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ClinicalTrials.gov public records Last synced Oct 15, 2026, 5:35 PM EDT

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Showing 697–720 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 1 Interventional

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

NCT02939547
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 15, 2026, 5:35 PM EDT
Withdrawn Not applicable Interventional Accepts healthy volunteers

The Sequestration of Holistic Stress Management Techniques for Adults

NCT06098755
View directory record Official record
Conditions
Stress, Job, PTSD, Hypervigilance, Hyperthyroidism, Migraine, Migraine With Aura, Immunodeficiency, Sickle Cell Trait, Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Fatigue, Anxiety, Depression, Flat Feet, Lordosis, TBI (Traumatic Brain Injury), Rheumatoid Arthritis, Arthritis, Masturbation, Priapism, Erectile Dysfunction, Flight Reaction, Hypertension, Sexual Arousal Disorder, Hemorrhoids, Fibroid
Interventions
Holistic Medicine (HM)
Behavioral
Lead sponsor
Goddess Zena I. Jones
Other
Eligibility
22 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
1
States / cities
Anchorage, Alaska
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Phase 3 Interventional Results available

Study of Eteplirsen in DMD Patients

NCT02255552
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 16 Years · Male only
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
37
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 32 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Oct 15, 2026, 5:35 PM EDT
Not yet recruiting Phase 1 Interventional

Escalating Doses of Memantine in Down Syndrome (MEDS-123)

NCT07531940
View directory record Official record
Conditions
Down Syndrome, Intellectual Disability, Alzheimer Dementia (AD)
Interventions
Memantine
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years to 32 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Recruiting No phase listed Observational

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

NCT05579548
View directory record Official record
Conditions
Phenylketonuria, Maternal
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Female only
Enrollment
50 participants
Timeline
2022 – 2032
U.S. locations
1
States / cities
Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies

NCT03059420
View directory record Official record
Conditions
Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome, Duane Radial Ray Syndrome, Mobius Syndrome, Brown Syndrome, Marcus Gunn Syndrome, Strabismus Congenital, Horizontal Gaze Palsy, Horizontal Gaze Palsy With Progressive Scoliosis, Facial Palsy, Facial Paresis, Hereditary, Congenital, Third Nerve Palsy, Fourth Nerve Palsy, Sixth Nerve Palsy, Synkinesis, Ocular Motility Disorders, Levator-Medial Rectus Synkinesis, Athabaskan Brainstem Dysgenesis, Tongue Paralysis, Ninth Nerve Disorder, Fifth Nerve Palsy, Seventh Nerve Palsy, Eleventh Nerve Disorder, Twelfth Nerve Disorder, Vagus Nerve Paralysis, Moebius Sequence
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Day and older
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Active, not recruiting Phase 3 Interventional

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

NCT06255782
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
Interventions
ECUR-506
Genetic
Lead sponsor
iECURE, Inc.
Industry
Eligibility
24 Hours to 7 Months · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Active, not recruiting No phase listed Observational

Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

NCT06529146
View directory record Official record
Conditions
Spinocerebellar Ataxias
Interventions
BHV-4157
Drug
Lead sponsor
Biohaven Therapeutics Ltd.
Industry
Eligibility
18 Years to 75 Years
Enrollment
909 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Huntington's Disease Biobank: Advancing Remote Monitoring and Deep Phenotyping

NCT06941662
View directory record Official record
Conditions
Huntington Disease
Interventions
computer vision, remote monitoring, blood, urine, wrist bands, mobile-app
Diagnostic Test
Lead sponsor
Stanford University
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2035
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Early Phase 1 Interventional

Using Ferumoxytol-Enhanced MRI to Measure Inflammation in Patients With Brain Tumors or Other Conditions of the CNS

NCT02452216
View directory record Official record
Conditions
Brain Injury, Central Nervous System Degenerative Disorder, Central Nervous System Infectious Disorder, Central Nervous System Vascular Malformation, Hemorrhagic Cerebrovascular Accident, Ischemic Cerebrovascular Accident, Primary Brain Neoplasm, Brain Cancer, Brain Tumors
Interventions
Ferumoxytol, Tissue Analysis, Magnetic Resonance Imaging
Drug · Other · Procedure
Lead sponsor
Michael Iv
Other
Eligibility
2 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Aug 31, 2017 · Synced Oct 15, 2026, 5:35 PM EDT
Completed No phase listed Observational

