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ClinicalTrials.gov public records Last synced Oct 15, 2026, 6:24 PM EDT

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

Showing 721–744 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated Phase 2 Interventional Results available

Apalutamide Plus Cetrelimab in Patients With Treatment-Emergent Small Cell Neuroendocrine Prostate Cancer

NCT04926181
View directory record Official record
Conditions
Small Cell Neuroendocrine Carcinoma, Prostate Cancer, Small Cell Carcinoma
Interventions
Apalutamide, Cetrelimab
Drug · Biological
Lead sponsor
Rahul Aggarwal
Other
Eligibility
18 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 3 Interventional Results available

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

NCT00272792
View directory record Official record
Conditions
Phenylketonurias
Interventions
Sapropterin Dihydrochloride, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
4 Years to 12 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2015 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 2 Interventional Results available

Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers

NCT00141765
View directory record Official record
Conditions
Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer, Brain Tumor, Recurrent
Interventions
Myeloablative Chemotherapy, Stem Cell Rescue
Procedure
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2010
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 20, 2014 · Synced Oct 15, 2026, 6:24 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
3
States / cities
Atlanta, Georgia • Cleveland, Ohio • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Completed No phase listed Observational Results available

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

NCT02763215
View directory record Official record
Conditions
Wilson Disease
Interventions
Standard of Care Medications
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2020 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 2 Interventional Results available

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

NCT02451696
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Interventions
Everolimus
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
2 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Oct 15, 2026, 6:24 PM EDT
Recruiting No phase listed Observational

Krabbe Disease Global Patient Registry

NCT02993796
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Recruiting Not applicable Interventional

Adapting RDAD for DS

NCT07425769
View directory record Official record
Conditions
Down Syndrome (DS), Alzheimer's Disease (AD)
Interventions
Remote Exercise Sessions, Caregiver Education Sessions
Behavioral
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Recruiting No phase listed Observational

Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)

NCT06593951
View directory record Official record
Conditions
Progressive Myoclonus Epilepsy Type 1, EPM1, CSTB-related Disease, Myoclonus Epilepsies, Progressive, Unverricht-Lundborg Disease, Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, PME, Progressive Myoclonus-Epilepsies
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Not applicable Interventional

A Randomized Exercise Trial for Wheelchair Users

NCT00866112
View directory record Official record
Conditions
Spinal Cord Injury, Multiple Sclerosis, Muscular Dystrophy, Cerebral Palsy, Spina Bifida, Amputation, Arthritis, Stroke
Interventions
Educational session to promote physical activity, Minimal contact
Other
Lead sponsor
University of Kansas
Other
Eligibility
18 Years to 65 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Lawrence, Kansas
Source: ClinicalTrials.gov public record
Updated Jun 15, 2012 · Synced Oct 15, 2026, 6:24 PM EDT
Completed No phase listed Observational

The Impact of Community-based Wellness Programs on The Triple Aim

NCT02938130
View directory record Official record
Conditions
Spina Bifida, Cerebral Palsy, Spinal Cord Injury, Traumatic Brain Injury
Interventions
Wellness Programs
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
7 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 2Phase 3 Interventional

Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

NCT04902781
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007, Placebo
Drug · Other
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
3
States / cities
Aurora, Colorado • Atlanta, Georgia • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 15, 2026, 6:24 PM EDT
Completed No phase listed Observational

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

NCT00302146
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
15-0 H20
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 110 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Not listed Phase 3 Interventional

Vitamin E in Aging Persons With Down Syndrome

NCT00056329
View directory record Official record
Conditions
Down Syndrome, Alzheimer Disease
Interventions
Vitamin E, multivitamin, Placebo
Drug
Lead sponsor
New York State Institute for Basic Research
Government
Eligibility
50 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2012
U.S. locations
14
States / cities
Farmington, Connecticut • Tampa, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated May 4, 2012 · Synced Oct 15, 2026, 6:24 PM EDT
Completed No phase listed Observational

Quality of Life in Younger Leukemia and Lymphoma Survivors

NCT00956475
View directory record Official record
Conditions
Anxiety Disorder, Cancer Survivor, Fatigue, Leukemia, Long-term Effects Secondary to Cancer Therapy in Adults, Lymphoma, Lymphoproliferative Disorder, Pain, Psychosocial Effects of Cancer and Its Treatment, Small Intestine Cancer
Interventions
telephone-based intervention, questionnaire administration, assessment of therapy complications, fatigue assessment and management, psychosocial assessment and care, quality-of-life assessment
Behavioral · Other · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 35 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 24, 2011 · Synced Oct 15, 2026, 6:24 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Fabry Screening Study

NCT01019629
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
2,724 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 13, 2018 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 3 Interventional Results available

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies

NCT03842969
View directory record Official record
Conditions
Huntington Disease
Interventions
RO7234292 (RG6042)
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years and older
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
14
States / cities
Birmingham, Alabama • La Jolla, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 15, 2026, 6:24 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type 2

NCT00598351
View directory record Official record
Conditions
Neurofibromatosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 75 Years
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Genetic Disease Gene Identification

NCT00916903
View directory record Official record
Conditions
Congenital Vertical Talus, Familial Encephalopathy With Neuroserpin Inclusion Bodies, Idiopathic Generalised Epilepsy, Familial Dementia, X-linked Mental Retardation
Interventions
Not listed
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
6 Months and older
Enrollment
176 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Jan 2, 2024 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 2 Interventional Results available

Impact of Probiotics on Urinary Symptoms in Spinal Cord Injury SCI and SB

NCT02748356
View directory record Official record
Conditions
Lower Urinary Tract Symptoms
Interventions
Lactobacillus
Drug
Lead sponsor
Medstar Health Research Institute
Other
Eligibility
6 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Apr 24, 2020 · Synced Oct 15, 2026, 6:24 PM EDT
Recruiting Not applicable Interventional

Deep Brain Stimulation for Spinocerebellar Ataxia

NCT07288437
View directory record Official record
Conditions
Spinocerebellar Ataxia (SCA), Spinocerebellar Ataxia Type 6
Interventions
Deep Brain Stimulation (DBS)
Device
Lead sponsor
University of California, San Francisco
Other
Eligibility
21 Years to 89 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 15, 2026, 6:24 PM EDT
Terminated Phase 1 Interventional

Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors

NCT00003926
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Retinoblastoma, Sarcoma
Interventions
amifostine trihydrate, busulfan, filgrastim, melphalan, thiotepa, peripheral blood stem cell transplantation (PBSC)
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
1 Year to 45 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 29, 2017 · Synced Oct 15, 2026, 6:24 PM EDT
Completed Phase 3 Interventional Results available

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

NCT00838435
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 6 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced Oct 15, 2026, 6:24 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)

NCT02316314
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 15, 2026, 6:24 PM EDT