting Not applicable Interventional

Measuring Aided Language Development

NCT06512168
View directory record Official record
Conditions
Down Syndrome, Cerebral Palsy, Apraxia of Speech, Speech Sound Disorder, Speech Disorders in Children, Speech and Language Disorder
Interventions
AAC Generative Language Intervention
Behavioral
Lead sponsor
University of Central Florida
Other
Eligibility
42 Months to 60 Months
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated May 16, 2025 · Synced Oct 14, 2026, 9:11 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome

NCT05783791
View directory record Official record
Conditions
Angelman Syndrome, Prader-Willi Syndrome
Interventions
Newborn Screening Assay
Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 6, 2023 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Not applicable Interventional

Nutritional Formulation for Angelman Syndrome

NCT03644693
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Nutritional Formulation Containing Exogenous Ketones, Placebo Formulation
Dietary Supplement
Lead sponsor
University of Colorado, Denver
Other
Eligibility
4 Years to 11 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Aug 20, 2020 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2016 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

NCT06193304
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 14, 2026, 9:11 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors

NCT00788125
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Lymphoma, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
carboplatin, dasatinib, etoposide phosphate, ifosfamide, microarray analysis, western blotting, immunohistochemistry staining method, laboratory biomarker analysis, therapeutic conventional surgery, radiation therapy
Drug · Genetic · Other + 2 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
1 Year to 25 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2022
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jul 14, 2023 · Synced Oct 14, 2026, 9:11 PM EDT
Completed No phase listed Observational

Study of the Neurobiology of Tourette Syndrome and Related Disorders

NCT00004325
View directory record Official record
Conditions
Tourette Syndrome, Obsessive Compulsive Disorder
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
12 Years to 45 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1988
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 1 Interventional Results available

Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome

NCT02738450
View directory record Official record
Conditions
Down Syndrome
Interventions
ACI-24 low dose, ACI-24 high dose, Placebo
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
25 Years to 45 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
4
States / cities
Phoenix, Arizona • La Jolla, California • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2021 · Synced Oct 14, 2026, 9:11 PM EDT
Enrolling by invitation No phase listed Observational

GABA and GSH in FRDA

NCT07635030
View directory record Official record
Conditions
Friedreich's Ataxia, FRDA
Interventions
MRI/ MRS (Magnetic Resonance Imaging /Magnetic Resonance Spectroscopy)
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
8 Years to 16 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 14, 2026, 9:11 PM EDT
Recruiting No phase listed Observational

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

NCT05588167
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Months and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

NCT04102501
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
RT001, Placebo
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
12 Years to 50 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 27, 2022 · Synced Oct 14, 2026, 9:11 PM EDT
Recruiting No phase listed Observational

Angelman Syndrome Natural History Study

NCT04507997
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2031
U.S. locations
8
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2025 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 4 Interventional Results available

Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia

NCT00140413
View directory record Official record
Conditions
Growth Hormone Deficiency, Septo-Optic Dysplasia, Hypopituitarism
Interventions
Nutropin AQ
Drug
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 5 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 1, 2015 · Synced Oct 14, 2026, 9:11 PM EDT
Completed Phase 3 Interventional Accepts healthy volunteers

Antenatal Phenobarbital to Prevent Neonatal Intracranial Hemorrhage

NCT00009620
View directory record Official record
Conditions
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Intracranial Hemorrhages
Interventions
Phenobarbital, Saline
Drug
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
Female only
Enrollment
610 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1993 – 1997
U.S. locations
11
States / cities
Palo Alto, California • New Haven, Connecticut • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2019 · Synced Oct 14, 2026, 9:11 PM EDT