Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 14, 2026, 6:25 AM EDT

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Showing 145–168 of 1,608 matching trials from the live ClinicalTrials.gov search.
Conditions
Urea Cycle Disorder
Interventions
KB195
Drug
Lead sponsor
Kaleido Biosciences
Industry
Eligibility
18 Years to 65 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Baltimore, Maryland + 3 more
Conditions
Generalized Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
Birmingham, Alabama • New Orleans, Louisiana • Ann Arbor, Michigan + 4 more
Conditions
Propionic Acidemia
Interventions
ornithine alpha ketoglutarate, glutamine, disodium citrate
Drug
Lead sponsor
Nicola Longo
Other
Eligibility
5 Years to 12 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Salt Lake City, Utah
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
Lactose Intolerance
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
18 Years to 75 Years
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Achondroplasia
Interventions
Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
0 Years to 32 Months
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Oakland, California • Baltimore, Maryland • Madison, Wisconsin
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Takeda
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
7
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
Conditions
Maculopathy, Age-Related, Choroidal Neovascularization
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
50 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
4
States / cities
Beverly Hills, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Conditions
PHP Ia, PHP IB, Php1C
Interventions
Theophylline
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years to 21 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
CAH - Congenital Adrenal Hyperplasia
Interventions
NBI-74788
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 50 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
6
States / cities
Aurora, Colorado • Indianapolis, Indiana • Ann Arbor, Michigan + 3 more
Conditions
Pyruvate Dehydrogenase Complex Deficiency Disease
Interventions
No intervention
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Terminated No phase listed Observational Accepts healthy volunteers

The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity

NCT01421888
Conditions
Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency, Citrullinemia, Ornithine Carbamoyltransferase Deficiency, Hyperargininemia, N-Acetylglutamate Synthase Deficiency
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Heart Diseases, Metabolism, Inborn Errors
Interventions
SCH-58235, Ezetimibe
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Suspended No phase listed Observational Accepts healthy volunteers

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease

NCT04031469
Conditions
Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder, Autism Spectrum Disorder High-Functioning, Alzheimer Disease, Alzheimer Dementia (AD), Alzheimer Dementia, Alzheimer Disease (AD), Crohn, Crohn Colitis, Crohn Disease (CD), Crohn Disease Colon, Myalgic Encephalomyelitis, ME/CFS, Myalgic Encephalomyelitis (ME), Myalgic Encephalomyelitis/Chronic Fatigue Syndrome, Psoriasis, Psoriasis Annularis, Psoriasis Chronic, Chronic Urinary Tract Infection, Ulcerative Colitis (Disorder), Ulcerative Colitis (UC), Ulcerative Colitis Acute, Ulcerative Colitis Chronic, Multiple Sclerosis, Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis (MS) Primary Progressive, Constipation Chronic Idiopathic, Constipation, Celiac, Celiac Disease, Celiac Sprue, Lyme Arthritis, Lyme Borreliosis, Nervous System, Lyme Disease, Chronic, Cholesterol, Cholesterol Level, High, Cancer, Colon Cancer, Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis, Rheumatoid Arthritis (RA), Rheumatoid Arthritis - Rheumatism, Chronic Fatigue Syndrome (CFS), PARKINSON DISEASE (Disorder), Parkinson Disease, Parkinson Disease (PD), Depression, Major Depressive Disorder (MDD), Depression Disorder, Depression in Adults, Anxiety, Anxiety Disorder Generalized, Obsessive Compulsive Disorder (OCD), Obsessive Compulsive Disorder OCD, Obsessive - Compulsive Disorder, Bipolar, Bipolar 1 Disorder, Bipolar Disorder (BD), Bipolar Disorder I and II, Bipolar and Related Disorders, Migraine, Migraine Disorder, Diabetes (DM), Diabetes, Lupus, Lupus Erythematosus, Epidermolysis Bullosa (EB), Mesothelioma, Mesothelioma Malignant, IBS (Irritable Bowel Syndrome), Irritable Bowel, IBS - Irritable Bowel Syndrome, Irritable Bowel Syndrome (IBS), Eczema, Eczema Atopic Dermatitis, Acne, Myasthaenia Gravis, Gout
Interventions
There is no intervention in this study
Other
Lead sponsor
ProgenaBiome
Other
Eligibility
12 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2030
U.S. locations
1
States / cities
Ventura, California
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
0 Years to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
Not yet recruiting No phase listed Observational Accepts healthy volunteers

Development of Healthcare Transition for Patients With Congenital Adrenal Hyperplasia

NCT07611786
Conditions
Congenital Adrenal Hyperplasia
Interventions
exposure to healthcare transition protocol
Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
16 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
Gout, Hyperuricemia
Interventions
dotinurad, dotinurad + allopurinol, allopurinol
Drug
Lead sponsor
Urica Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
4
States / cities
Clearwater, Florida • Miami Lakes, Florida • Mesquite, Texas + 1 more
Conditions
GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
AXO-AAV-GM2
Drug
Lead sponsor
Terence Flotte
Other
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Worcester, Massachusetts
Conditions
ARG1 Deficiency
Interventions
Loargys Arginine Test System
Device
Lead sponsor
Immedica Pharma US Inc
Industry
Eligibility
0 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1993
U.S. locations
6
States / cities
Los Angeles, California • San Francisco, California • St Louis, Missouri + 3 more
Conditions
GM1 Gangliosidosis
Interventions
LYS-GM101
Genetic
Lead sponsor
LYSOGENE
Industry
Eligibility
Up to 3 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Orange, California
Conditions
Acute Gout
Interventions
Canakinumab, Triamcinolone acetonide
Drug
Lead sponsor
Novartis
Industry
Eligibility
18 Years to 80 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
27
States / cities
Anniston, Alabama • Birmingham, Alabama • Buena Park, California + 23 more
Conditions
Phenylketonuria
Interventions
PTC923
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
247 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Gainesville, Florida • Indianapolis, Indiana + 4 more
Conditions
Gout
Interventions
placebo, BCX4208
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
18 Years to 69 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
24
States / cities
Scottsdale, Arizona • Tucson, Arizona • Irvine, California + 21 more