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ClinicalTrials.gov public records Last synced Oct 20, 2026, 11:04 PM EDT

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Showing 97–120 of 1,509 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Recruiting Phase 2 Interventional

A Study to Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)

NCT07280156
View directory record Official record
Conditions
Gout
Interventions
PRX-115, Methotrexate (MTX), PRX-115 placebo, Placebo-Methotrexate
Biological · Drug · Other
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
26
States / cities
Glendale, Arizona • Little Rock, Arkansas • Anaheim, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 3 Interventional Results available

A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH)

NCT05952869
View directory record Official record
Conditions
Hypercholesterolemia, Familial Hypercholesterolemia
Interventions
Enlicitide Decanoate, Placebo
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
303 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
13
States / cities
Daphne, Alabama • Boca Raton, Florida • Cutler Bay, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2026 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

NCT02124083
View directory record Official record
Conditions
Neimann-Pick Disease
Interventions
Vorinostat
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 22, 2018 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

RDEA3170 Bioavailability Study

NCT02336594
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 10 mg, RDEA3170 2.5 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Oct 13, 2017 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Phenylbutyrate Therapy for Maple Syrup Urine Disease

NCT01529060
View directory record Official record
Conditions
Maple Syrup Urine Disease
Interventions
Phenylbutyrate, Placebo powder
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
3 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 19, 2019 · Synced Oct 20, 2026, 11:04 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 20, 2026, 11:04 PM EDT
Active, not recruiting Phase 2 Interventional

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

NCT04551170
View directory record Official record
Conditions
Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy, Pseudohypoparathyroidism Type 1a
Interventions
Theophylline, Placebo
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Oct 20, 2026, 11:04 PM 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 20, 2026, 11:04 PM EDT
Enrolling by invitation Phase 3 Interventional

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265
View directory record Official record
Conditions
Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
Interventions
No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 2 Interventional Results available

Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C)

NCT02597127
View directory record Official record
Conditions
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes
Interventions
ALN-PCSSC, Normal Saline
Drug
Lead sponsor
The Medicines Company
Industry
Eligibility
18 Years to 99 Years
Enrollment
501 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
8
States / cities
Jacksonville, Florida • Indianapolis, Indiana • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated May 17, 2019 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 4 Interventional Results available

Study of KRYSTEXXA® (Pegloticase) Plus Methotrexate in Participants With Uncontrolled Gout

NCT03994731
View directory record Official record
Conditions
Gout
Interventions
Pegloticase, methotrexate, placebo, folic acid, gout flare prophylaxis regimen, fexofenadine, acetaminophen, methylprednisolone
Biological · Drug · Dietary Supplement
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
60
States / cities
Birmingham, Alabama • Anchorage, Alaska • Flagstaff, Arizona + 54 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Oct 20, 2026, 11:04 PM EDT
Completed No phase listed Observational

Health-related Quality of Life in Rare Kidney Stone

NCT02124395
View directory record Official record
Conditions
Primary Hyperoxaluria, Cystinuria, Adenine Phosphoribosyl Transferase Deficiency, Dent Disease
Interventions
Health-related Quality of Life in Rare Kidney Stone Questionnaire
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years and older
Enrollment
365 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
2
States / cities
Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 2, 2022 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 1 Interventional

Effect of S-adenosylmethionine (SAMe) on Blood Levels of Homocysteine

NCT00473200
View directory record Official record
Conditions
Hyperhomocysteinemia
Interventions
S-adenosylmethionine
Dietary Supplement
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 14, 2018 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Canakinumab Prefilled Syringes in Frequently Flaring Acute Gouty Arthritis Patients

NCT01356602
View directory record Official record
Conditions
Acute Gouty Arthritis
Interventions
Canakinumab pre-filled syringe, Canakinumab lyophilized powder, Triamcinolone Acetonide, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
397 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
69
States / cities
Anniston, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 62 more
Source: ClinicalTrials.gov public record
Updated Jan 29, 2014 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Study to Investigate the Effects of Food on Relative Bioavailability of ABP-671 Tablets in Healthy Subjects

NCT04303039
View directory record Official record
Conditions
Gout, Food-drug Interaction
Interventions
ABP-671
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 23, 2020 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes

NCT07408518
View directory record Official record
Conditions
Digestive Disease, Gut Health
Interventions
Probiotic, Placebo
Dietary Supplement · Other
Lead sponsor
Synbiotic Health
Industry
Eligibility
18 Years to 75 Years
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 20, 2026, 11:04 PM EDT
Recruiting No phase listed Observational

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

NCT05040178
View directory record Official record
Conditions
Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia
Interventions
Carglumic Acid
Drug
Lead sponsor
RECORDATI GROUP
Industry
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2025 · Synced Oct 20, 2026, 11:04 PM EDT
Enrolling by invitation Phase 2 Interventional Accepts healthy volunteers

Theophylline for Treatment of Pseudohypoparathyroidism

NCT04240821
View directory record Official record
Conditions
Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Interventions
Theophylline ER
Drug
Lead sponsor
Jaclyn Tamaroff
Other
Eligibility
2 Years to 99 Years
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 20, 2026, 11:04 PM EDT
Completed No phase listed Observational

Registry of Fabry Disease - A Multicenter Observational Study

NCT00055016
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
2
States / cities
Bethesda, Maryland • Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 2 Interventional Results available

Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT02265952
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Oral Febuxostat in Participants With Gout

NCT00430248
View directory record Official record
Conditions
Gout
Interventions
Febuxostat, Allopurinol
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years to 85 Years
Enrollment
2,269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
258
States / cities
Birmingham, Alabama • Hoover, Alabama • Hueytown, Alabama + 255 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2012 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 3 Interventional

Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

NCT04034485
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
lerodalcibep, evolocumab
Drug
Lead sponsor
LIB Therapeutics LLC
Industry
Eligibility
10 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
2
States / cities
Evanston, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 29, 2023 · Synced Oct 20, 2026, 11:04 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 20, 2026, 11:04 PM EDT
Completed Phase 1 Interventional

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

NCT00692926
View directory record Official record
Conditions
MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
Interventions
ALDHbr Umbilical Cord Blood Cells
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 55 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 29, 2017 · Synced Oct 20, 2026, 11:04 PM EDT