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ClinicalTrials.gov public records Last synced Oct 2, 2026, 1:59 AM EDT

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Showing 25–48 of 1,705 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated No phase listed Observational Accepts healthy volunteers

Investigation of Neurofeedback With Real-Time fMRI in Healthy Volunteers and Patients With Hyperkinetic Movement Disorders

NCT00885040
View directory record Official record
Conditions
Movement Disorder, Tourette Syndrome, Healthy Volunteer
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 60 Years
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2019 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Phase 1 Interventional

Open-Trial of EPI-743 for Adults With Tourette Syndrome

NCT01719523
View directory record Official record
Conditions
Tourette Syndrome
Interventions
EPI-743
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 5, 2014 · Synced Oct 2, 2026, 1:59 AM EDT
Terminated Phase 1 Interventional

12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder

NCT00004058
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition
Interventions
tetradecanoylphorbol acetate
Drug
Lead sponsor
University of Medicine and Dentistry of New Jersey
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2004
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 26, 2010 · Synced Oct 2, 2026, 1:59 AM 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 2, 2026, 1:59 AM EDT
Recruiting Phase 1Phase 2 Interventional

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease

NCT07398508
View directory record Official record
Conditions
Menkes Disease
Interventions
Droxidopa Oral Product, Placebo Control
Drug · Other
Lead sponsor
Stephen G. Kaler
Other
Eligibility
7 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Phase 2 Interventional Results available

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

NCT04007991
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
53
States / cities
Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 2, 2026, 1:59 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 2, 2026, 1:59 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetic, and Efficacy Study of AZD5213 in Adolescents With Tourette's Disorder

NCT01904773
View directory record Official record
Conditions
Tourette Syndrome
Interventions
AZD5213 and placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
12 Years to 17 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
7
States / cities
Orange, California • St. Petersburg, Florida • Summit, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2016 · Synced Oct 2, 2026, 1:59 AM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Gaucher Disease

NCT01716741
View directory record Official record
Conditions
Gaucher Disease
Interventions
Enzyme analysis
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 30, 2012 · Synced Oct 2, 2026, 1:59 AM EDT
Completed No phase listed Observational

Prediction of Brain Injury in Premature Infants

NCT02814383
View directory record Official record
Conditions
Intraventricular Hemorrhage, Periventricular Leukomalacia, Brain Injury, Extreme Prematurity
Interventions
Brain Monitoring Platform
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
0 Hours to 12 Hours
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
3
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 2, 2026, 1:59 AM EDT
Completed No phase listed Observational

VAlidation of a Lower Cost aneUploidy scrEen

NCT03087357
View directory record Official record
Conditions
Down Syndrome, Trisomy 18, Trisomy 13
Interventions
SmartNIPT
Other
Lead sponsor
Women and Infants Hospital of Rhode Island
Other
Eligibility
18 Years and older · Female only
Enrollment
2,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
13
States / cities
Los Angeles, California • Aurora, Colorado • Bridgeport, Connecticut + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2022 · Synced Oct 2, 2026, 1:59 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Not applicable Interventional Results available

PREMIUM Migraine Trial

NCT00355056
View directory record Official record
Conditions
Migraine Headaches, Patent Foramen Ovale
Interventions
Sham Procedure, AMPLATZER PFO Occluder
Other · Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years to 65 Years
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
30
States / cities
Phoenix, Arizona • Los Angeles, California • Colorado Springs, Colorado + 23 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 2, 2026, 1:59 AM EDT
Not yet recruiting Phase 2 Interventional

Neoadjuvant Pembrolizumab and Porustobart in Resectable pMMR/MSS Colon Cancer

NCT07808151
View directory record Official record
Conditions
Colon Cancer, Microsatellite Stable (MSS) Colon Cancer, Proficient Mismatch Repair (pMMR) Colon Cancer
Interventions
Pembrolizumab, Porustobart
Drug
Lead sponsor
Solstice Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
11
States / cities
Los Angeles, California • Tampa, Florida • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 2, 2026, 1:59 AM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)

NCT05764239
View directory record Official record
Conditions
Phenylketonuria
Interventions
SYNB1934v1, Placebo
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
14
States / cities
Culver City, California • Orange, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jun 20, 2024 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 2, 2026, 1:59 AM EDT
Terminated Phase 1 Interventional

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160
View directory record Official record
Conditions
Type 1 Gaucher Disease
Interventions
AT2101
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Miramar, Florida
Source: ClinicalTrials.gov public record
Updated Aug 19, 2010 · Synced Oct 2, 2026, 1:59 AM EDT
Terminated Phase 2Phase 3 Interventional

Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

NCT00803868
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
varenicline, placebo
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 24, 2012 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

NCT03325010
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Valbenazine, Placebo oral capsule
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 17 Years
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
34
States / cities
Sun City, Arizona • Rogers, Arkansas • Anaheim, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Oct 2, 2026, 1:59 AM EDT
Completed Early Phase 1 Interventional

Pimavanserin Treatment in TS

NCT04794413
View directory record Official record
Conditions
Tourette Syndrome, Tardive Dyskinesia
Interventions
Pimavanserin
Drug
Lead sponsor
Joseph Jankovic
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 12, 2021 · Synced Oct 2, 2026, 1:59 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 2, 2026, 1:59 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 2, 2026, 1:59 AM EDT
Terminated Phase 1 Interventional

FAAH Inhibitor Trial for Adults With Tourette Syndrome

NCT02134080
View directory record Official record
Conditions
Tourette Syndrome
Interventions
PF-04457845, Placebo
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 60 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Oct 2, 2026, 1:59 AM EDT