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 15, 2026, 2:16 PM 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 1297–1320 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Phenylketonuria (PKU)
Interventions: Pegvaliase; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 17
States / cities: Gainesville, Florida • Tampa, Florida • Chicago, Illinois + 14 more

Conditions: Behavioral Manifestations of Lesch-Nyhan Disease
Interventions: sapropterin; Drug
Lead sponsor: University of California, San Diego; Other
Eligibility: 4 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 1
States / cities: San Diego, California

Conditions: Phenylketonuria
Interventions: rAvPAL-PEG; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 16 Years to 70 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2015
U.S. locations: 9
States / cities: Aurora, Colorado • Gainesville, Florida • Louisville, Kentucky + 6 more

Conditions: Fabry Disease
Interventions: migalastat HCl; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 3
States / cities: Decatur, Georgia • New York, New York • Dallas, Texas

Conditions: Cardiomyopathy, Ischemic Heart Disease, Cardiac Amyloidosis
Interventions: Transthoracic Echocardiography (TTE); Diagnostic Test
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 10,040,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Glycogen Storage Disease Type II, Glycogenosis 2
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 8
States / cities: Aurora, Colorado • Wichita, Kansas • Cambridge, Massachusetts + 5 more

Conditions: Progeria, Hutchinson-Gilford Syndrome
Interventions: Lonafarnib, Zoledronic Acid, Pravastatin; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: Not listed
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Fabry Disease
Interventions: pegunigalsidase alfa; Drug
Lead sponsor: Chiesi Farmaceutici S.p.A.; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 97 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 15
States / cities: Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 11 more

Conditions: Fabry Disease
Interventions: Agalsidase alfa; Drug
Lead sponsor: Shire; Industry
Eligibility: 7 Years to 17 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2011
U.S. locations: 13
States / cities: Tucson, Arizona • Palm Beach Gardens, Florida • Lake Charles, Louisiana + 9 more

Conditions: Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
Interventions: Evuzamitide; Drug
Lead sponsor: Lorena Saelices; Other
Eligibility: Not listed
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 3
States / cities: Boston, Massachusetts • New York, New York • Dallas, Texas

Conditions: Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism, Pseudohypoparathyroidism Type Ia, Albright Hereditary Osteodystrophy
Interventions: Not listed
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 6 Years to 50 Years
Enrollment: 14 participants
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Fabry Disease
Interventions: Venglustat (GZ402671), Placebo; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 16 Years and older
Enrollment: 122 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 11
States / cities: Birmingham, Alabama • Los Angeles, California • Orange, California + 8 more

Conditions: Fabry Disease
Interventions: A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat); Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011
U.S. locations: 1
States / cities: Austin, Texas

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Cidofovir, Probenecid, Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 13 Years and older
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 12
States / cities: Los Angeles, California • Miami, Florida • Atlanta, Georgia + 7 more

Conditions: Pompe Disease
Interventions: Observational; Other
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2029
U.S. locations: 1
States / cities: Durham, North Carolina

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: Arthritis, Gouty
Interventions: canakinumab, dexamethasone, placebo matching canakinumab, placebo matching dexamethasone; Biological · Drug · Other
Lead sponsor: Novartis; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 2
States / cities: Birmingham, Alabama • New Brunswick, New Jersey

Conditions: Lysosomal Storage Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2008
U.S. locations: 2
States / cities: San Francisco, California • Washington D.C., District of Columbia

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 225 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 19
States / cities: Berkeley, California • Los Angeles, California • San Diego, California + 12 more

Conditions: Wilson Disease
Interventions: Not listed
Lead sponsor: Yale University; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: New Haven, Connecticut

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

Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions: Vutrisiran, Sterile Normal Saline (0.9% NaCl); Drug
Lead sponsor: Alnylam Pharmaceuticals; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 655 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 17
States / cities: La Mesa, California • Los Angeles, California • Stanford, California + 13 more

Conditions: Rheumatoid Arthritis, Osteoarthritis, Gout
Interventions: Not listed
Lead sponsor: American College of Rheumatology; Other
Eligibility: 18 Years and older
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2014
U.S. locations: 1
States / cities: Atlanta, Georgia

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: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026
U.S. locations: 1
States / cities: Cary, North Carolina

Search by objective public record fields.

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Trial of Kuvan in Lesch-Nyhan Disease

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

A Study Of Deep Learning For Echo Analysis, Tracking, And Evaluation

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

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

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels

Glucose Homeostasis in Pseudohypoparathyroidism

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Migalastat Food Effect Study

Comparison of Two Drugs, Cidofovir and Ganciclovir, in Treating Patients With AIDS Who Have CMV Retinitis

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Efficacy and Safety of a Single Dose of Canakinumab (ACZ885) in Hospitalized Patients With Acute Gout

The Classification and Cause of Leukodystrophies of Unknown Cause

A Randomized Study Comparing the Safety and Efficacy of Two Regimens of Oral Ganciclovir to Intravenous Ganciclovir Maintenance Therapy for Cytomegalovirus Retinitis in People With AIDS Who Have Received Prior Ganciclovir Therapy

Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

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

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Rheumatology Informatics System for Effectiveness

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