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ClinicalTrials.gov public records Last synced May 21, 2026, 7:22 PM EDT

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

Showing 1–24 of 1,503 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Brain Metabolic Disorder Clear all
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Diabetes, Obesity
Interventions
EXERCISE
Behavioral
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 45 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 25, 2025 · Synced May 21, 2026, 7:22 PM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, SoC Therapy
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2024 · Synced May 21, 2026, 7:22 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg, zagociguat 30mg, Placebo
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 21, 2026, 7:22 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Alzheimer Disease, Obesity, Metabolic Disease
Interventions
Not listed
Lead sponsor
Cyrus A Raji
Other
Eligibility
40 Years to 60 Years
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 10, 2026 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
Interventions
15 O Water
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
123 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 18, 2013 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D Drug Product (eli-cel)
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced May 21, 2026, 7:22 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Kuvan
Drug
Lead sponsor
Dr. Linda Randolph
Other
Eligibility
4 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 19, 2016 · Synced May 21, 2026, 7:22 PM EDT
Terminated Phase 3 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced May 21, 2026, 7:22 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
exercise training
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
21 Years to 70 Years · Female only
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
Standard of Care
Other
Lead sponsor
Takeda
Industry
Eligibility
Up to 5 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Feeding Behavior, Brain, Adolescent
Interventions
Energy Stimulus
Dietary Supplement
Lead sponsor
University of Colorado, Denver
Other
Eligibility
13 Years to 18 Years
Enrollment
63 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 7, 2025 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Wilson Disease
Interventions
Bis-Choline Tetrathiomolybdate
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Sacramento, California • Ann Arbor, Michigan • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 17, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease
Interventions
Velaglucerase alfa, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Shire
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2022 · Synced May 21, 2026, 7:22 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
Interventions
Sodium Phenylbutyrate, Arginine
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced May 21, 2026, 7:22 PM EDT
Approved For Marketing No phase listed Expanded access
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase Alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
U.S. locations
11
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2024 · Synced May 21, 2026, 7:22 PM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Ataxia Telangiectasia, Genetic Syndrome
Interventions
EryDex System
Combination Product
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, DT-216 matching Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced May 21, 2026, 7:22 PM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 21, 2026, 7:22 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Pseudohypoparathyroidism Type 1A, Albright Hereditary Osteodystrophy, Pseudopseudohypoparathyroidism
Interventions
Neurocognitive and psychosocial testing
Behavioral
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
2 Months to 89 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2030
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated May 27, 2025 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Alzheimer Disease, Parkinson Disease, Frontotemporal Lobar Degeneration
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
35 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 21, 2019 · Synced May 21, 2026, 7:22 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Wilson Disease
Interventions
UX701, Standard of Care (SOC)
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
11
States / cities
Los Angeles, California • Redwood City, California • Sacramento, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced May 21, 2026, 7:22 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced May 21, 2026, 7:22 PM EDT