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ClinicalTrials.gov public records Last synced Oct 4, 2026, 10:23 AM EDT

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Showing 49–72 of 1,957 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Disorders of Metabolism Clear all
Recruiting Phase 1 Interventional

Drug-Drug Interaction Study of ABP-671 in Gout Patients

NCT07367971
View directory record Official record
Conditions
Gout Chronic, Gout, DDI (Drug-Drug Interaction)
Interventions
ABP-671 plus allopurinol
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Cary, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Efficay and Safety of LC350189 Different Doses in Gout Patients With Hyperuricemia

NCT03934099
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
LC350189 formulated capsule, Placebo
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 75 Years
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
34
States / cities
Birmingham, Alabama • Chandler, Arizona • Mesa, Arizona + 28 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 3 Interventional

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00074971
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2004
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 4 Interventional Results available

Late-Onset Treatment Study Extension Protocol

NCT00455195
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
26
States / cities
Anchorage, Alaska • Phoenix, Arizona • Beverly Hills, California + 23 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 4 Interventional

Weight Loss in Response to Sibutramine (MERIDIA) is Influenced by the Inherited Genes

NCT00433641
View directory record Official record
Conditions
Obesity
Interventions
sibutramine
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 65 Years
Enrollment
181 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 14, 2011 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

NCT02445794
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Low dose cohort, High dose cohort
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
18 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Long Beach, California • Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 27, 2020 · Synced Oct 4, 2026, 10:23 AM EDT
Terminated Phase 4 Interventional Results available

Immune Tolerance Induction Study

NCT00701701
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Myozyme® (alglucosidase alfa)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Month and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
4
States / cities
Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2022 · Synced Oct 4, 2026, 10:23 AM EDT
Recruiting Phase 3 Interventional

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

NCT06128629
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
NTLA-2001, Placebo
Biological · Drug
Lead sponsor
Intellia Therapeutics
Industry
Eligibility
18 Years to 90 Years
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
38
States / cities
Tucson, Arizona • Los Angeles, California • Palo Alto, California + 29 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Assessment of the Effects of a DPP-4 Inhibitor (Sitagliptin) Januvia on Immune Function in Healthy Individuals

NCT00813228
View directory record Official record
Conditions
Diabetes Mellitus Type 1, Diabetes Mellitus Type 2
Interventions
Sitagliptin (Januvia)
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 29, 2019 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional

Open Label Safety/Efficacy Study of Arhalofenate in Combination With Febuxostat for Hyperuricemia in Gout Patients

NCT01416402
View directory record Official record
Conditions
Hyperuricemia, Gout
Interventions
Arhalofenate, Febuxostat, Colchicine
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Apr 3, 2015 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2

NCT03847909
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease
Interventions
DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
6 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
5
States / cities
San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated May 22, 2024 · Synced Oct 4, 2026, 10:23 AM EDT
Not listed Phase 2 Interventional

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

NCT00916747
View directory record Official record
Conditions
Progeria
Interventions
Lonafarnib, Zoledronic Acid, and Pravastatin
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 27, 2023 · Synced Oct 4, 2026, 10:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Turner Syndrome: Genotype and Phenotype

NCT00006334
View directory record Official record
Conditions
Turner's Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
10 Years and older
Enrollment
1,017 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 6, 2017 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional Results available

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

NCT00283387
View directory record Official record
Conditions
Hyperoxaluria
Interventions
Betaine, Placebo
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
6 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 16, 2013 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional Results available

A Study of GLWL-01 in Patients With Prader-Willi Syndrome

NCT03274856
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
GLWL-01, Placebo
Drug
Lead sponsor
GLWL Research Inc.
Other
Eligibility
16 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
4
States / cities
San Diego, California • Gainesville, Florida • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2020 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 4 Interventional

Efficacy Study of Extended-Release Niacin/Lovastatin Versus Usual Care

NCT00345657
View directory record Official record
Conditions
Hyperlipidemia, Mixed Hyperlipidemia, Dyslipidemia
Interventions
Niacin Extended Release/Lovastatin
Drug
Lead sponsor
In His Image
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Tulsa, Oklahoma
Source: ClinicalTrials.gov public record
Updated Jun 28, 2006 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 1 Interventional

Recapturing Immune Tolerance to Pegloticase for the Management of Tophaceous Gout

NCT06186219
View directory record Official record
Conditions
Tophaceous Gout
Interventions
Rituximab
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 3 Interventional

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 4, 2026, 10:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Assessment of Primary Care Providers' Knowledge and Beliefs Regarding Gout Management

NCT01151709
View directory record Official record
Conditions
Gout
Interventions
Not listed
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
838 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 7, 2012 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

NCT01728064
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Placebo, EPI-743 400 mg, EPI-743 200 mg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 4, 2026, 10:23 AM EDT
Completed No phase listed Observational

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793
View directory record Official record
Conditions
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
Interventions
Not listed
Lead sponsor
Azafaros B.V.
Industry
Eligibility
2 Years to 20 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 3 Interventional

Personalized Information or Basic Information in Helping Patients Make Decisions About Participating in a Clinical Trial

NCT00750009
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
educational intervention
Other
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
583 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
4
States / cities
Detroit, Michigan • Cleveland, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 27, 2020 · Synced Oct 4, 2026, 10:23 AM EDT
Completed Phase 2 Interventional

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

NCT00048906
View directory record Official record
Conditions
Fabry Disease
Interventions
DRX005B
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 4, 2026, 10:23 AM EDT
Terminated Phase 3 Interventional Accepts healthy volunteers Results available

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

NCT04221451
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 4, 2026, 10:23 AM EDT