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ClinicalTrials.gov public records Last synced Oct 16, 2026, 2:28 AM EDT

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Showing 769–792 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated Phase 3 Interventional Results available

Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome

NCT02834793
View directory record Official record
Conditions
Lennox-Gastaut Syndrome (LGS)
Interventions
Placebo, Perampanel
Drug
Lead sponsor
Eisai Inc.
Industry
Eligibility
2 Years and older
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
31
States / cities
Little Rock, Arkansas • Palo Alto, California • Gulf Breeze, Florida + 25 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2022 · Synced Oct 16, 2026, 2:28 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease

NCT02855476
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
University College, London
Other
Eligibility
11 Years to 75 Years
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2017
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 16, 2026, 2:28 AM EDT
Completed Phase 3 Interventional Results available

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

NCT03566017
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years to 60 Years
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 16, 2026, 2:28 AM EDT
Not listed Phase 3 Interventional

Magnesium Sulfate to Prevent Brain Injury in Premature Infants

NCT00065949
View directory record Official record
Conditions
Brain Injuries, Cerebral Palsy
Interventions
magnesium sulfate
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
0 Years to 12 Hours
Healthy volunteers
Healthy volunteers not accepted
Timeline
1987 – 2003
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 16, 2026, 2:28 AM EDT
Completed No phase listed Observational

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

NCT05642221
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 110 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 16, 2026, 2:28 AM EDT
Completed Phase 2 Interventional Results available

Atomoxetine and Huntington's Disease

NCT00368849
View directory record Official record
Conditions
Huntington Disease, Chorea
Interventions
atomoxetine, Matching Placebo
Drug
Lead sponsor
University of Iowa
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Dec 20, 2012 · Synced Oct 16, 2026, 2:28 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Pulmonary Hypertension, Hypoxia and Sickle Cell Disease

NCT00495638
View directory record Official record
Conditions
Chuvash Polycythemia, Cerebrovascular Disease, Pulmonary Hypertension, Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 20 Years
Enrollment
29 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2014
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Oct 16, 2026, 2:28 AM EDT
Withdrawn Phase 2 Interventional

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

NCT04094948
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Clenbuterol, Placebos
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 17, 2022 · Synced Oct 16, 2026, 2:28 AM EDT
Completed Phase 1 Interventional Results available

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558
View directory record Official record
Conditions
Lesch-Nyhan Disease
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
San Diego, California • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 16, 2026, 2:28 AM EDT
Completed No phase listed Observational

Study on Moebius Syndrome and Congenital Facial Weakness Disorders

NCT02055248
View directory record Official record
Conditions
Brain Disorders, Birth Defects, Craniofacial Differences
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
207 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 16, 2026, 2:28 AM EDT
Completed No phase listed Observational

Genetic Analysis of Oculocerebrorenal Syndrome of Lowe

NCT00359515
View directory record Official record
Conditions
Lowe Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 16, 2026, 2:28 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 16, 2026, 2:28 AM EDT
Terminated Phase 1 Interventional Results available

Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer

NCT03478904
View directory record Official record
Conditions
Prostate Cancer
Interventions
Enzalutamide Liquid, Enzalutamide Capsule
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 10, 2021 · Synced Oct 16, 2026, 2:28 AM EDT
Not listed Phase 2 Interventional

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

NCT00004293
View directory record Official record
Conditions
Gaucher's Disease
Interventions
glucocerebrosidase
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 16, 2026, 2:28 AM EDT
Completed Phase 2 Interventional Results available

Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)

NCT03233204
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma, Malignant Glioma, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Childhood Malignant Germ Cell Tumor, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Wilms Tumor
Interventions
Olaparib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
111
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 16, 2026, 2:28 AM EDT
Recruiting Not applicable Interventional

Neurologic Stem Cell Treatment Study

NCT02795052
View directory record Official record
Conditions
Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders, Stroke, Traumatic Brain Injury, Cadasil, Chronic Traumatic Encephalopathy, Cerebral Infarction, Cerebral Ischemia, Cerebral Stroke, Cerebral Hemorrhage, Parkinson, Multi-System Degeneration, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy, ALS, Amyotrophic Lateral Sclerosis, Neuropathy, Diabetic Neuropathies, Alzheimer Disease, Dementia, Frontotemporal Dementia, Lewy Body Disease, Cognitive Impairment, Lewy Body Variant of Alzheimer Disease
Interventions
Intravenous and Intranasal BMSC
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Westport, Connecticut • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 26, 2026 · Synced Oct 16, 2026, 2:28 AM 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 16, 2026, 2:28 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Use of Real-Time Functional Magnetic Resonance Imaging Neurofeedback to Improve Motor Function in Cerebellar Ataxia

NCT05436249
View directory record Official record
Conditions
Healthy
Interventions
Neurofeedback treatment, At-home therapy
Device · Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 5, 2024 · Synced Oct 16, 2026, 2:28 AM 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 16, 2026, 2:28 AM EDT
Not listed No phase listed Observational

Using Wearable and Mobile Data to Diagnose and Monitor Movement Disorders

NCT04231487
View directory record Official record
Conditions
Essential Tremor, Parkinson Disease, Huntington Disease, Dystonia, Primary, Spinocerebellar Ataxias, Movement Disorders
Interventions
Not listed
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years to 80 Years
Enrollment
210 participants
Timeline
2019 – 2022
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 21, 2022 · Synced Oct 16, 2026, 2:28 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 16, 2026, 2:28 AM EDT
No Longer Available No phase listed Expanded access

Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)

NCT02084121
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
3 Years and older · Male only
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 18, 2022 · Synced Oct 16, 2026, 2:28 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

NCT03678324
View directory record Official record
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Oct 16, 2026, 2:28 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

NCT04526197
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Celecoxib
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 21, 2023 · Synced Oct 16, 2026, 2:28 AM EDT