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Showing 1–24 of 1,512 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Brain Metabolic Disorder Clear all
Recruiting No phase listed Observational

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT05813678
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2033
U.S. locations
12
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease

NCT05085704
View directory record Official record
Conditions
Glut1 Deficiency Syndrome 1, Glucose Metabolism Disorders, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome, Glucose Transport Defect
Interventions
Magnetic resonance imaging
Device
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
16 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2029
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Terminated Phase 2 Interventional Results available

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

NCT03759678
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Completed Not applicable Interventional

Effect of a Medium Chain Triglyceride Supplemented Diet on Cognitive Function and Brain Activation in Type 1 Diabetes.

NCT01315171
View directory record Official record
Conditions
Hypo-unawareness, Type 1 Diabetes Mellitus
Interventions
Medium chain triglycerides
Dietary Supplement
Lead sponsor
Yale University
Other
Eligibility
18 Years to 55 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 16, 2018 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting Early Phase 1 Interventional Accepts healthy volunteers

Study of Biodistribution, Metabolism, Excretion and Brain Uptake of 11C-HY-2-15

NCT06032026
View directory record Official record
Conditions
Tauopathies
Interventions
11C-HY-2-15 PET, brain MRI, Amyloid PET scan, Neurological assessments
Drug · Diagnostic Test · Behavioral
Lead sponsor
University of Pennsylvania
Other
Eligibility
40 Years to 80 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Jul 21, 2026, 11:24 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Imaging of Brain mGluR1 Receptors Using [18F]FIMX

NCT02230592
View directory record Official record
Conditions
Anxiety Disorder, Major Depressive Disorder
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years to 55 Years
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 4, 2018 · Synced Jul 21, 2026, 11:24 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting Phase 2 Interventional

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

NCT06447025
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 60 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Terminated Phase 1Phase 2 Interventional

A Fabry Disease Gene Therapy Study

NCT04040049
View directory record Official record
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2023 · Synced Jul 21, 2026, 11:24 AM EDT
Terminated Phase 4 Interventional

Quality of Life and Nutritional Improvements in Cirrhotic Patients

NCT01842113
View directory record Official record
Conditions
Liver Cirrhosis, Hepatic Encephalopathy, Portal Hypertension
Interventions
Rifaximin, Lactulose, Lactulose Placebo, Rifaximin Placebo
Drug
Lead sponsor
Tampa General Hospital
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Sep 22, 2013 · Synced Jul 21, 2026, 11:24 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Discrimination and the Brain-Gut-Microbiome (BGM) Axis

NCT05120908
View directory record Official record
Conditions
Discrimination, Racial, Social Isolation, Stress
Interventions
Not listed
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
266 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 9, 2025 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria

NCT01659749
View directory record Official record
Conditions
Pregnancy, Phenylketonuria
Interventions
Metabolic camp
Behavioral
Lead sponsor
Emory University
Other
Eligibility
11 Years and older · Female only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2037
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Completed Not applicable Interventional

Trial of Aerobic Exercise Training in Stroke Survivors

NCT00891514
View directory record Official record
Conditions
Stroke, Insulin Resistance, Sedentary Lifestyle
Interventions
Aerobic Exercise, Stretching
Behavioral
Lead sponsor
Baltimore VA Medical Center
Federal
Eligibility
40 Years to 75 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 10, 2018 · Synced Jul 21, 2026, 11:24 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype and Biomarkers 2

NCT04875416
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Dementia
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
7 Years and older
Enrollment
217 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2031
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting No phase listed Observational

Language in Primary Progressive Aphasia

NCT00537004
View directory record Official record
Conditions
Primary Progressive Aphasia
Interventions
Not listed
Lead sponsor
Northwestern University
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Clinical Outcome Assessment for AT & BCI

NCT07407725
View directory record Official record
Conditions
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
Interventions
Eye Tracker, Mouth Operated Joystick, Non-invasive electroencephalogram (EEG) Headset, Implantable Brain-Computer-Interface, Personal Assistive Technology, Voice Control
Device
Lead sponsor
Shirley Ryan AbilityLab
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study - Mitochondrial Disease

NCT01532791
View directory record Official record
Conditions
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
4 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Completed Phase 2 Interventional

SNIFF 120: Study of Nasal Insulin to Fight Forgetfulness (120 Days)

NCT00438568
View directory record Official record
Conditions
Mild Cognitive Impairment, Alzheimer's Disease
Interventions
Regular Insulin, Placebo
Drug
Lead sponsor
University of Washington
Other
Eligibility
55 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2011
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 13, 2012 · Synced Jul 21, 2026, 11:24 AM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Jul 21, 2026, 11:24 AM EDT
Enrolling by invitation Phase 2 Interventional

Sirolimus for Leigh Syndrome

NCT06843811
View directory record Official record
Conditions
Leigh Syndrome
Interventions
Sirolimus
Drug
Lead sponsor
Matthew Demczko
Other
Eligibility
6 Months to 55 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Completed Phase 2 Interventional Results available

Effectiveness of Growth Hormone Releasing Hormone in Reducing Abdominal Fat in People Who Are Obese

NCT00675506
View directory record Official record
Conditions
Abdominal Obesity, Growth Hormone Deficiency
Interventions
Growth hormone releasing hormone (GHRH) 1-44, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 12, 2017 · Synced Jul 21, 2026, 11:24 AM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171
View directory record Official record
Conditions
OTC Deficiency
Interventions
DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
Genetic · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Jul 21, 2026, 11:24 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Remote Neurocognitive and Psychological Assessment in PKU

NCT04943393
View directory record Official record
Conditions
Phenylketonurias
Interventions
nointervention
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 40 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 21, 2022 · Synced Jul 21, 2026, 11:24 AM EDT