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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2Phase 3 Interventional

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

NCT00059280
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced Sep 27, 2026, 1:59 AM EDT
Terminated Phase 1 Interventional Results available

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

NCT02574910
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Abiraterone acetate
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
2 Years to 9 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
4
States / cities
Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Phase 3 Interventional Results available

Open-Label Lesinurad Monotherapy Extension Study in Gout

NCT01650246
View directory record Official record
Conditions
Gout
Interventions
lesinurad
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
143 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
57
States / cities
Birmingham, Alabama • Glendale, Arizona • Peoria, Arizona + 51 more
Source: ClinicalTrials.gov public record
Updated May 25, 2016 · Synced Sep 27, 2026, 1:59 AM EDT
Active, not recruiting Phase 1 Interventional

Study for the Treatment for CLN7 Disease

NCT04737460
View directory record Official record
Conditions
CLN7
Interventions
AAV9/CLN7
Genetic
Lead sponsor
Benjamin Greenberg
Other
Eligibility
1 Year to 18 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 30, 2025 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Not applicable Interventional

Pharmacokinetics and Pharmacodynamics of Anthocyanins

NCT03650140
View directory record Official record
Conditions
Gout
Interventions
Tart cherry extract 60 mL, Tart cherry extract 120 mL
Dietary Supplement
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 8, 2023 · Synced Sep 27, 2026, 1:59 AM EDT
Completed No phase listed Observational

A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia

NCT04255680
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Buccal Swabs and Blood Draws
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
20 participants
Timeline
2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 17, 2020 · Synced Sep 27, 2026, 1:59 AM EDT
Recruiting No phase listed Observational

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

NCT06305234
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2033
U.S. locations
11
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Sep 27, 2026, 1:59 AM EDT
Completed No phase listed Observational Results available

Chronic Liver Disease in Urea Cycle Disorders

NCT03721367
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Diagnostic Ultrasound
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
5 Years to 60 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 9, 2023 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)

NCT01831934
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Fluzone®
Biological
Lead sponsor
Stanford University
Other
Eligibility
13 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Not applicable Interventional Results available

SPECS: Safe Pediatric Euglycemia in Cardiac Surgery

NCT00443599
View directory record Official record
Conditions
Heart Defects, Congenital, Hyperglycemia
Interventions
Insulin, Usual Care
Drug · Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 36 Months
Enrollment
989 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 24, 2022 · Synced Sep 27, 2026, 1:59 AM 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 26, 2016 · Synced Sep 27, 2026, 1:59 AM EDT
Recruiting No phase listed Observational

A Long-term Follow-up Study of Gaucher Disease

NCT03190837
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
200 participants
Timeline
2017 – 2028
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 27, 2026, 1:59 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
View directory record Official record
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Phase 3 Interventional Results available

A Study of the Efficacy and Tolerability of Pancrelipase Microtablet (MT) Capsules for the Treatment of Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency

NCT00662675
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency, Steatorrhea, Malabsorption Syndromes, Cystic Fibrosis
Interventions
Pancrease MT 10.5, or MT 21, Placebo for Pancrease MT 10.5 or MT 21
Drug
Lead sponsor
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Industry
Eligibility
7 Years to 60 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
10
States / cities
Long Beach, California • Los Angeles, California • Orlando, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated May 8, 2014 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Phase 2 Interventional Results available

A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

NCT00879034
View directory record Official record
Conditions
Progeria, Hutchinson-Gilford Syndrome
Interventions
Lonafarnib, Zoledronic Acid, Pravastatin
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 12, 2019 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Influence of Endothelial Function on Central and Peripheral Causes Of Exercise Impairment in Type 2 Diabetes

NCT00786019
View directory record Official record
Conditions
Type 2 Diabetes
Interventions
Exercise program, Ascorbic Acid (Vitamin C)
Behavioral · Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
30 Years to 55 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2011
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 11, 2023 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Phase 1 Interventional

A Study of KRN23 in X-linked Hypophosphatemia

NCT00830674
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
Placebo, KRN23
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
4
States / cities
New Haven, Connecticut • Indianapolis, Indiana • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2024 · Synced Sep 27, 2026, 1:59 AM EDT
Withdrawn Phase 2 Interventional

Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome

NCT00514982
View directory record Official record
Conditions
Hermanski-Pudlak Syndrome, Colitis, Cytokines, Lymphocytes, Drug Evaluation
Interventions
Mesalamine, Infliximab, Corticosteroids, 6-Mercaptopurine, Tacrolimus, Adalimumab
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 27, 2026, 1:59 AM EDT
Not listed No phase listed Observational

Clinical Specimen Collection From Pompe Disease Patients

NCT04476550
View directory record Official record
Conditions
Pompe Disease
Interventions
Filgrastim
Drug
Lead sponsor
Serhat Gumrukcu, MD PhD
Other
Eligibility
3 Years to 30 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Toluca Lake, California
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Apolipoprotein CIII Reduction Via Colchicine

NCT02083510
View directory record Official record
Conditions
Hypertriglyceridemia, Gout, Pericarditis
Interventions
Colchicine
Drug
Lead sponsor
Scripps Translational Science Institute
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated May 29, 2014 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of GIMate Handheld Hydrogen Breath Monitor for Diagnosis of Lactose Malabsorption

NCT04754724
View directory record Official record
Conditions
Lactose Intolerance
Interventions
GIMate, H2 Check
Diagnostic Test
Lead sponsor
Vivante Health
Industry
Eligibility
18 Years to 55 Years
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 27, 2021 · Synced Sep 27, 2026, 1:59 AM EDT
Completed Phase 3 Interventional

Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia

NCT00099996
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 27, 2026, 1:59 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Role of Body Fat Distribution in Metabolic and Pulmonary Decline in Cystic Fibrosis (ORBIT-CF)

NCT04002882
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
2
States / cities
Birmingham, Alabama • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 17, 2026 · Synced Sep 27, 2026, 1:59 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Allergy and Immunology Natural History Study

NCT05584488
View directory record Official record
Conditions
Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Up to 99 Years
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Sep 27, 2026, 1:59 AM EDT