Evaluating Barriers to Stroke Screening and Prevention in Children With Sickle Cell Disease

NCT03621826
View directory record Official record
Conditions
Stroke Ischemic, Sickle Cell Disease
Interventions
Questionnaire/Interview
Other
Lead sponsor
Medical University of South Carolina
Other
Eligibility
2 Years to 24 Years
Enrollment
5,247 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Oct 15, 2026, 5:35 PM EDT
Not listed Not applicable Interventional

Clinical Characteristics and Temporal Properties of Individual Tics in Persistent Tic Disorder

NCT06385535
View directory record Official record
Conditions
Tics, Tic Disorders, Vocal Tic, Motor Tic, Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tics/Tremor
Interventions
Habit Reversal Training (HRT)
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 12 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 26, 2024 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Phase 2Phase 3 Interventional

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

NCT03231878
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
MIN-102, Placebos
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 15, 2026, 5:35 PM EDT
Recruiting Phase 3 Interventional

Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment

NCT06247085
View directory record Official record
Conditions
Homocystinuria
Interventions
Pegtibatinase, Placebo
Drug · Other
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • New Haven, Connecticut • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Phase 2 Interventional Results available

Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma

NCT00589784
View directory record Official record
Conditions
CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma, Neurofibromatosis
Interventions
Sunitinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
6
States / cities
Boston, Massachusetts • Basking Ridge, New Jersey • Commack, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2016 · Synced Oct 15, 2026, 5:35 PM EDT
Completed No phase listed Observational

Preliminary Study of the Scale To Assess Ataxia and Neurologic Dysfunction (STAND)

NCT02179333
View directory record Official record
Conditions
Spinocerebellar Ataxia - All Sub-types, Friedreich's Ataxia
Interventions
Ataxia rating scale
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 16, 2021 · Synced Oct 15, 2026, 5:35 PM EDT
Active, not recruiting Phase 2 Interventional

RNS System LGS Feasibility Study

NCT05339126
View directory record Official record
Conditions
Epilepsy, Lennox Gastaut Syndrome, Lennox-Gastaut Syndrome, Intractable, Seizures, Seizures, Generalized
Interventions
RNS System
Device
Lead sponsor
NeuroPace
Industry
Eligibility
12 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 15, 2026, 5:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Amyloid Deposition in Adults With Down Syndrome

NCT01303133
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
30 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2018
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 9, 2019 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Phase 2 Interventional Results available

A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod

NCT02215616
View directory record Official record
Conditions
Huntington's Disease
Interventions
Laquinimod, Placebo
Drug
Lead sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Industry
Eligibility
21 Years to 55 Years
Enrollment
352 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
16
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 13 more
Source: ClinicalTrials.gov public record
Updated May 4, 2020 · Synced Oct 15, 2026, 5:35 PM 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 15, 2026, 5:35 PM EDT
Completed Phase 1 Interventional

Gene Therapy for the Treatment of Brain Tumors

NCT00001328
View directory record Official record
Conditions
Brain Neoplasm, Neoplasm Metastasis
Interventions
Cytovene (Ganciclovir Sodium), G1TKSVNa.53 Producer Cell Line
Drug · Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
19 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

NCT02111200
View directory record Official record
Conditions
Urea Cycle Disorders, Inborn
Interventions
Sodium Benzoate, Sodium Phenylbutyrate
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 64 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 9, 2018 · Synced Oct 15, 2026, 5:35 PM EDT
Completed Phase 1 Interventional

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

NCT04519567
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 30, 2021 · Synced Oct 15, 2026, 5:35 PM EDT
Terminated Phase 4 Interventional Results available

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

NCT00230607
View directory record Official record
Conditions
Fabry Disease, Alpha Galactosidase A Deficiency
Interventions
agalsidase beta
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2024
U.S. locations
3
States / cities
Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Oct 15, 2026, 5:35 PM EDT