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ClinicalTrials.gov public records Last synced Oct 30, 2026, 3:59 PM EDT

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Showing 745–768 of 2,426 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation

NCT04199000
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
12
States / cities
San Diego, California • Aurora, Colorado • Jacksonville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 3 Interventional

Tigulixostat, Phase 3 Study, Placebo Controlled in Gout Patients

NCT05586958
View directory record Official record
Conditions
Gout, Hyperuricemia, Gout Flare, Tophi
Interventions
Tigulixostat, Placebo
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 85 Years
Enrollment
354 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
36
States / cities
Birmingham, Alabama • Covina, California • Clearwater, Florida + 31 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 30, 2026, 3:59 PM EDT
Recruiting No phase listed Observational

A Retrospective Study to Characterize Participants With Propionic Acidemia

NCT05769621
View directory record Official record
Conditions
Propionic Acidemia
Interventions
Not listed
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
2 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
6
States / cities
Stanford, California • Baltimore, Maryland • Ann Arbor, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 2 Interventional

Study of Tranilast Alone or in Combination With Allopurinol in Subjects With Hyperuricemia

NCT01052987
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Tranilast, Allopurinol, Combination - Tranilast and Allopurinol, 400 mg Allopurinol, High dose combination
Drug
Lead sponsor
Nuon Therapeutics, Inc.
Industry
Eligibility
21 Years to 70 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
3
States / cities
Honolulu, Hawaii • Evansville, Indiana • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 6, 2011 · Synced Oct 30, 2026, 3:59 PM EDT
Recruiting No phase listed Observational

Vascular Lab Resource (VLR) Biorepository

NCT05628948
View directory record Official record
Conditions
Cardiovascular Diseases, Metabolic Disease, Peripheral Artery Disease, Carotid Disease, Aneurysmal Disease, Venous Disease, Thrombosis, Lymphedema, Lipedema, Non-Atherosclerotic Chronic Arterial Occlusive Disease, Vasculitis, Fibromuscular Dysplasia, Arterial Dissection, May-Thurner Syndrome, Thoracic Outlet Syndrome, Vasospasm
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
500 participants
Timeline
2019 – 2031
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 19, 2025 · Synced Oct 30, 2026, 3:59 PM EDT
Withdrawn Phase 4 Interventional

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

NCT05494593
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions
ELAPRASE, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Drug
Lead sponsor
Takeda
Industry
Eligibility
Up to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
8
States / cities
Phoenix, Arizona • Oakland, California • San Diego, California + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2025 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Not applicable Interventional

Using D-Galactose as a Food Supplement in Congenital Disorders of Glycosylation

NCT02955264
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
D-Galactose
Dietary Supplement
Lead sponsor
Tulane University
Other
Eligibility
Up to 21 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Jan 8, 2021 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia

NCT04659863
View directory record Official record
Conditions
Familial Hypercholesterolemia - Homozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boca Raton, Florida
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 30, 2026, 3:59 PM EDT
Withdrawn Phase 4 Interventional

Clinical Trial to Determine the Efficacy of Sculptra™ Dermal Filler for the Correction of Contour Deformities Caused by Lipoatrophy

NCT00360139
View directory record Official record
Conditions
Lipodystrophy, Emaciation
Interventions
Sculptra Injection
Device
Lead sponsor
Dermatology Cosmetic Laser Medical Associates of La Jolla, Inc.
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 2008
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Aug 21, 2007 · Synced Oct 30, 2026, 3:59 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Instrumented Data Exchange for Ataxia Study

NCT04268147
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Friedreich Ataxia
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
12 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2024 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

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

NCT01594853
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 29, 2018 · Synced Oct 30, 2026, 3:59 PM EDT
Terminated Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim

NCT01182675
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 3 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 31, 2018 · Synced Oct 30, 2026, 3:59 PM EDT
Completed No phase listed Observational

Liver Fibrosis in Sickle Cell Disease

NCT02007746
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
Other · Procedure
Lead sponsor
University of Miami
Other
Eligibility
10 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Oct 30, 2026, 3:59 PM EDT
Completed No phase listed Observational

