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 Oct 24, 2026, 9:44 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 481–504 of 1,510 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions: BMN 110; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Up to 5 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 2
States / cities: Oakland, California • Manhasset, New York

Conditions: Mucopolysaccharidosis Type IIIA
Interventions: DNL126; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 0 Years to 18 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 4
States / cities: Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more

Conditions: Hypophosphatasia (HPP)
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 5 Years and older
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014
U.S. locations: 9
States / cities: Atlanta, Georgia • Indianapolis, Indiana • Boston, Massachusetts + 6 more

Conditions: Familial Chylomicronemia
Interventions: Plozasiran, Placebo; Drug
Lead sponsor: Arrowhead Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 9
States / cities: Boca Raton, Florida • Suwanee, Georgia • Indianapolis, Indiana + 5 more

Conditions: Pompe Disease (Late-onset)
Interventions: Clenbuterol, Placebos; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin Gene Mutation
Interventions: Not listed
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 30 Years to 80 Years
Enrollment: 500 participants
Timeline: 2022 – 2027
U.S. locations: 3
States / cities: New York, New York • Cleveland, Ohio • Dallas, Texas

Conditions: Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: Albert Einstein College of Medicine; Other
Eligibility: Up to 4 Weeks
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2029
U.S. locations: 9
States / cities: Brooklyn, New York • Manhasset, New York • New York, New York + 2 more

Conditions: Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
Interventions: laronidase; Drug
Lead sponsor: Patricia I. Dickson, M.D.; Individual
Eligibility: 6 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2015
U.S. locations: 3
States / cities: Oakland, California • Torrance, California • Minneapolis, Minnesota

Conditions: Fed
Interventions: CIPRO®XR; Drug
Lead sponsor: Dr. Reddy's Laboratories Limited; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Globulin, Immune, Ganciclovir; Drug
Lead sponsor: American National Red Cross; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions: Not listed
Lead sponsor: University of Minnesota; Other
Eligibility: Not listed
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2027
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Gaucher Disease
Interventions: Not listed
Lead sponsor: Yale University; Other
Eligibility: 3 Months to 75 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: GM1 Gangliosidosis
Interventions: Natural history; Other
Lead sponsor: LYSOGENE; Industry
Eligibility: Not listed
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Plymouth, Massachusetts

Conditions: Methylmalonic Acidemia
Interventions: mRNA-3705; Drug
Lead sponsor: ModernaTX, Inc.; Industry
Eligibility: 1 Year and older
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2034
U.S. locations: 3
States / cities: Los Angeles, California • Palo Alto, California • San Diego, California

Conditions: X-linked Hypophosphatemia
Interventions: Burosumab Injection [Crysvita]; Drug
Lead sponsor: Yale University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Fabry Disease
Interventions: Pegunigalsidase alfa; Biological
Lead sponsor: Protalix; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 7
States / cities: Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Gaucher's Disease
Interventions: Eliglustat; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 18 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Fabry's Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 325 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1995 – 2008
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Gout, Hyperuricemia
Interventions: Tranilast, Allopurinol, Combination - Tranilast and Allopurinol, 400 mg Allopurinol, High dose combination; Drug
Lead sponsor: Nuon Therapeutics, Inc.; Industry
Eligibility: 21 Years to 70 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010
U.S. locations: 3
States / cities: Honolulu, Hawaii • Evansville, Indiana • Dallas, Texas

Conditions: Neuronal Ceroid Lipofuscinosis Type 2
Interventions: Cerliponase Alfa; Drug
Lead sponsor: David L Rogers, MD; Other
Eligibility: 24 Months to 72 Months
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 1
States / cities: Columbus, Ohio

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

Conditions: Hypophosphatasia
Interventions: asfotase alfa; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 13 Years to 65 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 2
States / cities: St Louis, Missouri • Durham, North Carolina

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

Search by objective public record fields.

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Bioavailability Study of Ciprofloxacin 1000 mg ER Tablets of Dr. Reddy's Under Non-Fasting Conditions

A Pilot Study to Obtain Preliminary Information Regarding the Efficacy and Safety of the Combination of Immune Globulin and Ganciclovir as Compared to Ganciclovir Alone in the Treatment of Sight-Threatening CMV Retinitis in Patients With AIDS

A Natural History Study of the Gangliosidoses

Advancing Integrated Therapies for Gaucher Disease

Interviews and Video Capture in Patients With GM1 Gangliosidosis

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

Examining the Effect of Burosumab on Muscle Function

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

An Open-Label Safety Study of Oral Ganciclovir Maintenance Treatment of CMV Retinitis in People With Limited Venous Access

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

Analysis of the Nervous System in Patients With Fabry's Disease

Study of Tranilast Alone or in Combination With Allopurinol in Subjects With Hyperuricemia

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

Registry of Fabry Disease - A Multicenter Observational Study

Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)

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