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ClinicalTrials.gov public records Last synced Oct 29, 2026, 9:50 AM EDT

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Showing 1681–1704 of 1,713 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
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 29, 2026, 9:50 AM EDT
Completed No phase listed Observational Results available

CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness

NCT03652181
View directory record Official record
Conditions
CCM, Cavernoma, Cerebral Cavernous Malformation, Cerebral Cavernous Malformations 1, Cerebral Cavernous Malformations 2, Cerebral Cavernous Malformations 3, Cavernous Angioma
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
18 Years and older
Enrollment
123 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Not applicable Interventional Results available

The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study

NCT01917344
View directory record Official record
Conditions
Phenylketonuria
Interventions
MRI
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 19, 2015 · Synced Oct 29, 2026, 9:50 AM EDT
Terminated Phase 2 Interventional Results available

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 18, 2019 · Synced Oct 29, 2026, 9:50 AM EDT
Recruiting Not applicable Interventional

PHIL® Embolic System Pediatric IDE

NCT03731000
View directory record Official record
Conditions
Arterio-venous Fistula, Intracranial Arteriovenous Malformations
Interventions
PHIL® device
Device
Lead sponsor
Alejandro Berenstein
Other
Eligibility
Up to 21 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

NCT02536911
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 10, 2017 · Synced Oct 29, 2026, 9:50 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
zocaglusagene nuzaparvovec
Genetic
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
18 Years to 80 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
3
States / cities
Orange, California • Palo Alto, California • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 3 Interventional Results available

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

NCT00705939
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
2
States / cities
Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 29, 2026, 9:50 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

COVID-19 In-vitro Diagnostic Test and Androgen Receptor Gene Expression

NCT04530500
View directory record Official record
Conditions
Covid19, Androgen Receptor Gene Overexpression
Interventions
COVID-19 Androgen Sensitivity Test (CoVAST)
Genetic
Lead sponsor
University of California, Irvine
Other
Eligibility
18 Years to 90 Years · Male only
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
2
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated May 8, 2024 · Synced Oct 29, 2026, 9:50 AM EDT
Completed No phase listed Observational

Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy

NCT02124057
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy (SBMA), Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 18, 2022 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 1Phase 2 Interventional

Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

NCT01369901
View directory record Official record
Conditions
Motor Neuron Disease
Interventions
A functional exercise program, A stretching exercise program
Behavioral
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 90 Years · Male only
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 29, 2026, 9:50 AM EDT
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Vicriviroc (MK-7690) in Combination With Pembrolizumab (MK-3475) in Participants With Advanced/Metastatic Microsatellite Stable (MSS) Colorectal Cancer (CRC) (MK-7690-046)

NCT03631407
View directory record Official record
Conditions
Colorectal Neoplasms
Interventions
Vicriviroc, Pembrolizumab
Drug · Biological
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
Scottsdale, Arizona • Encinitas, California • Fresno, California + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 29, 2026, 9:50 AM EDT
Withdrawn Early Phase 1 Interventional

D-cycloserine Augmented Treatment for Youth With Tic Disorders

NCT04357951
View directory record Official record
Conditions
Tourette Syndrome
Interventions
d-cycloserine, Placebo pill, Behavior Therapy
Drug · Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
8 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 14, 2023 · Synced Oct 29, 2026, 9:50 AM EDT
Terminated No phase listed Observational

Studying Fentanyl in Patients With Cancer

NCT00899951
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, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
fentanyl citrate, pharmacological study
Drug · Other
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 29, 2026, 9:50 AM EDT
Active, not recruiting Phase 2 Interventional

Efficacy and Safety of Gemlapodect (NOE-105) in Adults and Adolescents With Tourette Syndrome

NCT06315751
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Gemlapodect, Placebo
Drug
Lead sponsor
Noema Pharma AG
Industry
Eligibility
18 Years and older
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
31
States / cities
Sun City, Arizona • Bellflower, California • Glendale, California + 28 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 29, 2026, 9:50 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Bilirubin Binding Capacity to Assess Bilirubin Load in Preterm Infants

NCT02691156
View directory record Official record
Conditions
Bilirubin-induced Neurologic Dysfunction, Hyperbilirubinemia, Kernicterus, Infant, Premature, Infant, Low Birth Weight
Interventions
Bilirubin Binding Capacity, End-tidal Carbon Monoxide, Carboxyhemoglobin
Diagnostic Test
Lead sponsor
Stanford University
Other
Eligibility
24 Weeks to 34 Weeks
Enrollment
143 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 1, 2021 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

NCT05089656
View directory record Official record
Conditions
Type 2 Spinal Muscular Atrophy
Interventions
OAV101, Sham control
Genetic · Procedure
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
6
States / cities
Farmington, Connecticut • Chicago, Illinois • Strasburg, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 3 Interventional Results available

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

NCT03746522
View directory record Official record
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2023 · Synced Oct 29, 2026, 9:50 AM EDT
Completed No phase listed Observational Results available

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

NCT02468570
View directory record Official record
Conditions
Phenylketonuria
Interventions
Administration of CANTAB and Subject Global Assessment
Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Lexington, Kentucky • Omaha, Nebraska • Oklahoma City, Oklahoma + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2020 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Not applicable Interventional

Anger Control Training for Youth With Tourette Syndrome

NCT00486551
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder, Oppositional Defiant Disorder, ADHD
Interventions
Anger control training
Behavioral
Lead sponsor
Yale University
Other
Eligibility
11 Years to 16 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 11, 2022 · Synced Oct 29, 2026, 9:50 AM EDT
Recruiting Phase 2 Interventional

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)

NCT04374305
View directory record Official record
Conditions
Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma, Ependymoma
Interventions
Brigatinib, Neratinib, Retifanlimab, Bevacizumab
Drug
Lead sponsor
Scott R. Plotkin, MD, PhD
Other
Eligibility
12 Years and older
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
6
States / cities
Los Angeles, California • Miami, Florida • Indianopolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 29, 2026, 9:50 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

NCT02420379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Oct 29, 2026, 9:50 AM 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 29, 2026, 9:50 AM EDT