Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Nov 5, 2026, 10:38 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 937–960 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Fabry Disease
Interventions: AT1001 150 mg; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2012
U.S. locations: 4
States / cities: Costa Mesa, California • Miami, Florida • Orlando, Florida

Conditions: Gaucher Disease Type I, Oxidative Stress, Inflammation
Interventions: Not listed
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years to 75 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2026
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Primary Hyperoxaluria
Interventions: DCR-PHXC, Placebo; Drug
Lead sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company; Industry
Eligibility: 6 Years and older
Enrollment: 43 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Parkinson Disease (PD), Type 1 Gaucher Disease
Interventions: Not listed
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: 40 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: New York, New York

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

Conditions: Barth Syndrome
Interventions: Resistance exercise and protein supplementation; Behavioral
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 15 Years to 35 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2019
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Fabry Disease
Interventions: Migalastat HCl 20 mg; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 6
States / cities: Atlanta, Georgia • Minneapolis, Minnesota • Charlotte, North Carolina + 3 more

Conditions: Morquio A Syndrome, Mucopolysaccharidosis IV A
Interventions: Not listed
Lead sponsor: Nadia Ali, PhD; Other
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Decatur, Georgia

Conditions: Cerebrotendinous Xanthomatosis (CTX)
Interventions: Not listed
Lead sponsor: Travere Therapeutics, Inc.; Industry
Eligibility: 2 Years to 21 Years
Enrollment: 442 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 44
States / cities: Birmingham, Alabama • La Jolla, California • Los Angeles, California + 38 more

Conditions: Hypertriglyceridemia, Hyperlipoproteinemia Type IV
Interventions: torcetrapib/atorvastatin, atorvastatin; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 39
States / cities: Anaheim, California • Huntington Beach, California • Los Angeles, California + 36 more

Conditions: Leigh Syndrome
Interventions: None (Observational study); Other
Lead sponsor: Taysha Gene Therapies, Inc.; Industry
Eligibility: Up to 18 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Gaucher Disease Type 1
Interventions: FLT201; Genetic
Lead sponsor: Spur Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 15
States / cities: Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more

Conditions: Hypophosphatasia
Interventions: asfotase alfa; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 99 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: Palm Harbor, Florida

Conditions: Mucopolysaccharidosis Type IIIB, MPS III B
Interventions: AX 250; Drug
Lead sponsor: Allievex Corporation; Industry
Eligibility: 0 Years to 18 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 2
States / cities: Oakland, California • Pittsburgh, Pennsylvania

Conditions: X-Linked Hypophosphatemia
Interventions: KRN23; Biological
Lead sponsor: Kyowa Kirin, Inc.; Industry
Eligibility: Not listed
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 5
States / cities: San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more

Conditions: Gout, Hyperuricemia, Gout Flare, Tophi
Interventions: Tigulixostat, Allopurinol, Placebo; Drug
Lead sponsor: LG Chem; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 2,202 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 124
States / cities: Birmingham, Alabama • Daphne, Alabama • Mobile, Alabama + 91 more

Conditions: Adrenal Hyperplasia, Congenital, Congenital Hypothyroidism, Cystic Fibrosis
Interventions: Not listed
Lead sponsor: Luminex Corporation; Industry
Eligibility: 1 Day to 7 Days
Enrollment: 7,462 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2012
U.S. locations: 3
States / cities: Albany, New York • Nashville, Tennessee • Taylorsville, Utah

Conditions: Lipodystrophy
Interventions: Not listed
Lead sponsor: PatientCrossroads; Industry
Eligibility: Not listed
Enrollment: 257 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Fabry Disease
Interventions: 4D-310; Biological
Lead sponsor: 4D Molecular Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2030
U.S. locations: 4
States / cities: La Jolla, California • Atlanta, Georgia • Pittsburgh, Pennsylvania + 1 more

Conditions: Gout, Chronic Kidney Disease (CKD)
Interventions: Lesinurad, XOI, Placebo, colchicine, corticosteroids; Drug
Lead sponsor: Ironwood Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 242 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 70
States / cities: Birmingham, Alabama • Tucson, Arizona • Covina, California + 59 more

Conditions: Gout, Hyperuricemia
Interventions: SAP-001; Drug
Lead sponsor: Shanton Pharma Pte. Ltd.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2025
U.S. locations: 2
States / cities: Tampa, Florida • Saint Paul, Minnesota

Conditions: Mucopolysaccharidosis II
Interventions: iduronate-2-sulfatase enzyme replacement therapy, Placebo; Biological
Lead sponsor: Shire; Industry
Eligibility: 5 Years to 25 Years · Male only
Enrollment: 96 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2005
U.S. locations: 4
States / cities: Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more

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

Conditions: Mucopolysaccharidosis I, Cognitive Decline
Interventions: Intrathecal recombinant human alpha iduronidase; Drug
Lead sponsor: agnes chen; Other
Eligibility: 6 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 1
States / cities: Torrance, California

Search by objective public record fields.

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria

Parkinson's Disease in Type 1 Gaucher Disease

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

Resistance Exercise in Barth Syndrome

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

A Study Comparing The Efficacy & Safety Of Torcetrapib/Atorvastatin And Atorvastatin In Subjects With High Triglycerides

A Study of the Natural Course of SURF1 Deficiency

A Gaucher Disease Gene Therapy Trial With FLT201

Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients

Clinical Evaluation of NeoPlex4 Assay and NeoPlex System

Lipodystrophy Connect Patient Registry

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

A Phase 4 Safety and Efficacy Study to Evaluate Lesinurad 200 mg in Participants With Gout and Renal Impairment

PK and Safety Evaluation Study of SAP-001 in Adult Subjects With Normal and Impaired Renal Function

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

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

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I