Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 18, 2026, 9:34 AM EDT

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Showing 1057–1080 of 1,605 matching trials from the live ClinicalTrials.gov search.
Not listed No phase listed Observational Accepts healthy volunteers

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

NCT02935283
Conditions
Urea Cycle Disorders
Interventions
MRI, Behavioral
Procedure · Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Gout
Interventions
RDEA3170 10 mg, allopurinol 300 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Overland Park, Kansas
Approved For Marketing No phase listed Expanded access

Copper Histidinate Treatment for Menkes Disease

NCT04074512
Conditions
Menkes Disease
Interventions
copper histidinate
Drug
Lead sponsor
Sentynl Therapeutics, Inc.
Industry
Eligibility
0 Years to 6 Years
U.S. locations
20
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 17 more
Completed Not applicable Interventional Accepts healthy volunteers

Habitual Diet and Avocado Trial

NCT03528031
Conditions
Intra-abdominal Fat, Metabolic Syndrome, High Cholesterol, Triglycerides High, Diet Habit, High Blood Sugar, Liver Fat, Dietary Modification, HDL Cholesterol, Low Serum, Cardiovascular Diseases, High Density Lipoprotein Deficiency, Low-density-lipoprotein-type, Cardiovascular Risk Factor, Diabetes
Interventions
Intervention Daily Avocado
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
25 Years and older
Enrollment
1,008 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
4
States / cities
Loma Linda, California • Los Angeles, California • Boston, Massachusetts + 1 more
Recruiting Not applicable Interventional

The ORIGIN-FH Study

NCT07470723
Conditions
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
Interventions
Screening for FH
Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Madison, Wisconsin
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Conditions
MPS-IH (Hurler Syndrome)
Interventions
Experimental: OTL-203, Active Comparator: Allo-HSCT
Genetic
Lead sponsor
Orchard Therapeutics
Industry
Eligibility
28 Days to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Developmental Bone Disease, Dwarfism, Skeletal Dysplasias
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Healthy
Interventions
Nikkomycin Z
Drug
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Tucson, Arizona
Completed Not applicable Interventional Accepts healthy volunteers

Resistance Exercise in Barth Syndrome

NCT01629459
Conditions
Barth Syndrome
Interventions
Resistance exercise and protein supplementation
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 35 Years · Male only
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2019
U.S. locations
1
States / cities
St Louis, Missouri
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Hypophosphatasia (HPP)
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 36 Months
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
6
States / cities
Little Rock, Arkansas • Wilmington, Delaware • Springfield, Missouri + 3 more
Conditions
Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions
Observational study
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Completed Not applicable Interventional Accepts healthy volunteers Results available

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

NCT01594853
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
Conditions
Hyperoxaluria, Primary
Interventions
Oxabact OC5 - Oxalobacter formigenes Strain HC-1
Biological
Lead sponsor
OxThera
Industry
Eligibility
3 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Conditions
Fabry Disease
Interventions
Lucerastat, Placebo
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 14 more
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
19
States / cities
Berkeley, California • Los Angeles, California • San Diego, California + 12 more
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
Interventions
BMN 190
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 15 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Sjogren-Larsson Syndrome
Interventions
ADX-629
Drug
Lead sponsor
University of Nebraska
Other
Eligibility
5 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Omaha, Nebraska
Completed Phase 1 Interventional Results available

Mild, Moderate and Severe Renal Impairment Study

NCT02219516
Conditions
Gout
Interventions
RDEA3170
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years · Male only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
4
States / cities
Lakewood, Colorado • Orlando, Florida • Minneapolis, Minnesota + 1 more
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Dichloroacetate (DCA), Placebo, Genotype
Drug · Other · Genetic
Lead sponsor
Saol Therapeutics Inc
Industry
Eligibility
6 Months to 17 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
10
States / cities
Orange, California • Stanford, California • Washington D.C., District of Columbia + 7 more
Conditions
Fabry Disease
Interventions
Agalsidase alfa
Drug
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2011
U.S. locations
13
States / cities
Tucson, Arizona • Palm Beach Gardens, Florida • Lake Charles, Louisiana + 9 more
Conditions
Phenylketonuria
Interventions
Sepiapterin
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 9 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Ornithine Transcarbamylase Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Orlando, Florida