A Natural History Study of Canavan Disease

NCT02851563
View directory record Official record
Conditions
Canavan Disease
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
Up to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 30, 2026, 3:59 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Integrated Cancer Repository for Cancer Research

NCT02012699
View directory record Official record
Conditions
Pancreatic Cancer, Thyroid Cancer, Lung Cancer, Esophageal Cancer, Thymus Cancer, Colon Cancer, Rectal Cancer, Gastrointestinal Stromal Tumors, Anal Cancer, Bile Duct Cancer, Duodenal Cancer, Gallbladder Cancer, Gastric Cancer, Liver Cancer, Small Intestine Cancer, Peritoneal Surface Malignancies, Familial Adenomatous Polyposis, Lynch Syndrome, Bladder Cancer, Kidney Cancer, Penile Cancer, Prostate Cancer, Testicular Cancer, Ureter Cancer, Urethral Cancer, Hypopharyngeal Cancer, Laryngeal Cancer, Lip Cancer, Oral Cavity Cancer, Nasopharyngeal Cancer, Oropharyngeal Cancer, Paranasal Sinus Cancer, Nasal Cavity Cancer, Salivary Gland Cancer, Skin Cancer, Central Nervous System Tumor, Central Nervous System Cancer, Mesothelioma, Breast Cancer, Leukemia, Melanoma, Sarcoma, Unknown Primary Tumor, Multiple Myeloma, Ovarian Cancer, Endometrial Cancer, Vaginal Cancer, Neuroendocrine Tumors, Plasma Cell Dyscrasia, Healthy Control
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
19 Years to 110 Years
Enrollment
999,999 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2099
U.S. locations
42
States / cities
Greenwood Village, Colorado • Manchester, Connecticut • Daytona Beach, Florida + 34 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 30, 2026, 3:59 PM EDT
Completed No phase listed Observational

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT00515398
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Observation
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
4
States / cities
Gainesville, Florida • St Louis, Missouri • Lake Success, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2008 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Not applicable Interventional

Simplified Diet Approach in Phenylketonuria

NCT02555579
View directory record Official record
Conditions
Phenylketonuria
Interventions
Dietary Protein Counting and Free Fruits and Vegetables
Behavioral
Lead sponsor
University of Nebraska
Other
Eligibility
13 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Oct 30, 2026, 3:59 PM EDT
Recruiting Phase 2 Interventional

Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin in Werner Syndrome

NCT05847179
View directory record Official record
Conditions
Werner Syndrome
Interventions
Progerinin
Drug
Lead sponsor
PRG Science & Technology Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 30, 2026, 3:59 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

NCT07530796
View directory record Official record
Conditions
Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
Interventions
scAAV9/AGA
Genetic
Lead sponsor
Rare Trait Hope
Other
Eligibility
4 Years to 45 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Shreveport, Louisiana
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 2Phase 3 Interventional Results available

TBI Dose De-escalation for Fanconi Anemia

NCT00352976
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant w/Laronidase for Hurler

NCT00176891
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler Syndrome
Interventions
Stem Cell Transplant, Laronidase ERT
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 7 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 4 Interventional Results available

Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

NCT00028262
View directory record Official record
Conditions
Infantile Neronal Ceroid Lipofuscinosis
Interventions
Cystagon
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 3 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2016 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy Study of PEG-uricase in the Treatment of Hyperuricemic Patients With Symptomatic Gout

NCT00325195
View directory record Official record
Conditions
Gout
Interventions
placebo, pegloticase
Other · Biological
Lead sponsor
Savient Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
50
States / cities
Birmingham, Alabama • Tucson, Arizona • Jonesboro, Arkansas + 45 more
Source: ClinicalTrials.gov public record
Updated Feb 28, 2011 · Synced Oct 30, 2026, 3:59 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

NCT01390077
View directory record Official record
Conditions
Alkaptonuria
Interventions
Nitisinone
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced Oct 30, 2026, 3:59 PM EDT