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ClinicalTrials.gov public records Last synced Oct 25, 2026, 1:52 AM EDT

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Showing 1513–1536 of 1,712 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Recruiting Not applicable Interventional

Investigating Changes in Premonitory Urges During Habit Reversal Training for Tics

NCT06825520
View directory record Official record
Conditions
Tics, Tourette Disorder, Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions
Habit Reversal Training
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2025 · Synced Oct 25, 2026, 1:52 AM EDT
Completed No phase listed Observational

Individuals Patterns of Disclosure About Huntington s Disease (HD) and the Association With Adaptation to HD

NCT00491842
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 1 Interventional

Oxaliplatin and Irinotecan in Treating Young Patients With Refractory Solid Tumors or Lymphomas

NCT00101270
View directory record Official record
Conditions
Childhood Burkitt Lymphoma, Childhood Central Nervous System Germ Cell Tumor, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway Glioma, Recurrent Colon Cancer, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Melanoma, Recurrent Nasopharyngeal Cancer, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Recurrent/Refractory Childhood Hodgkin Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
irinotecan hydrochloride, oxaliplatin
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 21 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Arcadia, California
Source: ClinicalTrials.gov public record
Updated Jun 5, 2013 · Synced Oct 25, 2026, 1:52 AM EDT
Completed No phase listed Observational

Development of the Virtual Unified Huntington's Disease Rating Scale

NCT05748288
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
17
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2023 · Synced Oct 25, 2026, 1:52 AM EDT
Not listed No phase listed Observational

NF2 Natural History Consortium

NCT00004483
View directory record Official record
Conditions
Schwannoma, Vestibular, Neurofibromatosis 2, Meningioma
Interventions
Not listed
Lead sponsor
House Ear Institute
Other
Eligibility
5 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 29, 2005 · Synced Oct 25, 2026, 1:52 AM EDT
Active, not recruiting Not applicable Interventional

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
No Intervention
Other
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
16 Years to 69 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Atlanta, Georgia • Kansas City, Kansas • Ann Arbor, Michigan + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396
View directory record Official record
Conditions
Canavan Disease
Interventions
AAV9 BBP-812
Biological
Lead sponsor
Aspa Therapeutics
Industry
Eligibility
Up to 30 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Recruiting No phase listed Observational

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

NCT04404530
View directory record Official record
Conditions
Phenylketonurias
Interventions
Palynziq
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
40
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 1 Interventional

HSV1716 in Patients With Non-Central Nervous System (Non-CNS) Solid Tumors

NCT00931931
View directory record Official record
Conditions
Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma, Soft Tissue Sarcoma, Neuroblastoma, Wilms Tumor, Malignant Peripheral Nerve Sheath Tumor, Clival Chordoma, Non-CNS Solid Tumors
Interventions
HSV1716
Biological
Lead sponsor
Timothy Cripe
Other
Eligibility
7 Years to 30 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 21, 2018 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Not applicable Interventional

Neurofeedback for Tourette Syndrome

NCT01702077
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder
Interventions
Neurofeedback, Sham feedback
Procedure
Lead sponsor
Yale University
Other
Eligibility
11 Years to 19 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 27, 2018 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Not applicable Interventional Results available

Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome

NCT02067819
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder
Interventions
Active Oral Orthotic Treatment, Placebo Oral Orthotic Treatment
Device
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
7 Years to 25 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2022 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 1Phase 2 Interventional Results available

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

NCT00661453
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
Valproic Acid and Levocarnitine
Drug
Lead sponsor
University of Utah
Other
Eligibility
2 Weeks to 12 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
6
States / cities
Baltimore, Maryland • Detroit, Michigan • Durham, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2015 · Synced Oct 25, 2026, 1:52 AM EDT
Terminated Phase 3 Interventional Results available

Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

NCT01751802
View directory record Official record
Conditions
Lesch-Nyhan Disease, Self-injurious Behavior
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Nitric Oxide Production in MELAS Syndrome

NCT01339494
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Arginine and citrulline supplementations
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
3 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2016 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 1 Interventional

A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain Cancer

NCT03423628
View directory record Official record
Conditions
Recurrent Glioblastoma Multiforme, Primary Glioblastoma Multiforme, Brain Neoplasms, Malignant, Leptomeningeal Disease (LMD)
Interventions
Radiation Therapy, AZD1390
Radiation · Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
5
States / cities
Boston, Massachusetts • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 25, 2026, 1:52 AM 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 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Glucose Metabolic, Amyloid, and Tau Brain Imaging in Down Syndrome and Dementia

NCT00966017
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
National Institute on Aging (NIA)
NIH
Eligibility
45 Years and older
Enrollment
108 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 29, 2009 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

NCT02797080
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
interferon γ-1b
Drug
Lead sponsor
Amgen
Industry
Eligibility
11 Years to 27 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 2 Interventional Results available

Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)

NCT03213678
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Malignant Glioma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Wilms Tumor
Interventions
Biospecimen Collection, Computed Tomography, FDG-Positron Emission Tomography, Magnetic Resonance Imaging, Samotolisib, X-Ray Imaging
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
125
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 96 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 25, 2026, 1:52 AM EDT
Not listed Not applicable Interventional

Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)

NCT05174559
View directory record Official record
Conditions
Phenylketonurias
Interventions
PreKUnil® LNAA Medical Food for PKU
Other
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 4, 2023 · Synced Oct 25, 2026, 1:52 AM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia

NCT00537680
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone, Placebo
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 17 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 11, 2023 · Synced Oct 25, 2026, 1:52 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL) Study

NCT05677880
View directory record Official record
Conditions
CADASIL
Interventions
Study Procedures
Other
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older
Enrollment
660 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
12
States / cities
Los Angeles, California • San Francisco, California • Denver, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 25, 2026, 1:52 AM EDT