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ClinicalTrials.gov public records Last synced Oct 12, 2026, 4:17 PM EDT

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Showing 193–216 of 1,707 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 4 Interventional Results available

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
9
States / cities
Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

NCT01994889
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2019 · Synced Oct 12, 2026, 4:17 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria

NCT03097250
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years to 25 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 19, 2018 · Synced Oct 12, 2026, 4:17 PM EDT
Withdrawn No phase listed Observational

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 8, 2015 · Synced Oct 12, 2026, 4:17 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Amyloid Plaque and Tangle Imaging in Alzheimer's Disease and Down Syndrome

NCT00965835
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
National Institute on Aging (NIA)
NIH
Eligibility
21 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 29, 2009 · Synced Oct 12, 2026, 4:17 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

The Effect of tDCS on Subcortical Brain Functioning

NCT01602276
View directory record Official record
Conditions
Brain Damage, Chronic
Interventions
Transcranial Direct Current Stimulation (tDCS)
Procedure
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 79 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 20, 2018 · Synced Oct 12, 2026, 4:17 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT05127226
View directory record Official record
Conditions
Angelman Syndrome
Interventions
ION582
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
San Diego, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 12, 2026, 4:17 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 12, 2026, 4:17 PM EDT
Withdrawn No phase listed Observational

Breast Cancer BRCA1 Carriers: a Pilot Study

NCT05062174
View directory record Official record
Conditions
BRCA1 Mutation, High-grade Serous Ovarian Cancer, TNBC - Triple-Negative Breast Cancer
Interventions
Mifepristone 200 MG, Prophylactic mastectomy
Drug · Procedure
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Carmel, Indiana • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 15, 2022 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 1 Interventional Results available

Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease

NCT03664804
View directory record Official record
Conditions
Huntington's Disease
Interventions
No Study Drug was Administered in this Study
Other
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 65 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Wichita, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 3, 2025 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 3 Interventional Results available

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

NCT00104247
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2014 · Synced Oct 12, 2026, 4:17 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

NCT05028764
View directory record Official record
Conditions
Friedreich Ataxia, Healthy Volunteers
Interventions
Buccal Swabs, Blood Draws and Skin Punch Biopsy
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 24, 2022 · Synced Oct 12, 2026, 4:17 PM EDT
Active, not recruiting Not applicable Interventional

AMPLATZER PFO Occluder Post Approval Study

NCT03309332
View directory record Official record
Conditions
Stroke, Patent Foramen Ovale
Interventions
AMPLATZER™ PFO Occluder
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years to 60 Years
Enrollment
1,214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2030
U.S. locations
78
States / cities
Birmingham, Alabama • Huntsville, Alabama • Chandler, Arizona + 63 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Oct 12, 2026, 4:17 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neuroimaging of Dystonia

NCT02046447
View directory record Official record
Conditions
Primary Cervical Dystonia, DYT 1 Dystonia
Interventions
Primary Cervical Dystonia (Trihexyphenidyl), Controls Primary Cervical Dystonia (Trihexyphenidyl), DYT 1 Dystonia (Healthy Control), DYT 1 Dystonia
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 22, 2017 · Synced Oct 12, 2026, 4:17 PM EDT
Completed No phase listed Observational

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304
View directory record Official record
Conditions
Gaucher Disease
Interventions
Imiglucerase
Drug
Lead sponsor
University Research Foundation for Lysosomal Storage Diseases, Inc.
Other
Eligibility
18 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Beverly Hills, California • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 12, 2026, 4:17 PM EDT
Not listed Not applicable Interventional

Online-Delivered Comprehensive Behavioral Intervention for Tics-Enhanced (CBIT-E)

NCT05050734
View directory record Official record
Conditions
Persistent Tic Disorders, Tourette Disorder
Interventions
Comprehensive Behavioral Intervention for Tics - Enhanced (CBIT-E)
Behavioral
Lead sponsor
Marquette University
Other
Eligibility
9 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Oct 8, 2021 · Synced Oct 12, 2026, 4:17 PM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 8, 2014 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)

NCT05107128
View directory record Official record
Conditions
Huntington's Disease
Interventions
SAGE-718, Placebo
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
25 Years to 65 Years
Enrollment
189 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
34
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

NCT01721733
View directory record Official record
Conditions
Leigh Syndrome
Interventions
Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years to 17 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
4
States / cities
Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 3 Interventional Results available

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600
View directory record Official record
Conditions
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa, Placebo
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2015 · Synced Oct 12, 2026, 4:17 PM EDT
Active, not recruiting Not applicable Interventional

Incontinence and Quality of Life in Children With Spina Bifida

NCT03410667
View directory record Official record
Conditions
Spina Bifida, Myelomeningocele
Interventions
Phase One: Exploration, Phase Two: Creation, Phase Three: Reflection, Aim 3
Behavioral
Lead sponsor
Indiana University
Other
Eligibility
8 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

NCT02445794
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Low dose cohort, High dose cohort
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
18 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Long Beach, California • Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 27, 2020 · Synced Oct 12, 2026, 4:17 PM EDT
Completed Not applicable Interventional

Medication Management Among Individuals With Neurodevelopmental Disabilities

NCT03121651
View directory record Official record
Conditions
Spina Bifida, Cerebral Palsy
Interventions
RL-adaptive application
Device
Lead sponsor
University of Michigan
Other
Eligibility
18 Years to 99 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Apr 2, 2019 · Synced Oct 12, 2026, 4:17 PM EDT