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ClinicalTrials.gov public records Last synced Oct 29, 2026, 2:07 PM EDT

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Showing 577–600 of 2,425 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Completed Phase 1 Interventional

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

NCT04095273
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Oct 29, 2026, 2:07 PM EDT
Recruiting No phase listed Observational

Pancreas Registry and High Risk Registry

NCT02775461
View directory record Official record
Conditions
Pancreas Cancer, Pancreatitis, Chronic Pancreatitis, Pancreatic Cyst, Family History of Pancreas Cancer, Genetic Mutations
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years and older
Enrollment
1,368 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2033
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Phase 3 Interventional Results available

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
12 Years to 17 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
5
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Phase 3 Interventional Results available

Study of the Safety and Efficacy of Long-Term Rilonacept Treatment for the Prevention of Gout Flares

NCT01459796
View directory record Official record
Conditions
Gout
Interventions
Rilonacept, Placebo, Allopurinol
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 80 Years
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
74
States / cities
Birmingham, Alabama • Mesa, Arizona • Phoenix, Arizona + 71 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2017 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

NCT04990388
View directory record Official record
Conditions
Glycogen Storage Disease Type III
Interventions
UX053, Placebo, Antipyretic, H2 Blocker, H1 Blocker
Biological · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
4
States / cities
Orange, California • Atlanta, Georgia • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 29, 2026, 2:07 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Drug Discovery for Parkinson's With Mutations in the GBA Gene

NCT05536388
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease, Healthy, GBA Gene Mutation
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 29, 2026, 2:07 PM EDT
Recruiting No phase listed Observational

Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress

NCT04541654
View directory record Official record
Conditions
Li-Fraumeni Syndrome, TP53 Gene Mutation, Hereditary Cancer Syndrome, Clonal Hematopoiesis, Mosaicism
Interventions
Data and Specimen Collection
Genetic
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
3
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 29, 2026, 2:07 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 29, 2026, 2:07 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Comparison of KADIAN 100 mg When Dosed With Alcohol Under Fasting and Fed Conditions Compared to Water

NCT00768183
View directory record Official record
Conditions
Healthy
Interventions
KADIAN Capsule + alcohol (under fasting conditions), KADIAN Capsule + water (under fasting conditions), morphine sulfate IR oral solution + water, KADIAN Capsule + alcohol (under fed conditions)
Drug
Lead sponsor
Actavis Inc.
Industry
Eligibility
21 Years to 40 Years · Male only
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006
U.S. locations
1
States / cities
Lincoln, Nebraska
Source: ClinicalTrials.gov public record
Updated Jul 16, 2020 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

NCT00697073
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 18 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 11, 2023 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Phase 1 Interventional

A Study of Foscarnet Plus Ganciclovir in the Treatment of Cytomegalovirus of the Eye in Patients With AIDS Who Have Already Been Treated With Ganciclovir

NCT00000970
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Phase 3 Interventional Results available

Clinical Importance of Treating Iron Overload in Sickle Cell Disease

NCT00981370
View directory record Official record
Conditions
Anemia, Sickle Cell, Transfusion Hemosiderosis
Interventions
deferasirox
Drug
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
14 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 8, 2017 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Phase 2 Interventional

Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

NCT00280995
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
ISIS 301012
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
5
States / cities
Chicago, Illinois • New York, New York • Winston-Salem, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2016 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Phase 3 Interventional Results available

An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

NCT03938272
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 8, 2021 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Phase 2 Interventional

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

NCT04732429
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
HST5040, Placebo
Drug
Lead sponsor
HemoShear Therapeutics
Industry
Eligibility
2 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
14
States / cities
San Diego, California • New Haven, Connecticut • Washington D.C., District of Columbia + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 29, 2026, 2:07 PM EDT
Active, not recruiting Phase 2 Interventional

Metyrapone for Mild Autonomous Cortisol Secretion (MACS)

NCT06106295
View directory record Official record
Conditions
Autonomous Cortisol Secretion, Mild Autonomous Cortisol Secretion (MACS)
Interventions
Metyrapone
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 29, 2026, 2:07 PM EDT
Recruiting No phase listed Observational

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
9,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2034
U.S. locations
91
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 70 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
8 Months and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Phase 2 Interventional

CMV Retinitis Retreatment Trial

NCT00000766
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2011 · Synced Oct 29, 2026, 2:07 PM EDT
Completed Not applicable Interventional

A Study of Valganciclovir in the Treatment of Cytomegalovirus (CMV) Retinitis in Patients With AIDS

NCT00002222
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 29, 2026, 2:07 PM EDT
Completed No phase listed Observational

Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))

NCT01880983
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP)
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
7 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2020
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Phase 2 Interventional Results available

Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

NCT02093221
View directory record Official record
Conditions
Type 1 Diabetes Mellitus
Interventions
180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI, Placebo
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
6 Years to 35 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
36
States / cities
Tucson, Arizona • Los Angeles, California • Orange, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 5, 2018 · Synced Oct 29, 2026, 2:07 PM EDT
Terminated Not applicable Interventional

Adaptive Device for Insulin Pens for Arthritic Patients

NCT02779816
View directory record Official record
Conditions
Diabetes Mellitus, Arthritis
Interventions
Pen device
Device
Lead sponsor
Levenson, David I., M.D.
Individual
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Boca Raton, Florida
Source: ClinicalTrials.gov public record
Updated Aug 14, 2019 · Synced Oct 29, 2026, 2:07 PM EDT
Recruiting No phase listed Observational

Natural History Study of Parathyroid Disorders

NCT04969926
View directory record Official record
Conditions
Parathyroid Cancer, Primary Hyperparathyroidism, Pseudohypoparathyroidism, Inheritable Bone Diseases, Multiple Endocrine Neoplasia, Type 1
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
6 Months to 100 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2031
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 29, 2026, 2:07 PM EDT