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ClinicalTrials.gov public records Last synced Oct 16, 2026, 6:16 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 817–840 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 1Phase 2 Interventional Results available

Ecopipam Treatment of Tourette Syndrome

NCT01244633
View directory record Official record
Conditions
Tourette's Syndrome
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
4
States / cities
Baltimore, Maryland • Summit, New Jersey • Manhasset, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2015 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Not applicable Interventional

A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

NCT03911089
View directory record Official record
Conditions
Urea Cycle Disorder, Inborn Errors of Metabolism
Interventions
UCD Anamix Infant
Other
Lead sponsor
Nutricia Research
Industry
Eligibility
Up to 30 Days
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Oct 16, 2026, 6:16 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of the Nervous System in Patients With Fabry's Disease

NCT00001491
View directory record Official record
Conditions
Fabry's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
325 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 16, 2026, 6:16 AM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

NCT00244218
View directory record Official record
Conditions
Phenylketonuria
Interventions
tetrahydrobiopterin (BH4)
Drug
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
10 Years and older
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 21, 2021 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 1Phase 2 Interventional

Temozolomide Plus Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Malignant Glioma or Recurrent CNS or Other Solid Tumors

NCT00005952
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Head and Neck Cancer, Kidney Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
Interventions
filgrastim, temozolomide, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Duke University
Other
Eligibility
Up to 18 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 20, 2013 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

NCT00789568
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride, Moxifloxacin, Moxifloxacin placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Fargo, North Dakota
Source: ClinicalTrials.gov public record
Updated Jul 23, 2021 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 2 Interventional

A Study in Subjects With Late Prodromal & Early Manifest HD to Assess the Safety, Tolerability, pk, and Efficacy of Pepi

NCT02481674
View directory record Official record
Conditions
Huntington's Disease
Interventions
VX15/2503, Placebo
Drug
Lead sponsor
Vaccinex Inc.
Industry
Eligibility
21 Years and older
Enrollment
301 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
29
States / cities
Birmingham, Alabama • La Jolla, California • San Francisco, California + 24 more
Source: ClinicalTrials.gov public record
Updated May 2, 2022 · Synced Oct 16, 2026, 6:16 AM EDT
Recruiting No phase listed Observational

Gonadal Tissue Freezing for Fertility Preservation in Individuals at Risk for Ovarian Dysfunction, Premature Ovarian Insufficiency and Clinically Indicated Gonadectomy

NCT04948658
View directory record Official record
Conditions
Turner Syndrome, Post-menarcheal Adolescents, Ovarian Disfunction, Galactosemia, Variations in Sex Characteristics, Differences in Sex Development
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 35 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 16, 2026, 6:16 AM EDT
Terminated Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228
View directory record Official record
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 12, 2023 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

NCT03516487
View directory record Official record
Conditions
Phenylketonuria, Healthy
Interventions
SYNB1618, Placebo
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years to 64 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
4
States / cities
Boston, Massachusetts • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 3 Interventional

Personalized Information or Basic Information in Helping Patients Make Decisions About Participating in a Clinical Trial

NCT00750009
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
educational intervention
Other
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
583 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
4
States / cities
Detroit, Michigan • Cleveland, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 27, 2020 · Synced Oct 16, 2026, 6:16 AM EDT
Completed No phase listed Observational

Pompe Gene Therapy- Screening for Eligibility

NCT03285126
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 2, 2019 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls

NCT01523964
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Testing with EIM
Other
Lead sponsor
Dart Therapeutics. LLC
Other
Eligibility
3 Years to 12 Years · Male only
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
6
States / cities
Aurora, Colorado • Boston, Massachusetts • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2013 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 2 Interventional

A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

NCT05485987
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 6 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 22, 2025 · Synced Oct 16, 2026, 6:16 AM EDT
Completed Phase 3 Interventional Results available

Tanezumab In Osteoarthritis Of The Knee (2)

NCT00830063
View directory record Official record
Conditions
Arthritis, Osteoarthritis
Interventions
tanezumab 10 mg, tanezumab 5 mg, naproxen, placebo
Biological · Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
832 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
94
States / cities
Anniston, Alabama • Mesa, Arizona • Paradise Valley, Arizona + 72 more
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Oct 16, 2026, 6:16 AM EDT
Active, not recruiting Not applicable Interventional

Effects of a Physical Therapy Intervention on Motor Delay in Infants Admitted to a Neonatal Intensive Care Unit

NCT05568264
View directory record Official record
Conditions
Motor Delay, Premature Birth, Intraventricular Hemorrhage, Hypoxic-Ischemic Encephalopathy, Bronchopulmonary Dysplasia
Interventions
Physical Therapy intervention
Other
Lead sponsor
Shirley Ryan AbilityLab
Other
Eligibility
33 Weeks to 48 Weeks
Enrollment
222 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 16, 2026, 6:16 AM 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 16, 2026, 6:16 AM EDT
Recruiting Not applicable Interventional

Early Feasibility Study (EFS) Evaluating Percutaneous Repair of the Atrial Septum With a Novel PFO Occluder: The PROTEA-PFO Study

NCT07172464
View directory record Official record
Conditions
PFO, PFO - Patent Foramen Ovale, Cryptogenic Stroke, Patent Foramen Ovale, PFO-associated Stroke
Interventions
Recross P3 Occluder (P3O) System
Device
Lead sponsor
Recross Cardio, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
5
States / cities
Los Angeles, California • Gainsville, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 16, 2026, 6:16 AM EDT
Recruiting No phase listed Observational

Sensory Symptoms in Tourette Syndrome

NCT03851484
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 16, 2026, 6:16 AM EDT
Completed No phase listed Observational

Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

NCT01391637
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease, PMD
Interventions
HuCNS-SC transplant in the lead-in phase
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 13, 2016 · Synced Oct 16, 2026, 6:16 AM EDT
Completed No phase listed Observational

Prognostic Value of Transforming Growth Factor-Beta 1 in Normal Pressure Hydrocephalus

NCT00600795
View directory record Official record
Conditions
Normal Pressure Hydrocephalus
Interventions
CSF collection
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 16, 2026, 6:16 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Skeletal Muscle Effects of GH in Boys

NCT04020913
View directory record Official record
Conditions
Growth Hormone Deficiency, Idiopathic Short Stature
Interventions
Somatropin injection
Drug
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
6 Years to 11 Years · Male only
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 4, 2025 · Synced Oct 16, 2026, 6:16 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

NCT04537377
View directory record Official record
Conditions
Wilson's Disease
Interventions
VTX-801
Genetic
Lead sponsor
Vivet Therapeutics SAS
Industry
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
Sacramento, California • New Haven, Connecticut • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 16, 2026, 6:16 AM EDT