Search | Clinical Trials Finder

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ClinicalTrials.gov public records Last synced Nov 4, 2026, 2:53 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 865–888 of 1,512 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions: sebelipase alfa; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2017
U.S. locations: 5
States / cities: Eureka, California • Sacramento, California • San Francisco, California + 2 more

Conditions: Parkinson Disease, GBA Gene Mutation, Gaucher Disease
Interventions: PET scan, neuroQWERTY; Diagnostic Test
Lead sponsor: Pacific Parkinson's Research Centre; Other
Eligibility: 18 Years to 80 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 2
States / cities: Portland, Oregon • Seattle, Washington

Conditions: Phenylketonuria
Interventions: Dietary Protein Counting and Free Fruits and Vegetables; Behavioral
Lead sponsor: University of Nebraska; Other
Eligibility: 13 Years to 65 Years
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 4
States / cities: Omaha, Nebraska

Conditions: Wilson Disease
Interventions: Standard of Care Medications; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 5
States / cities: New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more

Conditions: Congenital Adrenal Hyperplasia
Interventions: Lu AG13909; Drug
Lead sponsor: H. Lundbeck A/S; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Parkinson Disease, Gaucher Disease
Interventions: Not listed
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 50 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Lactose Intolerance
Interventions: RP-G28, Placebo; Drug · Other
Lead sponsor: Ritter Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 377 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016
U.S. locations: 1
States / cities: Princeton, New Jersey

Conditions: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Metabolic Syndrome
Interventions: ELM Group, ELM Classes, ELM Individual; Behavioral · Other
Lead sponsor: Rush University Medical Center; Other
Eligibility: 18 Years to 72 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2021
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Adrenoleukodystrophy
Interventions: Leriglitazone, Placebo; Drug
Lead sponsor: Minoryx Therapeutics, S.L.; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 116 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2025
U.S. locations: 3
States / cities: Stanford, California • Baltimore, Maryland • Boston, Massachusetts

Conditions: Pompe Disease
Interventions: rAAV1-CMV-GAA (study agent) Administration, RMST; Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 2 Years to 18 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD), Nervous System Diseases, Brain Diseases, Central Nervous System Diseases, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Sphingolipidoses, Hereditary Central Nervous System Demyelinating Diseases, Metabolic Inborn Brain Diseases, Lysosomal Storage Diseases, Metabolic Diseases, Sulfatidosis
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: Up to 12 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 4
States / cities: Torrance, California • Washington D.C., District of Columbia • Chicago, Illinois + 1 more

Conditions: Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Week to 110 Years
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 1995
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Primary Hyperoxaluria Type 3
Interventions: Not listed
Lead sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company; Industry
Eligibility: 2 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 2
States / cities: Boston, Massachusetts • New York, New York

Conditions: Congenital Adrenal Hyperplasia
Interventions: Crinecerfont, Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 18 Years and older
Enrollment: 182 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 20
States / cities: Los Angeles, California • San Diego, California • San Francisco, California + 17 more

Conditions: Metachromatic Leukodystrophy
Interventions: Warfarin; Drug
Lead sponsor: The Cooper Health System; Other
Eligibility: 1 Year to 10 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 1
States / cities: Camden, New Jersey

Conditions: Hypercholesterolemia, Familial Hypercholesterolemia
Interventions: Enclitide Chloride, Placebo; Drug
Lead sponsor: Merck Sharp & Dohme LLC; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 381 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022
U.S. locations: 20
States / cities: Beverly Hills, California • Sacramento, California • Santa Ana, California + 17 more

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Zodasiran; Drug
Lead sponsor: Arrowhead Pharmaceuticals; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Parathyroid Cancer, Primary Hyperparathyroidism, Pseudohypoparathyroidism, Inheritable Bone Diseases, Multiple Endocrine Neoplasia, Type 1
Interventions: Not listed
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: 6 Months to 100 Years
Enrollment: 3,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2031
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Phenylketonurias
Interventions: Pegvaliase; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 18 Years to 65 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Congenital Adrenal Hyperplasia
Interventions: Pre-Screening; Other
Lead sponsor: Adrenas Therapeutics Inc; Industry
Eligibility: 18 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Gaucher's Disease
Interventions: human glucocerebrosidase gene into autologous peripheral blood stem cells; Genetic
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 18 Years to 65 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1999
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Amino Acid Metabolism, Inborn Errors
Interventions: Protein and calorie controlled diet, Ornithine transcarbamylase vector; Behavioral · Genetic
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 6 Months to 64 Years
Enrollment: 66 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 1999
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Bloating
Interventions: OPTI-BIOME™ Bacillus subtilis MB40, Placebo; Dietary Supplement · Other
Lead sponsor: BIO-CAT Microbials, LLC; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2017
U.S. locations: 2
States / cities: Foothill Ranch, California • Orlando, Florida

Search by objective public record fields.

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

The GBA Multimodal Study in Parkinson's Disease

Simplified Diet Approach in Phenylketonuria

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

Biomarker Analysis for GBA Associated Parkinson's Disease

Efficacy, Safety, and Tolerability Study of RP-G28 in Subjects With Lactose Intolerance

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

A Pilot Study of a Lifestyle Intervention on the Metabolic Syndrome (3ELM Study)

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Natural History Study of Children With Metachromatic Leukodystrophy

Genetic Studies of Lysosomal Storage Disorders

Natural History of Patients With PH3 and a History of Stone Events

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

A Study of the Efficacy and Safety of Enclitide Chloride (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-008)

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Natural History Study of Parathyroid Disorders

Nutrition Status of Adults Treated With Pegvaliase

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population