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ClinicalTrials.gov public records Last synced Oct 28, 2026, 4:07 PM EDT

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Showing 481–504 of 2,423 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Completed Phase 2 Interventional Results available

Safety and Effect of Doxycycline in Patients With Amyloidosis

NCT01677286
View directory record Official record
Conditions
Amyloidosis
Interventions
Doxycycline 100 mg po bid x 12 months
Drug
Lead sponsor
Boston University
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 1, 2017 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 2 Interventional Results available

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

NCT01573754
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Hydroxychloroquine, Phlebotomy
Drug · Procedure
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2021
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 17, 2023 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Taste Evaluation of Different Liquid Formulations With Eliglustat

NCT02422654
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 55 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated May 27, 2015 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 4 Interventional Results available

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
9
States / cities
Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Not applicable Interventional

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

NCT00002273
View directory record Official record
Conditions
Colitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
21 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 3 Interventional Results available

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

NCT00694109
View directory record Official record
Conditions
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
Interventions
Mipomersen Sodium
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
12 Years and older
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
20
States / cities
Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 28, 2026, 4:07 PM EDT
Terminated Early Phase 1 Interventional

Tislelizumab for the Treatment of Recurrent Mismatch Repair Deficient Endometrial Cancer

NCT04906382
View directory record Official record
Conditions
Lynch Syndrome, Recurrent Endometrial Carcinoma, Recurrent Endometrial Clear Cell Adenocarcinoma, Recurrent Uterine Corpus Carcinosarcoma, Mismatch Repair Deficiency, Recurrent Endometrial Cancer, Metastatic Endometrial Cancer
Interventions
Biopsy, Carboplatin, Paclitaxel, Tislelizumab
Procedure · Drug · Biological
Lead sponsor
Floor Backes, MD
Other
Eligibility
18 Years and older · Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 13, 2025 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout and Moderate Renal Impairment

NCT02128490
View directory record Official record
Conditions
Gout, Moderate Renal Impairment
Interventions
Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
189 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
89
States / cities
Birmingham, Alabama • Muscle Shoals, Alabama • Phoenix, Arizona + 75 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2016 · Synced Oct 28, 2026, 4:07 PM EDT
Recruiting No phase listed Observational

Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)

NCT05299710
View directory record Official record
Conditions
Cancers
Interventions
Laparoscopic surgery
Procedure
Lead sponsor
Erin Rowell
Other
Eligibility
Up to 11 Years · Female only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2035
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 2 Interventional Results available

An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism

NCT04538989
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
RZ358 Sequential Group Cohort 1, RZ358 Sequential Group Cohort 2, RZ358 Sequential Group Cohort 3, RZ358 Sequential Group Cohort 4
Drug
Lead sponsor
Rezolute
Other
Eligibility
2 Years to 45 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated May 28, 2025 · Synced Oct 28, 2026, 4:07 PM EDT
Terminated Phase 2 Interventional Results available

Amlexanox for Type 2 Diabetes and Obesity

NCT01842282
View directory record Official record
Conditions
Diabetes Mellitus Type 2, Non Alcoholic Fatty Liver Disease, Obesity
Interventions
Amlexanox
Drug
Lead sponsor
University of Michigan
Other
Eligibility
18 Years to 75 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Oct 28, 2026, 4:07 PM 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 4, 2008 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 3 Interventional Results available

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

NCT00007020
View directory record Official record
Conditions
Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
Interventions
Cholic Acids
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2009
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 28, 2026, 4:07 PM EDT
Active, not recruiting Not applicable Interventional

Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer

NCT00542373
View directory record Official record
Conditions
Erythroplakia, Fanconi Anemia, Lichen Planus, Oral Cavity and Lip Precancerous Condition, Oral Cavity Leukoplakia, Oral Neoplasm, Premalignant Lesion, Tobacco Use Disorder
Interventions
Cytology Specimen Collection Procedure, Diagnostic Microscopy, Diagnostic Procedure, Fluorescence Imaging, Fluorescence Spectroscopy
Other · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 28, 2026, 4:07 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

NCT05635266
View directory record Official record
Conditions
Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
Interventions
Specimen sample
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Waltham, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Not applicable Interventional Results available

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

NCT01773954
View directory record Official record
Conditions
Age-Related Macular Degeneration, Macular Degeneration
Interventions
Intravitreal Aflibercept Injection
Drug
Lead sponsor
MidAtlantic Retina
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 2, 2018 · Synced Oct 28, 2026, 4:07 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

NCT00344331
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 120 Years
Enrollment
900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 26, 2026 · Synced Oct 28, 2026, 4:07 PM EDT
Terminated Not applicable Interventional Results available

The Blood Pressure Effects of Febuxostat in Patients Previously Treated With Allopurinol: A Pilot Study

NCT01701622
View directory record Official record
Conditions
Blood Pressure, Gout
Interventions
febuxostat
Drug
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
18 Years to 80 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Jan 12, 2018 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Phase 4 Interventional Results available

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

NCT00701129
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Alglucosidase Alfa, Methotrexate, Rituximab
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 13, 2014 · Synced Oct 28, 2026, 4:07 PM EDT
Recruiting No phase listed Observational

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

NCT05825131
View directory record Official record
Conditions
Sanfilippo Syndrome Type C
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
12 Months and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 28, 2026, 4:07 PM EDT
Completed No phase listed Observational

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566
View directory record Official record
Conditions
Fabry Disease
Interventions
This is a non-interventional study
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 28, 2026, 4:07 PM EDT
Completed Not applicable Interventional

Music in Reducing Anxiety and Pain in Adult Patients Undergoing Bone Marrow Biopsy for Hematologic Cancers or Other Diseases

NCT00376922
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Psychosocial Effects of Cancer and Its Treatment
Interventions
music therapy
Procedure
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 120 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 28, 2026, 4:07 PM EDT
Recruiting Phase 2 Interventional

Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)

NCT07587021
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1, PH1
Interventions
YOLT-203, Placebo
Genetic · Drug
Lead sponsor
YolTech Therapeutics Co., Ltd
Industry
Eligibility
6 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Birmingham, Alabama • Aurora, Colorado • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 28, 2026, 4:07 PM EDT