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Showing 1–24 of 1,821 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: CNS Metabolic Disorders Clear all
Completed No phase listed Observational Results available

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

NCT00937794
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Neurobehavioral testing, Visual and auditory assessments
Behavioral · Other
Lead sponsor
Shire
Industry
Eligibility
32 Months to 18 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 7, 2026, 2:40 PM EDT
Not yet recruiting Not applicable Interventional

Endoscopic Shunts Embolization for Refractory Hepatic Encephalopathy

NCT07553858
View directory record Official record
Conditions
Hepatic Encephalopathy, Liver Cirrhosis, Portal Shunt Systemic
Interventions
Endoscopic ultrasound-guided transgastric embolization of the spontaneous portosystemic shunts, Interventional Radiology for shunt embolization/retrograde tranvenous obliteration
Procedure
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Sep 7, 2026, 2:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Risk Factors, Costs, and Impacts of ED Boarding

NCT07532564
View directory record Official record
Conditions
Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease), Sepsis, Hypertension, Heart Failure, Pneumonia, Urinary Tract Infection (Diagnosis), Chest Pain, Psychiatric Disorder, Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
30,486 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Sep 7, 2026, 2:40 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Quantitative Assessment of Sucking for Early Diagnosis of Brain Injury in Infants at High Risk

NCT03246243
View directory record Official record
Conditions
Hypoxic-Ischemic Encephalopathy, Hypoglycemia, Neonatal Abstinence Syndrome
Interventions
nfant feeding solution
Device
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 6 Months
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 20, 2021 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 3 Interventional

A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Tolerability of Oral Lixivaptan Capsules in Subjects With Euvolemic Hyponatremia

NCT00876798
View directory record Official record
Conditions
Euvolemic Hyponatremia
Interventions
Lixivaptan, Placebo
Drug
Lead sponsor
CardioKine Inc.
Industry
Eligibility
18 Years and older
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
66
States / cities
Birmingham, Alabama • Montgomery, Alabama • Phoenix, Arizona + 54 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2011 · Synced Sep 7, 2026, 2:40 PM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 3 Interventional

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

NCT00225615
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
14
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2009 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

NCT00992459
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, Buphenyl (NaPBA)
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
21
States / cities
Long Beach, California • Los Angeles, California • Stanford, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2024 · Synced Sep 7, 2026, 2:40 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 17, 2025 · Synced Sep 7, 2026, 2:40 PM EDT
Terminated Phase 2 Interventional Results available

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

NCT00004418
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
glyceryl trierucate/glyceryl trioleate
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Months to 8 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2014
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2020 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 2 Interventional Results available

A Trial of Tocilizumab in ALS Subjects

NCT02469896
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Motor Neuron Disease
Interventions
Tocilizumab, Placebo
Drug · Other
Lead sponsor
Barrow Neurological Institute
Other
Eligibility
18 Years to 75 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
5
States / cities
Phoenix, Arizona • Kansas City, Kansas • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Not applicable Interventional

Obesity/Overweight in Persons With Early and Chronic Spinal Cord Injury (SCI)

NCT01467817
View directory record Official record
Conditions
Overweight, Obese, Spinal Cord Injury
Interventions
Lifestyle Intervention, Exercise Control
Behavioral
Lead sponsor
University of Miami
Other
Eligibility
18 Years to 65 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Nov 29, 2016 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 1 Interventional

1-Octanol to Treat Essential Tremor

NCT00016679
View directory record Official record
Conditions
Essential Tremor
Interventions
1-Octanol
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 7, 2026, 2:40 PM EDT
Recruiting Not applicable Interventional

Effect of Physiologic Insulin Administration on Insulin Sensitivity and Cognition

NCT07265323
View directory record Official record
Conditions
Alzheimer s Disease, Alzheimer Blood Biomarkers, Insulin Sensitivity
Interventions
Physiological Insulin Intervention
Other
Lead sponsor
Pennington Biomedical Research Center
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Baton Rouge, Louisiana
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A First in Human Study of ORT247 in Healthy Volunteers

NCT06769620
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
ORT247, Placebo
Drug
Lead sponsor
Orthogonal Neuroscience Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Sep 7, 2026, 2:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of ALS Reversals 2: Genetic Analyses

NCT03464903
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Sep 7, 2026, 2:40 PM EDT
Completed No phase listed Observational

The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study

NCT00071903
View directory record Official record
Conditions
Pseudotumor Cerebri, Cystinosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 50 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 7, 2026, 2:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Digital, Analyte and Neurologic Biomarkers of Acute and Chronic Brain Injury and Recovery in CQT Instructors

NCT05917665
View directory record Official record
Conditions
Traumatic Brain Injury
Interventions
Not listed
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Fort Bragg, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 23, 2026 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Not applicable Interventional

Nutritional Formulation for Angelman Syndrome

NCT03644693
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Nutritional Formulation Containing Exogenous Ketones, Placebo Formulation
Dietary Supplement
Lead sponsor
University of Colorado, Denver
Other
Eligibility
4 Years to 11 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Aug 19, 2020 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Phase 1 Interventional

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

NCT01005004
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
HuCNS-SC cells
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 5 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 14, 2015 · Synced Sep 7, 2026, 2:40 PM EDT
Completed Not applicable Interventional

Clinical Decision Support for Medication Management and Adherence

NCT00979225
View directory record Official record
Conditions
Hypertension, Diabetes Mellitus, Stroke, Myocardial Ischemia, Heart Failure, Asthma
Interventions
Medication Management report, Care manager email notices
Other
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 10, 2012 · Synced Sep 7, 2026, 2:40 PM EDT
Not listed Phase 1Phase 2 Interventional

Determining the Safety of L-serine in ALS

NCT01835782
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
L-Serine
Drug
Lead sponsor
Phoenix Neurological Associates, LTD
Other
Eligibility
18 Years to 85 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
2
States / cities
Phoenix, Arizona • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 29, 2015 · Synced Sep 7, 2026, 2:40 PM EDT
Recruiting Phase 1 Interventional

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355
View directory record Official record
Conditions
Friedreich's Ataxia (FA)
Interventions
SGT-212
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 7, 2026, 2:40 PM EDT
Recruiting Phase 2 Interventional

Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis

NCT07023835
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
50 mg Usnoflast, 75 mg Usnoflast, Placebo
Drug
Lead sponsor
Zydus Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
15
States / cities
La Jolla, California • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 7, 2026, 2:40 PM EDT