Search | Clinical Trials Finder

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ClinicalTrials.gov public records Last synced Nov 6, 2026, 7:52 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 985–1008 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions: Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 135 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1995 – 2010
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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

Conditions: Smith-Lemli-Opitz Syndrome
Interventions: diet; Behavioral
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1995
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Familial Hypercholesterolaemia
Interventions: rosuvastatin calcium; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 315 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: DHDDS-Congenital Disorder of Glycosylation, DHDDS-CDG, Congenital Disorder of Glycosylation
Interventions: Nicotinamide Mononucleotide (NMN) Nucleosidase; Drug
Lead sponsor: Eva Morava-Kozicz; Other
Eligibility: 4 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: New York, New York

Conditions: Generalised Lipodystrophy, Partial Lipodystrophy
Interventions: Metreleptin; Drug
Lead sponsor: Chiesi Farmaceutici S.p.A.; Industry
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2031
U.S. locations: 16
States / cities: Birmingham, Alabama • Duarte, California • Atlanta, Georgia + 11 more

Conditions: Fabry Disease
Interventions: migalastat HCl; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 65 Years · Female only
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 1
States / cities: Decatur, Georgia

Conditions: Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
Interventions: Not listed
Lead sponsor: Alnylam Pharmaceuticals; Industry
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2033
U.S. locations: 13
States / cities: La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more

Conditions: Gout, Hyperuricemia, Gout Flare, Tophi
Interventions: Tigulixostat, Placebo; Drug
Lead sponsor: LG Chem; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 354 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 36
States / cities: Birmingham, Alabama • Covina, California • Clearwater, Florida + 31 more

Conditions: Hypercholesterolemia, Familial, Hyperlipidemia
Interventions: torcetrapib/atorvastatin, atorvastatin; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 14
States / cities: San Diego, California • Washington D.C., District of Columbia • Lutz, Florida + 11 more

Conditions: Fabry Disease
Interventions: Fabrazyme (agalsidase beta); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 7 Years to 15 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2005
U.S. locations: 1
States / cities: Tucson, Arizona

Conditions: Mucopolysaccharidosis I
Interventions: JR-171; Drug
Lead sponsor: JCR Pharmaceuticals Co., Ltd.; Industry
Eligibility: Not listed
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Oakland, California

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

Conditions: Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions: Abatacept; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Prostate Cancer, Renal Cancer, Urethral Cancer, Advanced Solid Tumor, Metastatic Castration-resistant Prostate Cancer, Solid Tumor, Adult
Interventions: Copper-64 labeled Granzyme B (64Cu-GRIP B), Positron Emission Tomography (PET); Drug · Procedure
Lead sponsor: Rahul Aggarwal; Other
Eligibility: 18 Years and older
Enrollment: 91 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: San Francisco, California

Conditions: G6PD Deficiency
Interventions: RPQ, SPQ, Placebo; Drug
Lead sponsor: University of Mississippi, Oxford; Other
Eligibility: 18 Years to 65 Years
Enrollment: 18 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: University, Mississippi

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 700 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 19
States / cities: Berkeley, California • Los Angeles, California • San Diego, California + 12 more

Conditions: Fabry Disease, Healthy
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Male only
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2000 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions: Not listed
Lead sponsor: Children's Hospital of Orange County; Other
Eligibility: 4 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 1
States / cities: Orange, California

Conditions: Gout
Interventions: RDEA3170 and Placebo; Drug
Lead sponsor: Ardea Biosciences, Inc.; Industry
Eligibility: 20 Years to 55 Years · Male only
Enrollment: 46 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013
U.S. locations: 1
States / cities: Glendale, California

Conditions: Prehypertension, Gout, Pulse Wave Velocity, Hypertension, 24 Hour Blood Pressure
Interventions: Febuxostat; Drug
Lead sponsor: University of California, San Diego; Other
Eligibility: 18 Years to 50 Years
Enrollment: 47 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2014
U.S. locations: 1
States / cities: San Diego, California

Conditions: Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions: Clofarabine, Fludarabine Injection; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 17 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Mucopolysaccharidosis II
Interventions: tividenofusp alfa; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: Up to 18 Years
Enrollment: 99 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 8
States / cities: Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more

Conditions: Gout
Interventions: lesinurad, febuxostat; Drug
Lead sponsor: Ardea Biosciences, Inc.; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 196 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 55
States / cities: Birmingham, Alabama • Tempe, Arizona • Glendale, California + 50 more

Search by objective public record fields.

Stem Cell Transplant for Inborn Errors of Metabolism

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)

Phase II Study of Dietary Cholesterol for Smith-Lemli-Opitz Syndrome

An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

Assessing the Safety and Tolerability of NMN in DHDDS-CDG

MEASuRE: Metreleptin Effectiveness And Safety Registry

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Tigulixostat, Phase 3 Study, Placebo Controlled in Gout Patients

Study To Evaluate The Safety And Efficacy Of Torcetrapib/Atorvastatin In Subjects With Familial Hypercholerolemia

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

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

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

64Cu-GRIP B in Patients With Advanced Malignancies

Primaquine Enantiomers in G6PD Deficient Human Volunteers

A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus

PET Scans in Normal Volunteers and Patients With Fabry Disease

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

Single and Multiple Dose Study in Japanese Subjects

Study of Febuxostat Effect on Blood Pressure in Patients With High Normal Blood Pressure

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Lesinurad and Febuxostat Combination Extension Study in Gout